Archive for the ‘Gene Therapy Research’ Category

Cell Therapy Technologies Market Forecast 2020-2030

LONDON, March 25, 2020 /PRNewswire/ -- Consumables, Equipment, System & Software, Cell Processing, Cell Processing Equipment, Single Use Equipment, Cell Preservation, Distribution, Handling, Process Monitoring & Quality Control, Human Cells, Animal Cells, Life Sciences & Research Companies, Research Institutes

Visiongain estimates that the global cell therapy technologies market will grow at a CAGR of 15% in the first half of the forecast period. In 2020, North America is estimated to hold 38% of the global cell therapy technologies market.

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The 201-page Visiongain report provides clear detailed insight into the cell therapy technologies market. Discover the key drivers and challenges affecting the market.

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Report Scope

Global Cell Therapy Technologies Marketfrom2020-2030

Forecast of the Global Cell Therapy Technologies Market byType of Product: Consumables Equipment:Cell Processing Equipment, Single Use Equipment, Other Equipment System & Software

Forecast of the Global Cell Therapy Technologies byProcess Type: Cell processing Cell preservation, distribution and handling Process monitoring and quality control

Forecast of the Global Cell Therapy Technologies byCell Type: Human cells:Stem cells, Differentiated cells Animal cells

Forecast of the Global Cell Therapy Technologies byEnd User: Life Sciences and Research Companies Research Institutes

This report provides individual revenue forecasts to 2030 for thesenational markets: The US Canada Mexico UK Germany France Italy Spain Japan China India South Korea Singapore Malaysia Russia Brazil Argentina UAE South Africa Nigeria Mexico

Our study discusses the selectedleading companiesthat are the major players in the respiratory inhalers market: GE Healthcare Lonza Group Merck KGaA Terumo Bct, Inc. Thermo Fisher Scientific, Inc. & Other Companies

For the leading companies, we feature product portfolios, business segment breakdowns, recent developments & key expansion strategies etc.

This report discussesfactors that drive and restrainthis market. As well asopportunitiesandchallengesfaced by this market.

This report discusses thePorter's Five Forces Analysisof the Cell Therapy Technologies Market.

Key questions answered by this report: How is the Cell Therapy Technologies Market evolving? What is driving and restraining factors of the Cell Therapy Technologies Market? What are the market shares of each segment of the overall Cell Therapy Technologies Market in 2020? How will each Cell Therapy Technologies submarket segment grow over the forecast period and how much revenue will these submarkets account for in 2030? How will the market shares for each Cell Therapy Technologies submarket develop from 2021 to 2030? What will be the main driver for the overall market from 2021 to 2030? Will leading national Cell Therapy Technologies Markets broadly follow the macroeconomic dynamics, or will individual national markets outperform others? How will the market shares of the national markets change by 2030 and which geographical region will lead the market in 2030? Who are the leading players and what are their prospects over the forecast period? How will the industry evolve during the period between 2020 and 2030?

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Companies covered in the report include:

Affymetrix, Inc.AkouosAllCellsApplikon Biotechnology Inc.ATLATL CentreAutolus LimitedBeckman Coulter, Inc.Becton, Dickinson and CompanyBioengineering AGBiological IndustriesBioWa, IncBrammer BioC.R. Bard, Inc.CaridianBCT, IncCell and Gene Therapy Asia Technology CentreCentre for Process Innovation (CPI)CMC Biologics (Asahi Glass Co.)Cobra BiologicsCocoon PlatformCryoportDanaher CorporationDiNAQOR AGEMD Performance MaterialsEMD SeronoEppendorf AGEuropean Molecular Biology Laboratory (EMBL)Finesse Solutions, Inc.Flexsafe RM TXFloDesign SonicsFlowJo, LLCFood and Drug Administration (FDA)Gamida CellG-CON ManufacturingGE healthcareGenScriptInfors HTIntegrated DNA Technologies, Inc.LaVision BioTecLonza Group, GE HealthcareMassachusetts Eye and Ear (MEE)Meissner Filtration Products, Inc.Merck KGaAMerck SeronoMesoblastMilliporeSigmaMiltenyi BiotecNova BiomedicalPall Corporation (Pall)Patheon N.V.Penn State UniversityPharmaCell B.VSartorius AGSartorius Stedim BiotechScinogySelecta Biosciences, IncSiemensSolaris BiotechStafa Cellular TherapyStafaCTStemcell TechnologiesTerumo Bct, Inc. (A Subsidiary of Terumo Corporation)Thermo Fisher Scientific, Inc.Tillotts Pharma AGTranstem LabUniCAR TherapyWorld Courier

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Visiongain Report Looks at Opportunities Within the $23bn Cell Therapy Technologies Market - Yahoo Finance

Introduction

Sangamo Therapeutics (SGMO) is a clinical-stage biotech company focusing on the research and development of genomic medicine across 4 distinct technology platforms: gene therapy, cell therapy, in vivo genome editing, and in vivo genome regulation.

Sangamo is best-known for developing its proprietary gene-editing technology, zinc finger proteins ("ZFPs"), which is a naturally occurring class of transcription factor proteins found in humans and other species. The company has used its internal know-how and technical expertise to develop a proprietary synthetic ZFP platform with potential clinical utility in ex vivo gene-edited cell therapy, in vivo genome editing, and in vivo genome regulation.

ZFPs also can be engineered to make zinc finger nucleases ("ZFNs") which proteins that can be used to specifically modify DNA sequences by knocking in or knocking out select genes, or genome editing, and ZFP transcription-factors ("ZFP-TFs") which are proteins that can be used to selectively increase or decrease gene expression.

Sangamo is developing a series of clinical programs, which are either wholly-owned or partnered with well-established pharma and biotech companies, to focus on 3 therapeutics areas in inherited metabolic disease ("IMDs"), central nervous system ("CNS), and inflammatory and autoimmune diseases. Its full list of clinical pipeline programs is listed in Figure 1.

Figure 1 Sangamo Therapeutics' Clinical Pipeline (Source)

The company's most advanced program is an investigational gene therapy for severe hemophilia A, SB-525. SB-525 is developed under a global collaboration with Pfizer (PFE), of which the rights of SB-525 have been transferred to Pfizer to run a phase 3 trial. In December 2019, both companies announced updated initial data from the phase1/2 trial of SB-525. SB-525 was generally well-tolerated and demonstrated a sustained increase in Factor VIII activity. SB-525 has been granted RMAT, Orphan Drug, and Fast Track designation by the FDA as well as Orphan Medicinal Product Designation by the European Medicines Agency ("EMA").

Beyond SB-525, the company is also investigating 2 wholly-owned gene therapy. ST-920 is being evaluated to treat Fabry disease, a rare inherited metabolic disease, in a phase 1 study in the US and UK. SB-920 has received Orphan Drug designation by the FDA. The company also plans to advance ST-101 into clinical trials in 2021 to treat phenylketonuria ("PKU") which is a rare inherited disorder that originates from a defect in the PAH gene and results in a harmful accumulation of phenylalanine in cells throughout the body.

Sangamo is working with Sanofi (NASDAQ:SNY) to develop ex vivo gene-edited cell therapies, ST-400 and BIVV-003, for transfusion-dependent beta-thalassemia ("TDT") and sickle cell disease ("SCD") respectively. Both ST-400 and BIVV-003 are related product candidates using the same technology involving gene editing of a patient's own hematopoietic stem progenitor cells using non-viral delivery of ZFN technology.

Sangamo is the phase 1/2 study of ST-400 in 6 patients with TDT while Sanofi is recruiting the phase1/2 study evaluating BIVV-003 in patients with SCD, and Sanofi is responsible for the subsequent development, manufacturing, and commercialization of both programs.

In Dec 2009, Sangamo presented interim results for the first 3 patients ST-400. As of the data cut date, 2 more patients have been enrolled although they were not included in the interim updates. The 3 patients treated with ST-400 experienced prompt hematopoietic reconstitution, demonstrating neutrophil engraftment in 14-22 days and platelet engraftment in 22-35 days. No emerging clonal hematopoiesis had been observed as measured by on-target indel pattern monitoring in the three treated patients. The downside of the data readout is that its treatment of TDT appears to be not as efficacious as other competitors such as bluebird bio (BLUE).

Sangamo also has a global collaboration and license agreement with Kite Pharma, a wholly-owned subsidiary of Gilead Sciences (GILD), for the development of engineered cell therapies for cancer. The company is working together with Kite to design ZFNs and viral vectors to disrupt and insert select genes in T cells and natural killer cells. The first program of this agreement expected to start a clinical trial in 2020 is KITE-307, which is an allogeneic anti-CD19 CAR-T cell therapy. Given the well-documented struggles of Kite's approved autologous CAR-T, Yescarta, the success of allogeneic CAR-Ts will be very beneficial.

Lastly, Sangamo is also evaluating the potential of regulatory T-cells ("Tregs") genetically modified with a CAR ("CAR-Tregs") in solid organ transplantation. CAR-Treg cell therapies are being conducted in several preclinical studies in autoimmune and inflammatory diseases such as multiple sclerosis ("MS") and inflammatory bowel disease ("IBD"). The most advanced CAR-Treg cell therapy is TX200, which is an autologous treatment for the prevention of solid organ transplant rejection and the clinical trial is expected to be initiated in 2020.

Sangamo is only planning to start a new clinical trial for its in vivo genome editing programs. SB-913 is a second-generation ZFNs program that will be used to treat Mucopolysaccharidosis type II ("MPS II") and a new clinical trial is planned to start this year.

The company had previous programs from first-generation ZFNs that have been halted as they did not demonstrate enough clinical benefits. The company plans to use data from the SB-913 study to definite the next steps for its in vivo genome editing programs.

The company also has several preclinical programs evaluating their ZFP-TF technology as a novel therapeutic approach for CNS diseases. In December, Sangamo announced a collaboration with Biogen (BIIB) to develop and commercialize ST-501 for tauopathies including Alzheimer's disease, ST-502 for synucleinopathies including Parkinson's disease, a third undisclosed neuromuscular disease target, and up to 9 additional neurological disease targets. Under the terms of the agreement, Biogen will pay Sangamo $350M upfront, inclusive of a license fee and equity investment, and Sangamo is eligible to receive up to $2.37B in future milestones.

Sangamo also has a partnership with Pfizer and Takeda (NYSE:TAK) to develop and evaluate ZFP-TFs. The company is working with Pfizer to evaluate ALS and frontotemporal lobar degeneration ("FTLD") that are linked to the mutations in the C9ORF72 gene. In the partnership with Takeda, the company is evaluating a preclinical program for Huntington's disease in which ZFP-TF is designed to differentially down-regulate the mutated disease-causing huntingtin gene ("HTT gene") while preserving the expression of the normal version of the gene.

As of 31 December, 2019, cash and equivalents on hand was $385M. The amount is excluding the $350M injection from the collaboration with Biogen, and when factored in, cash on hand should comfortably be in the range of high $600-700M. This should give them a comfortable runway to fund all operations well into 2021, an important point given that the recent stock market crash which limits any secondary offering options.

Impressively, the company has managed to strike several high-profile partnerships with 5 global biotech/big pharma companies. Such partnerships not only validate Sangamo's technology and capabilities, but they also provide future avenues of funding with as much as $6.34B royalties on net product sales and potential milestone payments due to the company.

Figure 1 Sangamo Therapeutics' Partnerships (Source)

In terms of competition, the company competes with several players, particularly in the cell and gene therapy space. bluebird bio has more advanced programs in both TDT and SCD and, to date, has shown much better efficacy. There are also other companies such as CRISPR Therapeutics (CRSP) that are using an alternative gene-editing method, CRISPR/Cas9 in gene therapy. Other companies such as Editas Medicine (EDIT) and Intellia Therapeutics (NTLA) are also developing CRISPR/Cas9 for treatments in TDT and SCD, although it must be noted that these are not their lead programs.

In terms of allogeneic CAR-T, there are more established players such as Allogene Therapeutics (ALLO), Cellectis (CLLS), and Precision Biosciences (DTIL). The main difference among these companies is primarily the choice of gene-editing tools with Allogene and Cellectis using TALEN while Precision is using ARCUS. All these companies are, currently, in a similar stage of clinical development.

In addition to healthy donors derived allogeneic therapies, Fate Therapeutics (FATE) is developing allogeneic therapies from induced pluripotent stem cells ("iPSCs") as a renewable cell source. The advantage of this is that product consistency and potency will be improved, and the manufacturing process will be akin to the well-established biologics where they are produced from a single cell line. It is notable to note that Allogene is also investigating using iPSCs as a renewable cell source. Also, Atara Biotherapeutics (ATRA) is developing an Epstein-Barr Virus ("EBV")-based allogeneic T cell therapy platform. Their lead program is in Phase 3 and a BLA filing is expected by the second half of the year. That should put them in the lead position of commercializing an allogeneic T cell therapy and the company is gradually moving into allogeneic CAR-T space as well.

Sangamo is, currently, trading at a market cap of around $700M, which is almost as much as its cash position. While its cash position will eventually deplete to fund operations and clinical trials, the current valuation means that there is also no value for its technology and intellectual position. I consider it a good time to take up a small position in Sangamo, especially if investors have a time horizon of at least a year to weather the COVID-19 black swan event and wait for further clinical updates from the company.

It must be cautioned though that investing in clinical-stage biotech can be extremely risky, given the binary nature of the field. This is especially so, given the market turmoil from the COVID-19 pandemic. The pandemic has also led to several countries announcing lockdowns, which have disrupted supply chain and operations. Several clinical trials have already been delayed globally and this may impact Sangamo negatively, as their cash burn will continue even if clinical trials are delayed.

Disclosure: I am/we are long ATRA, BLUE. I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.

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Sangamo Therapeutics: Market Cap Is At A Bargain Relative To Its Cash Position - Seeking Alpha

Chimeric antigen receptor (CAR) T-cell therapies have produced encouraging clinical outcomes, demonstrating their therapeutic potential in mitigating tumour development. However, another form of T-cell immunotherapy based on T-cell receptors (TCR) has also shown great potential in this field. Here, Nikki Withers speaks to Miguel Forte who elaborates on the process and explains why he is excited about seeing an idea translate into an industrial proposition.

STIMULATING the natural defences of a persons immune system to kill cancer cells, known as immunotherapy, has become a novel and exciting approach to treat cancer. For example, the role of T cells in cell-mediated immunity has inspired the development of several strategies to genetically modify T cells, such as chimeric antigen receptor (CAR) T-cell therapy, to target cancer cells. In recent years, CAR T-cell therapy has received much attention from researchers and the press alike, and the landmark approval and clinical successes of Novartis Kymriah (the first FDA-approved treatment to include a gene therapy step in the United States) and Gilead/Kite Pharmas Yescarta (the first CAR T-cell therapy for adults living with certain types of non-Hodgkin lymphoma) has prompted a surge of further research. However, this approach which involves isolating cells from a patient, bioengineering them to express CARs that identify and attach to tumour cells and injecting them back into the patient has several limitations, according to Miguel Forte, former CEO of Zelluna Immunotherapy and currently CEO of Bone Therapeutics.

Forte has been working on a T-cell immunotherapy approach that primarily focuses on the T-cell receptors (TCRs). Similar to CAR therapies, TCR therapies modify the patients T lymphocytes ex vivo before being injected back into the patients body. However, they differ in their mechanisms for recognising antigens. CAR T-cell therapy can be compared to a policeman, with a photograph of the criminal, being able to identify them on the street, explained Forte. It is an artificial way of guiding those cells to the cancer when the cancer cells are in suspension. The difficulty with CAR is that it cannot always penetrate and deliver an effect in solid tumours. TCR therapy, which utilises the natural mechanisms that T cells use to recognise the antigen and therefore the cancer, is better suited to penetrate the tumour ie, the policeman is able to go inside the building where a criminal is hiding.

It is obviously more costly at the beginning of the development when you are fine tuning your process, compared to when you progress to a larger scale as you approach the market

Of note, this approach targets the TCR- peptide/major histocompatibility complex (MHC) interaction, which enables eradication of tumour cells. Intracellular tumour-related antigens can be presented as peptides in the MHC on the cell surface, which interact with the TCR on antigen-specific T cells to stimulate an anti-tumour response. Imagine you, or the cells, are not just a soldier in an army but a captain that can bring other immune cells into the mix. TCRs and these cells, once they go in, have a direct kill activity and an immunostimulatory activity to other cells to have a more comprehensive effect of killing the tumour cells. Forte concluded that this approach is scientifically appealing and could bring value to a large array of solid tumours.

The benefits of TCR therapies are evident; however, as with all new approaches, it is not without its challenges. The first relates to the manufacturing of these therapies; the process requires extracting patient material, changing it and then returning it to the patient. Unlike drug discovery with small molecules where you have an inert, well-defined, chemically-established component, with biologics you go up a notch in terms of complexity, Forte explained, adding that while small molecules are unidimensional, biologics are three-dimensional and, thus, more complex and challenging to manufacture. You need to remember that your product, the cells, are a living being. It is something that replicates, changes and responds to its environment. This makes it a lot more challenging to characterise and define the right specifications of the product. The initial challenge is to put in place a consistent and reliable manufacturing process.

Generating the necessary pre-clinical data can also prove challenging; studies are easier to conduct in animal models when you are working with chemical entities rather than human cells, according to Forte. Finally, when the product does get to clinic, there are elements of manufacturing, supply and logistics that can prove challenging; however, companies are starting to provide solutions for this. Working in cell and gene therapy we need to apply what we have done with other products, explained Forte. You need to adapt to the complexity and diversity of the product you have in hand. Here, you have a live product. Something that responds. It is similar to having a child; you can modulate it, but you can never fully control the behaviour of something you are shaping.

Bringing a new drug to market, from drug discovery through clinical trials to approval, can be a costly process, especially when developing cell-based therapies. These are more expensive than developing chemistry or biologics, but when biologics started to be developed, they were also very expensive, explained Forte. We are now seeing a reduction of those costs as more companies are developing products and consequently more solutions are surfacing.

Forte was involved in developing his first cell therapy product about 10 years ago. At this time, it was difficult; a lot of solutions you had to build in house. Nowadays, you can import this from solutions already available so you can concentrate on the specificity; for instance, the viral vector for gene editing your cells or the cytokine concentration for the expansion of your cells. He added that as these therapies grow, so too does the competition, resulting in reduced costs. However, the price and return on investment must correlate with benefit. It is obviously more costly at the beginning of the development when you are fine tuning your process, compared to when you progress to a larger scale as you approach the market.

The well-publicised success story of Emily Whitehead a six-year-old leukaemia patient who was one of the first patients to receive CAR T-cell therapy is a prime example of the success of immunotherapy treatments. Even though these patients may need to continue medications, they can live a relatively normal life. The gene- edited cells remain in the individual and continue to control the cancer by restoring the immune systems capabilities, said Forte. He hopes that similar results will be seen with TCR therapies: Hopefully, a significant fraction of patients will have a clinical and biological response that will reduce the tumour bulk, give them a quality life and remain doing so by controlling the cancer for a significant amount of time.

Forte concluded that the possibilities for TCR- based immunotherapies are exciting and hopefully products will be developed that will deliver an immediate and sustained effect in cancer patients.

About the author

MIGUEL FORTE

Miguel is currently the CEO of Bone Therapeutics and visiting Professor at the Lisbon University in Portugal. He also serves as Chief Commercialization Officer and Chair of the Commercialization Committee of the International Society of Cellular Therapy (ISCT) and is Member of Board of Directors of ISCT and ARM. Miguel was CEO of Zelluna Immunotherapy until the end of 2019. Miguel holds a masters degree from the Faculty of Medicine of the University of Lisbon, Portugal, a PhD in Immunology from the University of Birmingham, UK, an accreditation as Specialist in Infectious Diseases and a certificate on Health Economics of Pharmaceuticals and Medical Technologies (HEP). He is Fellow of the Faculty of Pharmaceutical Medicine of the RCP in the UK.

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TCR therapy an attractive alternative to CAR T for immunotherapy - Drug Target Review

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Global Hemophilia Gene Therapy Market 2020:Global Industry Size, Share, Trends, Status and 2026 Forecast Spark Therapeutics, Freeline Therapeutics,...

Collaboration to leverage synergies between Monoclonal Antibody and Non-Viral Gene Delivery Platforms

SAN DIEGO and BOSTON, March 23, 2020 (GLOBE NEWSWIRE) -- In response to the government call for rapidly deployable countermeasures, Sorrento Therapeutics, Inc. (Nasdaq: SRNE, Sorrento) and SmartPharm Therapeutics Inc. (SmartPharm) today announced a research and development collaboration to develop a next-generation, gene-encoded antibody vaccine for COVID-19. The collaboration will utilize monoclonal antibodies against SARS-CoV-2 virus discovered and/or generated by Sorrento that will be encoded into a gene for delivery utilizing SmartPharms non-viral nanoparticle platform.

Over the past 10+ years, Sorrento has extensively utilized the G-MABTM Library, one of the largest and most diverse fully human antibody libraries in the biopharma space, for discovering potent immuno-oncology and anti-infective antibodies against over 100 drug targets. In the effort to more quickly resolve the global COVID-19 crisis, our company has initiated a rapidly accelerated program for the identification of potent neutralizing antibodies against SARS-CoV-2 coronavirus antigens that may be used for either treatment or prophylaxis, said Henry Ji, CEO of Sorrento Therapeutics. We expect our platform to produce many candidate neutralizing antibodies for SmartPharm to incorporate into its powerful gene delivery platform. We look forward to our partnership with SmartPharm as part of our goal to make a meaningful impact in this truly global effort.

As a company founded by infectious disease physicians, including myself, we are passionate about applying our novel gene delivery platform to this national and global health crisis, said Jose Trevejo CEO of SmartPharm Therapeutics. Given the disproportionate mortality in elderly and immune-compromised, it is critical that we develop novel technologies that will better protect our populations that are particularly vulnerable to severe coronavirus infection.

Unlike classical antigen-based vaccines, which rely on a patients immune system to establish efficacy, SmartPharms gene-encoded antibody platform is designed to directly neutralize the coronavirus by producing the protective antibody directly in the muscle of the individual. This gene-encoded monoclonal antibody delivery platform or Gene MAb bypasses the in vitro antigen production process and potential for vaccine-induced side-effects in immunized individuals. This is especially important in susceptible populations like the elderly, where antigen-based vaccines are significantly less effective for the prevention of respiratory infections such as influenza or coronavirus. The companies expect that this novel approach will enable faster progression to clinic, pending agreement with the FDA.

As part of the collaboration, Sorrento and SmartPharm expect to develop a gene-encoded antibody or antibodies that can be administered as a prophylaxis against SARS-CoV-2 infection. Plans for the collaboration may include candidate development as well as filing of an IND application in the next few months.

About SmartPharm Therapeutics

SmartPharm Therapeutics Inc. is a privately held, pharmaceutical company focused on developing next-generation, non-viral gene therapies for the treatment of serious or rare diseases with the vision of creating Biologics from Within. SmartPharm is currently developing a novel pipeline of non-viral, gene-encoded proteins for the treatment of conditions that require biologic therapy such enzyme replacement and tissue restoration. SmartPharm commenced operations in 2018 and is headquartered in Cambridge, MA, USA. For more information, please visit http://www.smartpharmtx.com.

About Sorrento Therapeutics, Inc.

Sorrento is a clinical stage, antibody-centric, biopharmaceutical company developing new therapies to turn malignant cancers into manageable and possibly curable diseases. Sorrento's multimodal multipronged approach to fighting cancer is made possible by its extensive immuno-oncology platforms, including key assets such as fully human antibodies (G-MAB library), clinical stage immuno-cellular therapies (CAR-T, DAR-T), intracellular targeting antibodies (iTAbs), antibody-drug conjugates (ADC), and clinical stage oncolytic virus (Seprehvir).

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Sorrento's commitment to life-enhancing therapies for patients is also demonstrated by our effort to advance a first-in-class (TRPV1 agonist) non-opioid pain management small molecule, resiniferatoxin (RTX), and ZTlido (lidocaine topical system) 1.8% for the treatment of post-herpetic neuralgia. Resiniferatoxin is completing a phase IB trial for intractable pain associated with cancer and a phase 1B trial in osteoarthritis patients. ZTlido was approved by the FDA on February 28, 2018. For more information visit http://www.sorrentotherapeutics.com

Forward-Looking Statements

This press release and any statements made for and during any presentation or meeting contain forward-looking statements related toSorrento Therapeutics, Inc., under the safe harbor provisions of Section 21E of the Private Securities Litigation Reform Act of 1995 and subject to risks and uncertainties that could cause actual results to differ materially from those projected. Forward-looking statements include statements regarding the collaboration and expected scope, terms and timing thereof and plans related thereto; the expected timing for the initiation and completion of ongoing studies for coronavirus using antibodies and data read-outs related thereto; the number of antibodies expected to be identified; the expected timing for commencing and completing registrational studies, including any potential for faster progression to the clinic, and for submitting an IND application for antibody technology for the treatment and/or prevention for coronavirus; the potency of any antibodies and ability to provide efficacy; any potential market for antibody therapy for the treatment and prevention of coronavirus and Sorrentos potential position in the anti-viral immunity industry. Risks and uncertainties that could cause our actual results to differ materially and adversely from those expressed in our forward-looking statements, include, but are not limited to: risks related to Sorrento's and its subsidiaries', affiliates and partners technologies and prospects and collaborations with partners, including, but not limited to, the collaboration with SmartPharm, using gene-encoded antibodies for the treatment and prevention of coronavirus infections; risks related to seeking regulatory approvals and conducting and results of clinical trials; the clinical and commercial success of the treatment and prevention of coronavirus infections using gene-encoded antibodies; the viability and success of using gene-encoded antibodies for treatments in anti-viral therapeutic areas, including coronavirus; clinical development risks, including risks in the progress, timing, cost, and results of clinical trials and product development programs; risk of difficulties or delays in obtaining regulatory approvals; risks that clinical study results may not meet any or all endpoints of a clinical study and that any data generated from such studies may not support a regulatory submission or approval; risks related to seeking regulatory approvals and conducting clinical trials; risks of supplying drug product; risks related to leveraging the expertise of its employees, subsidiaries, affiliates and partners to assist the company in the execution of its strategies; risks related to Sorrentos debt obligations; risks related to the global impact of COVID-19 and other risks that are described in Sorrento's most recent periodic reports filed with theSecurities and Exchange Commission, including Sorrento's Annual Report on Form 10-K for the year endedDecember 31, 2019, and subsequent Quarterly Reports on Form 10-Q filed with theSecurities and Exchange Commission, including the risk factors set forth in those filings. Investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date of this release and we undertake no obligation to update any forward-looking statement in this press release except as required by law.

Media and Investor Relations

Contact: Alexis Nahama, DVM (SVP Corporate Development)

Telephone: 1.858.203.4120

Email:mediarelations@sorrentotherapeutics.com

Sorrento and the Sorrento logo are registered trademarks of Sorrento Therapeutics, Inc.COVIDTRAP, Saving-Lifeand Improving-Lifeare trademarks of Sorrento Therapeutics, Inc.ZTlido and G-MAB are trademarks owned by Scilex Pharmaceuticals Inc. and Sorrento, respectively.Seprehvir, is a registered trademark of VirttuBiologics Limited, a wholly-owned subsidiary of TNK.Therapeutics, Inc. and part of the group of companies owned by Sorrento Therapeutics, Inc.All other trademarks are the property of their respective owners. 2020 Sorrento Therapeutics, Inc. All Rights Reserved.

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SORRENTO AND SMARTPHARM TO COLLABORATE TO DEVELOP NOVEL GENE-ENCODED ANTIBODY VACCINE INTENDED TO PROTECT AGAINST COVID-19 - Yahoo Finance

DUBLIN--(BUSINESS WIRE)--The "Virus Filtration Market Size, Outlook and Growth Opportunities, 2019- 2025" report has been added to ResearchAndMarkets.com's offering.

The 'Virus Filtration market outlook to 2025' report includes the latest predictions of the global Virus Filtration market along with geography, therapy area, and applications. The report specifically focuses on different types of Virus Filtration with special attention to their emergence over the forecast period to 2025.

The research report provides objective measures to tap into future opportunities that will be available over the next six-years. It also highlights key areas to watch over the future along with detailed insights into drivers and challenges across different Virus Filtration applications and products.

Companies Mentioned

The Virus Filtration market report aggregates the current market size based on volume and average price data. It also includes a six-year outlook to 2025 based on anticipated growth rates for each sub-segment and industry as a whole.

The base case outlook in the next six-years for different types of Virus Filtration, across different verticals and countries, is provided. It additionally examines the key dynamics around companies, markets, along with key trends, drivers and challenges facing the Virus Filtration worldwide.

The report covers a broad region from the Asia Pacific, North America, Europe, Latin America, and Middle East Africa from 2018 to 2025. 12 countries across these regions are analyzed independently in the Virus Filtration report. Impact of domestic market conditions, price variations, competition, government policies, healthcare spending, technological and local market conditions are assessed for country-level forecasts.

Global spending on healthcare continues to increase significantly with the aging population, increased access to treatment and ongoing advancements in technology. This provides strong support for Virus Filtration market growth in the medium to long term future. While growth will remain steady in the developed markets, Asian and other emerging markets will be key for Virus Filtration market growth from 2019 to 2025.

To address the expanding need for advanced Virus Filtration products, companies are rapidly embracing the new market dynamics, primarily focusing on new launches, specifically to suit local and regional demand patterns. Accordingly, detailed discussion about broader implications of key strategies, product launches, and other latest Virus Filtration market developments are included in the research work.

Key Topics Covered:

1. Table of Contents

2. A Review of 2018 and Outlook to 2025

2.1. Trend Analysis

2.2. Critical Success Factors

2.3. Demand and Growth Dynamics by Type

2.4. Demand and Growth Dynamics by Application

2.5. Demand and Growth Dynamics by Market

3. Strategic Analysis

3.1. Largest Drivers of Virus Filtration Market Growth

3.2. Different Challenges Faced by Virus Filtration Companies

3.3. Five Forces Analysis

4. Global Virus Filtration Market Outlook by Product

4.1 Kits, Reagents, and Consumables

4.2 Filtration Systems

4.3 Services

5. Global Virus Filtration Market Outlook by Application

5.1 Biological

5.1.1 Vaccines and Therapeutics

5.1.2 Blood and Blood Products

5.1.3 Cellular and Gene Therapy Products

5.1.4 Tissue and Tissue Products

5.1.5 Stem Cell Products

5.2 Medical Devices

5.3 Water Purification

5.4 Air Purification

6. Global Virus Filtration Market Outlook by End User

6.1 Pharmaceutical and Biotechnology Companies

6.2 CRO's

6.3 Research Institutes

6.4 Medical Device Companies

7. Asia Pacific Virus Filtration Market Outlook

7.1 Leading Virus Filtration Types contributing to Asia Pacific market

7.2 Top Applications contributing to Asia Pacific Virus Filtration

7.3 Top countries contributing to Asia Pacific Virus Filtration

8. Europe Virus Filtration Market Outlook

8.1 Leading Virus Filtration Types contributing to Europe market

8.2 Top Applications contributing to Europe Virus Filtration

8.3 Top countries contributing to Europe Virus Filtration

9. North America Virus Filtration Market Outlook

9.1 Leading Virus Filtration Types contributing to North America market

9.2 Top Applications contributing to North America Virus Filtration

9.3 Top countries contributing to North America Virus Filtration

10 South and Central America Virus Filtration Market Outlook

10.1 Leading Virus Filtration Types contributing to South and Central America market

10.2 Top Applications contributing to South and Central America Virus Filtration

10.3 Top countries contributing to South and Central America Virus Filtration

11 Middle East Africa Virus Filtration Market Outlook

11.1 Leading Virus Filtration Types contributing to Middle East Africa market

11.2 Top Applications contributing to Middle East Africa Virus Filtration

11.3 Top countries contributing to Middle East Africa Virus Filtration

12 Company Profile Snapshots

12.1 Top Companies Operating in Virus Filtration market

13 Recent Industry Developments

14 Appendix

For more information about this report visit https://www.researchandmarkets.com/r/cjw9dh

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Outlook on the Global Virus Filtration Industry to 2025 - Largest Drivers of Virus Filtration Market Growth - ResearchAndMarkets.com - Business Wire

RESEARCH TRIANGLE PARK, N.C. and CHAPEL HILL, N.C., March 18, 2020 (GLOBE NEWSWIRE) -- Asklepios BioPharmaceutical, Inc. (AskBio), a leading clinical-stage adeno-associated virus (AAV) gene therapy company, today announced that it has entered into a research collaboration and licensing agreement with the University of North Carolina at Chapel Hill (UNC) for the development and commercialization of gene therapy for Angelman syndrome.

This collaboration allows us to leverage groundbreaking research from UNC and apply our AAV development capabilities to find a gene therapy treatment for Angelman syndrome, said Sheila Mikhail, JD, MBA, AskBio Chief Executive Officer and co-founder. We look forward to advancing this program together.

Angelman syndrome is a rare neurogenetic disorder caused by the loss of function of the UBE3A gene. The disorder occurs in approximately one in 15,000 people, or about 500,000 individuals worldwide, and there is currently no cure. In addition to life-altering symptoms such as speech and motor deficits, more than 80 percent of Angelman syndrome patients experience epilepsy, which typically does not respond well to standard anti-seizure medications.

A UNC School of Medicine team, led by Mark Zylka, PhD, and Ben Philpot, PhD, has generated preclinical evidence that gene therapy may help individuals with Angelman syndrome by improving seizure and motor outcomes.

Individuals with Angelman syndrome face lifelong challenges, and our gene therapy approaches hold the potential to correct this disorder at its genetic roots. We are incredibly excited to partner with AskBio, as they have been vanguards of clinical gene therapies for rare diseases, said Mark Zylka, PhD, Director of the UNC Neuroscience Center. Ben Philpot, PhD, Associate Director of the UNC Neuroscience Center added, We look forward to advancing this transformative treatment to the clinic and potentially improving the lives of individuals with Angelman syndrome.

The partnership between AskBio and UNC could transform the lives of people living with Angelman syndrome by providing them with a potential therapy for this rare disease, said Amanda Moore, Angelman Syndrome Foundation CEO. The Angelman Syndrome Foundation has long been proud to support the work of UNC researchers, Drs. Ben Philpot and Mark Zylka, and invest in science that positively affects the Angelman syndrome community. The collaboration between UNC and AskBio brings us a step closer to delivering a viable gene therapy to the people and families we serve.

The financial terms of the agreement were not disclosed.

More about Angelman SyndromeDeletion of the maternally inherited copy of the UBE3A gene causes Angelman syndrome. Symptoms include microcephaly (small head circumference), severe intellectual disability, seizures, balance and movement problems (ataxia), lack of speech, and sleep problems. Behavioral symptoms include frequent laughing, smiling and excitability. Angelman syndrome was first described in 1965, yet no treatment options have been approved in the 55 years since. While individuals with the disorder have a normal lifespan, they require life-long care and are not able to live independently.

About Angelman Syndrome FoundationThe mission of the Angelman Syndrome Foundation is to advance the awareness and treatment of Angelman syndrome through education and information, research and support for individuals with Angelman syndrome, their families and other concerned parties. We exist to give them a reason to smile, with the ultimate goal of finding a cure. To learn more, visit https://www.angelman.org.

About AskBioFounded in 2001, Asklepios BioPharmaceutical, Inc. (AskBio) is a privately held, clinical-stage gene therapy company dedicated to improving the lives of children and adults with genetic disorders. AskBios gene therapy platform includes an industry-leading proprietary cell line manufacturing process called Pro10 and an extensive adeno-associated virus (AAV) capsid and promoter library. Based in Research Triangle Park, North Carolina, the company has generated hundreds of proprietary third-generation AAV capsids and promoters, several of which have entered clinical testing. An early innovator in the space, the company holds more than 500 patents in areas such as AAV production and chimeric and self-complementary capsids. AskBio maintains a portfolio of clinical programs across a range of neurodegenerative and neuromuscular indications with a current clinical pipeline that includes therapeutics for Pompe disease, limb-girdle muscular dystrophy 2i/R9 and congestive heart failure, as well as out-licensed clinical indications for hemophilia (Chatham Therapeutics acquired by Takeda) and Duchenne muscular dystrophy (Bamboo Therapeutics acquired by Pfizer). Learn more at https://www.askbio.com or follow us on LinkedIn.

Media Contacts: AskBio Robin Fastenau Vice President, Communications +1 984.275.2705 rfastenau@askbio.com Angelman Syndrome Foundation Amanda Moore Chief Executive Officer +1 317.514.6918 amoore@angelman.org UNC Health | UNC School of Medicine Mark Derewicz Director, Research & News +1 984.974.1915 Mark.Derewicz@unchealth.unc.edu

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AskBio Enters Research Collaboration and Licensing Agreement with University of North Carolina (UNC) for Angelman Syndrome - Associated Press

Article In Brief

Mice with an open- and closed-traumatic brain injury were injected with immunomodulatory nanoparticles that reduced brain swelling and damage on MRI.

Investigators used a novel approach to prevent the swelling that can occur after traumatic brain injury (TBI) in a mouse model: they injected nanoparticles that trick white blood cells into going after them instead of rushing to the injured brain and causing an inflammatory and immune response.

Mice with TBI that were given three injections of the immunomodulatory nanoparticles beginning two to three hours after injury showed less brain swelling and damage on MRI as compared with mice with TBI that did not get the nanoparticles; the treated mice also performed better on functional tests.

The immunomodulatory nanoparticle treatment, if further proven in preclinical trials and human trials, would not undo damage from the initial injury to the brain. But it could help prevent the body from setting off a cascade of immune and inflammatory cells in reaction to the injury, which in turn can cause brain swelling and even more damage to brain tissue.

We certainly haven't gone and magically prevented that initial damage, said Jack Kessler, MD, professor of neurology at Northwestern University Feinberg School of Medicine and the senior author of the paper. What we can do is prevent the secondary damage, which is substantial.

Predicting which TBI patients will develop edema of the brain isn't easy, so having a preventive treatment like the nanoparticles that could be administered upfront could be life-altering, Dr. Kessler said.

He said some patients with head injuries come into the hospital walking and talking, but then their brain swells, and they die.

According to background in the study, published January 10 online in Annals of Neurology, each year more than 2.5 million people in the US have a traumatic brain TBI and more than five million Americans live with at least one sequela of TBI.

After the primary injury, there is substantial secondary injury attributable to infiltrating immune cells, cytokine release, reactive oxygen species, excitotoxicity, and other mechanisms, the study authors wrote. Despite many preclinical and clinical trials to limit such secondary damage, no successful therapies have emerged.

The nanoparticles tested in the mouse experiments are made of material used in biodegradable sutures. The paper specifically described the particles as highly negatively charged, 500 nm-diameter particles composed of the Food and Drug Administration (FDA)-approved biodegradable biopolymer carboxylated poly (lactic-co glycolic) acid.

The nanoparticles (IMPs), which seem like foreign invaders to the body's immune system, attract the attention of large white blood cells known as monocytes, which have been implicated in the secondary damage that occurs with TBI.

IMPs bind to the macrophage receptor with collagenous structure (MARCO) on monocytes and monocytes bound to IMPs no longer home to sites of inflammation but rather are sequestered in the spleen, where the cells die, the study authors wrote.

The mouse study involved two types of head injury. In some of the mice, the researchers performed a craniotomy to create a controlled cortical impact. Other mice received a closed head injury involving a direct blow to the head. Both types of injuries were meant to mimic what occurs in humans with TBI.

Injections of the nanoparticles were given two to three hours after the brain injury, and again at 24 hours and 48 hours post-injury. Control animals with similar brain injuries were given saline solution at the same time points.

Outcomes for the mice who received the nanoparticles were better by multiple measures, including MRI and a motor function test called the ladder rung walking test that is used in mouse experiments.

IMP administration resulted in remarkable preservation of both tissue and neurological function, in both models of head injury, the paper said. After acute treatment, there was a reduction in the number of immune cells infiltrating into the brain, mitigation of the inflammatory status of the infiltrating cells, improved electrophysiological visual function, improved long-term motor behavior, reduced edema formation as assessed by magnetic resonance imaging, and reduced lesion volumes on anatomic examination.

Dr. Kessler said that in the case of mice with an open head injury, the size of their brain lesion was 50 percent smaller in the treated animals compared with those that did not get the nanoparticles.

He said MRI showed significantly less brain swelling and less compression of the ventricles, both signs that secondary damage was minimized.

Dr. Kessler said that right now the only recourse for severe brain swelling is to do a craniotomy to relieve pressure in the skull.

He said one of the appeals of the nanoparticle treatment is that an emergency medical technician could do it in the field or the emergency room personnel could inject it.

But Dr. Kessler is also cautious about too many predications based on a pre-clinical study, saying he is fond of telling medical students that if I had a nickel for every mouse we cured, I'd be a rich man.

Sripadh Sharma, PhD, an MD-PhD student at Northwestern and the study's first author, said the nanoparticle therapy needs to be tested further in animal models before it could go into human testing. The researchers also want to learn more about how the nanoparticles bring about a reduced immune response in the body.

Dr. Sharma noted that while immune responses are a good thing in the face of injury or infection, sometimes nature doesn't always get it right, so too much of a good thing is a bad thing. And that can be the case with TBI.

He said it has been shown by another collaborator on the study, Stephen Miller, PhD, that when the scavenger receptors on the monocytes detect the light negative charge of the nanoparticles, the monocytes engulf and bind to the particles and apoptose in the spleen instead of going to the site of injury.

More studies need to be done to optimize what dose and what time these particles need to be given following a head injury, said Dr. Sharma.

Similar nanoparticle therapy is being tested for other medical conditions, including celiac disease and myocardial infarction, Dr. Kessler said.

Michael J. Schneck, MD, FAAN, professor of neurology (and neurosurgery) at Loyola University Chicago, said the study was well-designed and thorough, using two different head injury models and multiple outcome measures, including brain imaging, functional testing, and brain tissue analysis. Dr. Schneck said the paper made him wonder whether a similar approach using immune-modulating nanoparticles could reduce inflammatory-related damage following stroke and spinal cord injury.

Dr. Schneck said the concept of trying to dampen the immune response after TBI to prevent edema is not new, but the Northwestern researchers took the idea in a new direction. The nanoparticle therapy is particularly intriguing, he said, because it is fairly simple and involves the use of a material that is already approved by the US FDA, which could mean that it would take less time to move the therapy from the laboratory into clinical trials.

This is a very elegant study with interesting translational potential, he said. But it is a mouse model and its application to (human) TBI and other forms of central nervous system injury remains to be validated.

Jiangbing Zhou, PhD, associate professor of neurosurgery and biomedical engineering at Yale University, said that as someone who does research in the field of nanomedicine, he was surprised by the study's findings and wants to understand how this simple formulation particle could achieve this marked efficacy.

The study looks very exciting, but I want to know more about the mechanism, said Dr. Zhou, whose research focuses on developing translational nanomedicine, gene therapy, and stem cell therapy for neurological disorders including TBI.

He had these and other questions about the study: Why do the particles interact specifically with the inflammatory monocytes but not the others? How do the particles, which are made of safe biomaterials, efficiently kill the inflammatory monocytes in the spleen? What is happening and why?

Javier Crdenas, MD, director of the Barrow Concussion and Brain Injury Center at the Barrow Neurological Institute, said the study on the immune-modulating nanoparticle therapy for TBI was very promising, though he stressed that he is always cautiously optimistic when he sees a mouse study.

It is definitely a novel approach to addressing the secondary sequelae of brain injury and they might have something that minimizes that and hopefully improves outcomes, Dr. Crdenas said.

He said the study also raises some questions, including how the immune-modulating approach would fare in patients who have multiple injuries, not just to the head.

Dr. Crdenas said brain injuries often do not happen in isolation, with patients also having broken bones, lacerations, and other organ damage.

We don't know how this (nanoparticle treatment) would affect other organs, other immune responses elsewhere in the body, he said.

Dr. Crdenas said the field of TBI research has been disappointed before by studies of new therapies that looked promising in animal models and clinical testing but ultimately failed. He noted, for instance, that progesterone and hypothermia did not turn out to be good at preventing brain swelling.

We will wait and see, he said of the nanoparticles.

Drs. Sharma, Schneck, Zhou, and Crdenas had no disclosures.

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Nanoparticle Therapy Might Help Reduce Brain Swelling in... : Neurology Today - LWW Journals

This article was first published on Wednesday, March 18, 2020 in ProPublica.

By David Armstrong, Annie Waldman and Daniel Golden

On the fourth floor of the University of Florida cancer research building, the once-bustling laboratory overseen by professor Weihong Tan is in disarray. White lab coats are strewn over workbenches. Storage drums and boxes, including some marked with biohazard warnings, are scattered across the floor. A pink note stuck to a machine that makes copies of DNA samples indicates the device is broken.

No one is here on this weekday afternoon in February. On a shelf, wedged next to instruction manuals and binders of lab records, is a reminder of bygone glory: a group photo of Tan surrounded by more than two dozen smiling students and employees.

As the Florida lab sat vacant, a different scene unfolded half a world away in China, where a team of 300 scientists and researchers worked furiously to develop a fast, easy test for COVID-19. The leader of that timely project? Tan, the former Florida researcher.

The 59-year-old Tan is a stark example of the intellectual firepower fleeing the U.S. as a result of a Trump administration crackdown on university researchers with ties to China. Tan abruptly left Florida in 2019 during an investigation into his alleged failure to fully disclose Chinese academic appointments and funding. He moved to Hunan University in south-central China, where he now conducts his vital research.

Tan, a chemistry professor whose research has focused on diagnosing and treating cancer, quickly pivoted to working on a coronavirus test when the outbreak began in China. Boosted by a Chinese government grant, he teamed up with researchers at two other universities in China and a biotechnology company to create a test that produces results in 40 minutes and can be performed in a doctor's office or in non-medical settings like airport screening areas, according to a 13-page booklet detailing the test's development and benefits. It has been tried successfully on more than 200 samples from hospitals and checkpoints, according to the booklet, which Tan shared with a former Florida colleague. It's not clear how widely the test is being used in China.

Epidemiologists say that testing is vital to mitigate the spread of the virus. But the U.S. has lagged well behind China, South Korea, and Italy in the number of people tested. It's hard to know if Tan's test would have made a difference. The slow U.S. ramp-up has been blamed largely on bureaucratic barriers and a shortage of chemical agents needed for testing.

A star researcher funded by the National Institutes of Health, Tan taught for a quarter century at Florida and raised two sons in Gainesville. He was also a participant in the Thousand Talents program, China's aggressive effort to lure top scientists from U.S. universities, and had been working part time at Hunan University for even longer than he had taught at Florida. Last year, alerted by NIH, Florida began investigating his outside activities.

Tan declined to answer questions about his departure from Floria or his new test, but he provided documentation that his department chairman at Florida was "supportive" of his research in China as recently as 2015. He is one of three University of Florida researchers along with others from the University of Texas MD Anderson Cancer Center and the University of Louisville who relocated to China while under investigation for allegedly hiding Chinese funding or affiliations with universities there.

Such nondisclosure may well be pervasive. A ProPublica analysis found more than 20 previously unreported examples of Thousand Talents professors who appear not to have fully revealed their moonlighting in China to their U.S. universities or NIH.

NIH has contacted 84 institutions regarding 180 scientists whom it suspects of hiding outside activities or funding, and it has referred 27 of them for federal investigation, said Michael Lauer, the agency's deputy director for extramural research. "There's no reason why the U.S. government should be funding scientists who are engaged in unethical behavior. It doesn't matter how brilliant they are," said Lauer, who declined to discuss specific professors under scrutiny. "If they don't have integrity, we can't trust them for anything. How can we be sure that the data they're producing is accurate?"

Yet the government's investigations and prosecutions of scientists for nondisclosure a violation previously handled within universities and often regarded as minor may prove counterproductive. The exodus of Tan and his colleagues highlights a disturbing irony about the U.S. crackdown; it is unwittingly helping China achieve a long-frustrated goal of luring back top scientific talent.

Thousand Talents aimed to reverse China's brain drain to the West by offering elite Chinese scientists premier salaries and lab facilities to return home permanently. Finding relatively few takers, it let participants like Tan keep their U.S. jobs and work in China on the side.

By investigating Tan and other Chinese researchers for nondisclosure, the U.S. government is accomplishing what Thousand Talents has struggled to do. None of the professors identified in this article have been charged with stealing or inappropriately sharing intellectual property. Yet in the name of safeguarding American science, federal agencies are driving out innovators, who will then make their discoveries and insights in China instead of the U.S. The potential drawbacks hark back to an episode in the McCarthy era, when a brilliant rocket scientist at the California Institute of Technology was deported by the U.S. for supposed Communist sympathies and became the father of China's missile program.

John Brown, the FBI's assistant director of counterintelligence, told the U.S. Senate in November that participants in Thousand Talents and other Chinese talent programs "are often incentivized to transfer to China the research they conduct in the United States, as well as other proprietary information to which they can gain access, and remain a significant threat to the United States."

A spokesperson for the Chinese Embassy in Washington, D.C., disputed such characterizations. "The purpose of China's 'Thousand Talents Plan' is to promote talent flow between China and other countries and to galvanize international cooperation in scientific and technological innovation," Fang Hong said. While firmly opposing any "breach of scientific integrity or ethics we also condemn the attempt to describe the behaviors of individual researchers" as "systematic" intellectual property theft by the Chinese government. "It is extremely irresponsible and ill-intentioned to link individual behaviors to China's talent plan."

Steven Pei, a University of Houston physics professor and former chair of the advocacy group United Chinese Americans, said that both countries have gone too far. "The Chinese government overreached and the American government overreacted," Pei said. "China tried to recruit but it was unsuccessful. Now we help them do what they cannot do on their own."

Pei added that U.S. universities are failing to protect their Chinese faculty: "When the pressure comes down, they throw the researchers under the bus."

NIH has long viewed collaborations with China as a boon for biomedical research, even initiating a formal partnership with China's National Natural Science Foundation in 2010. But it became concerned in 2016 when it learned from the FBI that an Asian faculty member at MD Anderson had shared federal grant proposals he was reviewing with researchers at other institutions a violation of NIH rules.

Examining the grant applications of its federally funded researchers, NIH found many undisclosed foreign ties, particularly with research institutions in China. Some researchers were accepting dual appointments at Chinese universities and publishing results of U.S.-funded research under their foreign affiliation. Often, these foreign positions were not reported to the NIH or even the researchers' own American universities.

In August 2018, the NIH launched an investigation to ensure that its researchers weren't "double dipping" by receiving foreign funds for NIH-funded work or diverting intellectual property produced by federally backed research to other countries. The NIH found at least 75 researchers with ties to foreign talent programs who were also responsible for reviewing grant proposals. In some cases, Lauer said, Thousand Talents scientists with access as peer reviewers to confidential grant applications have downloaded them and emailed them to China. Other researchers have disclosed consulting or teaching in China but haven't acknowledged that they've signed an employment contract with a Chinese university or are heading a lab, he said. NIH gave the names of "individuals of possible concern" to the researchers' institutions but did not make them public.

To gauge the extent of the problem, ProPublica matched Thousand Talents recipients identified on Chinese-language websites with their disclosures to their universities and grant applications to NIH, which we obtained through public records requests. We found at least 14 researchers who apparently did not disclose foreign affiliations to their U.S. universities, which included the University of Wisconsin, Stony Brook University and Louisiana State University. We couldn't determine if these researchers were also on NIH's confidential list.

Of 23 Thousand Talents recipients in our survey who have sought NIH funding, none reported conflicts of interest with Chinese universities to the agency. Just three revealed these positions in the bio sections of their grant applications. Because NIH redacted foreign funding from the applications it provided to us, citing personal privacy restrictions, we couldn't tell if the researchers reported any grants from institutions in China.

It's not always easy to define or prosecute theft of intellectual property in academia, especially if the research is considered basic and doesn't require a security clearance. Unlike corporations that protect trade secrets, universities see science as an open, global enterprise and promote international collaborations. Practices such as photographing another research team's specially designed lab equipment may be considered unethical by some, but they aren't necessarily unlawful. Thus the U.S. government is trying to clamp down on suspected intellectual property theft by targeting nondisclosure.

Yet the link between hiding Thousand Talents affiliations and stealing research secrets may be tenuous. Universities bear some responsibility for the nondisclosure, because they are supposed to certify the accuracy of information supplied to NIH. Until recently, many schools were lax in enforcing disclosure rules. "It's fair to say, at some universities, they have not really been paying attention to how their faculty spend their time," Lauer said. One professor was away for 150 days a year and the university didn't notice, he said.

Non-Chinese scientists, including doctors paid by pharmaceutical , also underreported outside income. Nor did universities want to restrict partnerships with Chinese universities; in the prevailing culture of globalization, they encouraged foreign collaborations and sought to open branches in China to boost their international prestige and attract outstanding, full-tuition-paying students.

Now times have changed, and Chinese scientists at U.S. universities are trapped in the backwash. Even those who rejected overtures from China have been hounded. Xifeng Wu, an epidemiological researcher, worked at MD Anderson for nearly three decades and amassed an enormous dataset to help cancer researchers understand patient histories. She twice turned down invitations to join Thousand Talents. But she collaborated with and accepted honorary positions at research institutions in China, where she grew up and attended medical school. Although she said she earned no income from these posts, NIH identified her as a concern, and MD Anderson found that she did not always fully disclose her Chinese affiliations.

In early 2019, she left MD Anderson one of at least four researchers who were pushed out of the center in the wake of the federal investigations. She has become dean of the School of Public Health, with a well-equipped laboratory, at Zhejiang University in southeast China.

Dong Liang, Wu's husband and the chair of the pharmaceutical and environmental health sciences department at Texas Southern University, felt that MD Anderson buckled under pressure from NIH, which provided the institution with more than $145 million in federal grants in 2018.

"A few years back, they wanted the collaborations [with China]," said Liang. "And now, they take it back." The disclosure rules, said Liang, weren't clear, "and now it becomes a violation."

Professors who were in the process of being fired could have exercised their rights to a hearing before a faculty panel as well as "several rounds of peer discussions," but they instead left "on their own volition," MD Anderson spokeswoman Brette Peyton said. "As the recipient of significant NIH funding," MD Anderson had a responsibility to follow up on the agency's concerns, or risk losing federal money, she said.

Baylor College of Medicine in Houston took a less punitive approach than MD Anderson. When NIH alerted the Baylor College of Medicine that at least four researchers there all ethnically Chinese erred in their disclosures, Baylor corrected the documents and allowed them to continue working.

China began sending students to the U.S. in the late 1970s in the hope that they would return with American know-how and foster China's technological prowess. But, especially after the Tiananmen Square massacre in 1989, many of the students stayed in the U.S. after earning their degrees.

The Chinese government has been the most assertive government in the world in introducing policies targeted at triggering a reverse brain drain.

A 2012 paper by David Zweig and Huiyao Wang

Established in 2008, Thousand Talents was intended to lure prominent scientists of Chinese ethnicity under age 55 back to China for at least half the year with generous salaries and research funds and facilities, as well as perks such as housing, medical care, jobs for spouses and schools for children. Some Thousand Talents employment contracts require members to sign nondisclosure agreements related to their research and employment with Chinese institutions, according to a November 2019 report by the U.S. Senate's Permanent Subcommittee on Investigations.

"The Chinese government has been the most assertive government in the world in introducing policies targeted at triggering a reverse brain drain," David Zweig, a professor at Hong Kong University of Science and Technology, and Huiyao Wang, director general of the Center for China and Globalization in Beijing, wrote in 2012.

The program succeeded in attracting 7,000 foreign scientists and researchers as of 2017, the Senate subcommittee reported. But it had trouble enticing professors at elite U.S. universities, who were reluctant to uproot their families and leave their tenured sinecures. It created a second tier for recruits who were "essentially unwilling to return full-time," Zweig and Wang wrote. They could keep their U.S. jobs and come to China for a month or two. Complaints arose in China about "fake returnees" who "work nominally in China for six months" but "in fact, most of them are still abroad," according to a 2014 op-ed on the BBC News Chinese website.

Scandals marred the program's reputation in the U.S. In 2014, Ohio State contacted the FBI about engineering professor Rongxing Li, who had fled to China. Li, a Thousand Talents member, allegedly had access to restricted NASA information. The U.S. attorney's office did not bring charges against Li, who is teaching at Tongji University in Shanghai. Another Thousand Talents member, Kang Zhang, a professor of ophthalmology at the University of California, San Diego, resigned last year after reports that he failed to disclose being the primary shareholder of a Chinese company whose focus overlapped with his UC research. No charges were filed against Zhang, now a professor at Macau University of Science and Technology.

Struggling to attract top researchers, Thousand Talents also reached out to non-Chinese scientists, like Charles Lieber, the Harvard chemistry chairman charged in January with making false statements to the U.S. government by denying his involvement with Thousand Talents and with Wuhan University of Technology. His three-year Thousand Talents contract called for Wuhan to pay Lieber $50,000 a month plus more than $1.5 million for a research lab, according to the Department of Justice. Lieber has not yet entered a plea. His attorney, Peter Levitt, declined comment.

"In the last five years, there has been a definite deliberate move toward targeting non-ethnic Chinese," said Frank Figliuzzi, former FBI assistant director for counterintelligence. "They've been getting so many rejections from their own people who don't want to go back home and have fallen in love with their Western culture and their life. Or their wife won't go back. Or their kids won't go back.

"The other thing that we've seen, which I think is very troubling, 'Hey, you don't have to come back home full time.' In the intel community, we call that a RIP, recruitment in place."

Staying in the U.S. meant that Thousand Talents recipients had to report their Chinese positions to their American universities. Some didn't. Richard Hsung, a professor of pharmaceutical sciences at the University of Wisconsin, affirmed annually on disclosure forms that he had "no reportable outside activities." He acknowledged in an interview that, from 2010 to 2013, he was in Thousand Talents and worked part time as a visiting professor at Tianjin University, which has 25,000 students and is 70 miles southeast of Beijing.

He said that he didn't mention the Tianjin position because the disclosure forms confused him. He includes "National Thousand Talent Distinguished Visiting Professor at Tianjin University" among his honors on the faculty website. "I was not flaunting it, but I was not hiding it," he said.

His stints in China helped the University of Wisconsin, he said. "When there's an opportunity such as this one, you take it, it expands the visibility, it expands interacting with more students in training, and they come here to help us."

Also unreported was Hsung's relationship with a biotech company in Shanghai. In corporate records, Shanghai Fangnan Biological Technology Co. says that it "was founded by the national 'Thousand Talents Plan' specially invited experts," and it names Hsung as a director. Hsung said he was unaware of being listed as a board member and is asking the company to remove his name. He has consulted for the company "from time to time" but is compensated for expenses only, he said. "I have not been involved in any of their projects nor have they supported my research here," he added in an email.

When there's an opportunity such as this one, you take it, it expands the visibility, it expands interacting with more students in training, and they come here to help us.

Richard Hsung, professor of pharmaceutical sciences

University of Wisconsin spokeswoman Meredith McGlone said that Hsung should have reported his job at Tianjin on outside activities forms, as well as an "unexpected honorarium of less than $5,000" from the Shanghai biotech firm. He has since updated his disclosure form to reflect the honorarium, she said. While the university has no "uniform penalty" for nondisclosure, she said, the appropriate response in cases, like Hsung's, where there is no "evidence of intent to mislead" would be "additional training and perhaps a letter to the personnel file."

The university convened a working group last year to "consider policies and practices intended to bolster security without sacrificing the free exchange of ideas," McGlone said. It then added a question to the disclosure form: "Do you have an ongoing relationship with a foreign research institute or foreign entity?"

Each year, the University of Florida's chemistry department evaluates its 40 or so faculty members by criteria that include amounts raised for research funding and the number and impact of studies published. Weihong Tan, who joined the department in 1996, was usually ranked among the top three professors every year, said a department official who asked not to be identified.

Tan's research group developed a new way of generating molecules that bind to targeted cells, as a possible approach to identifying and treating cancers. He collaborated with researchers in other departments and became close with top deans and research officials on campus. He was popular with students. Each week, dozens of graduate and postgraduate researchers lined up in the hall outside his office, waiting to meet with him. He also won prestigious chemistry awards and developed an international reputation.

While at Florida, Tan maintained a connection to Hunan University in China, where he studied as an undergraduate. His curriculum vitae states he was an adjunct professor at the school from 1993 through at least 2019, when he left Florida. The part-time teaching job is the CV's only reference to any professional work in China.

In his annual disclosures to Florida, Tan did report positions and income in China, but not everything alleged by university investigators. In 2017, he said he was working 10 hours a week at Hunan for a salary of $30,000. In 2018, he said his hours had doubled to 20 a week, for $50,000. In 2019, he reported working a total of 20 hours a week for Hunan and the Institute of Molecular Medicine at Renji Hospital in Shanghai. His combined pay from the positions was $120,000, according to his form.

The association with Hunan began during a gap in Tan's resume between receiving a 1992 doctoral degree from the University of Michigan and starting postdoctoral work in 1994 at the U.S. Department of Energy's Ames Laboratory.

In recent years, according to colleagues, Tan's work in China intensified. He was making frequent trips there, sometimes traveling twice a month from Gainesville, one said. Tan told colleagues that his research in China complemented his Florida work, and that it was easier to conduct testing on people in China than in the U.S. His research in Florida focused on basic science testing that didn't involve patients.

Tan knew his increasing workload in China was putting a strain on his full-time position in the U.S. He told a colleague he was considering asking for a leave of absence from Florida. It's unclear if he did request a leave.

In January 2019, the NIH notified Florida that Tan might have undisclosed affiliations with foreign institutions as well as foreign research funding. The university then launched its own inquiry. It provided investigator notes regarding Tan and two other researchers allegedly involved in Chinese talent programs to a special state legislative committee reviewing foreign influence on publicly funded research. Those notes do not name the faculty members under investigation, instead referring to them by numbers such as "faculty 1." The details for faculty 1 including date of hire, area of research, department and Chinese affiliations match those of Tan.

Faculty members two and three appear to be Lin Yang, an NIH-funded professor of biomedical engineering, and Chen Ling, an up-and-coming pediatric cancer researcher.

Florida hired Yang from the University of Kentucky in 2014 as part of a "Preeminence Initiative" to boost its ranking among public universities. Yang traveled to Beijing for a Thousand Talents interview in 2016, according to the university's investigative notes. The following year, he was selected for the program at a Chinese university.

The effect of this is universities are bleeding good people.

Peg O'Connor, attorney for Lin Yang, ex-Florida professor of biomedical engineering

Yang resigned his Florida position last year after the university began looking into his alleged failure to disclose his association with China's Thousand Talents program. University investigators also allege that he hid being chief executive, founder and owner of an unidentified China-based company.

In an email, Yang said he disputes many of Florida's findings. He said he applied for a talent program but then turned it down. He said he never had any foreign grants or academic appointments in China while employed by Florida. Yang's attorney, Peg O'Connor, said the University of Florida began a push in 2010 to encourage overseas collaborations. "To be punished for doing what the university called on you to do doesn't make sense to me," she said. "The effect of this is universities are bleeding good people."

Ling, a part-time research associate professor, won multiple grants to study gene therapy techniques that target the most common pediatric liver cancer. "Early in a very promising career, Ling is already making great strides in the development of innovative therapies for cancer," the chairman of the medical school's pediatrics department said in a 2012 press release.

Ling left Florida last year. The university investigative notes that appear to refer to Lin allege that he failed to inform NIH that he was participating in a Chinese government sponsored talent program, and that he received an unreported research grant from a Chinese foundation.

However, Ling did report working at Fudan University in Shanghai to University of Florida officials in 2018. His disclosure, which can be viewed at ProPublica's Dollars for Profs site, shows that Fudan paid him $53,732 for activities that included "establishing a regular molecular biological laboratory, conducting gene therapy research, teaching curriculum, publishing manuscripts." He indicated that the activity would require eight months of work each year. It's unclear if Florida officials relayed this information to NIH.

Ling, who did not respond to emails seeking comment, is continuing his research as a professor at Fudan. A former Florida colleague described him as "very smart" but somewhat naive in dealing with conflict of interest issues. "I don't think he did anything with malicious intent," said the colleague. "He paid a heavy price for this."

Mengsheng Qiu, a neurobiologist at the University of Louisville, not only disclosed a part-time position in China to his department, but he even accepted a pay cut at Louisville to offset his foreign income. Nevertheless, NIH targeted him.

Qiu, 56, received his bachelor's and master's degrees in China and his doctorate from the University of Iowa in 1992. After spending five years as a postdoctoral fellow at the University of California, he joined the Louisville faculty in 1997 and was tenured in 2001.

Qiu has received several NIH grants over the past two decades. Fred Roisen, the department's former chair, described Qiu as an excellent and dedicated researcher. "He had a very active lab that published extensively," Roisen said. "His students were highly sought after for postdocs at all the best schools in the U.S. Some were Chinese and some were not. I could only give him the highest recommendation. His lab, if I went in Saturday and Sunday, there were always people working there."

Qiu joined the Thousand Talents program in 2009, taking a part-time job at Hangzhou Normal University, which announced that it had hired him as its first scholar "under a high-level creative talents program" that aimed to "attract elites from all walks of life at home and abroad." Someone at Hangzhou sent the announcement to Louisville administrators, who did not know that Qiu was seeking a position in China and were taken aback, according to a friend of Qiu's. The friend said that Qiu had not informed Louisville because terms of the Hangzhou job were still being negotiated.

I just knew he had access to a lab in China. I never had a negative thought connected with him whatsoever.

Fred Roisen, former department chair, University of Louisville

Louisville and Qiu then agreed to reduce his salary to compensate for his time in China. "We negotiated a pay cut that was proportional to the time he was away," Roisen said. "If he was taking two months off, that was two months' pay you don't get."

Roisen said he didn't know about Qiu's participation in the Thousand Talents program. "I just knew he had access to a lab in China. I never had a negative thought connected with him whatsoever." By working in China, Qiu "was trying to get additional help for projects," Roisen said. "Some tissues were not readily available in the U.S." The current chair, William Guido, declined comment.

His wife, Ling Qiu, said that each time Qiu visited China, he received the university's approval. "They wanted him to report everything," she said. "He said, 'I did.' Every time I go to China, I tell you."

"He is a good citizen," she said. "He does not even use a coupon if it is expired." Thousand Talents paid for his travel to China and his work there, she said, but it wasn't big money. "I wish," she said. She added in an email, "He did not do anything wrong but I don't know the details about his research activity."

In recent years, a Louisville colleague said, Qiu had a "lag" in federal funding, and NIH turned down one of his grant applications. "I suspect, and we all felt, that this might have been due to him putting a lot of emphasis on his Chinese involvement in those laboratories and less here," the colleague said.

The colleague added that at Qiu's most recent career review, "We did think it was interesting he managed to publish 17 papers in the previous five years, some in prestigious journals, with such a small laboratory. I think some of it was done in China. That might be why they're looking into it."

People close to Qiu said that the probe has been going on at least since the summer of 2019, and that he met for half a day on campus with investigators. University of Louisville spokesman John Karman III declined comment, citing "an ongoing investigation involving this former faculty member." Also citing the investigation, Louisville declined to provide any outside activity forms that Qiu submitted to the university.

Last December, Qiu retired from Louisville. He's now head of the Life Science Research Institute at Hangzhou Normal University. Friends said that he preferred working in China because he helped set up the institute there and the lab conditions and students were better than at Louisville. He felt unjustly overshadowed at Louisville by more prominent Ivy Leaguers in his field. When Hangzhou Normal renewed Qiu's 10-year appointment in 2019, a friend said, it asked him to become full-time, forcing him to choose between China and Louisville. "He must have balanced the two," the friend said. "Finally, he came up with a choice."

Qiu's wife, a doctor who was in private practice in the U.S., said that the investigation drove him out. It made him "very, very depressed," she said. He told her that it reminded him of China's persecution of intellectuals during the Cultural Revolution. "They sent me an email and asked me a bunch of questions," he told her. "Maybe I go to jail."

Qiu declined comment through his wife. She said in a February email that he was quarantined at home in Hangzhou because of the coronavirus, while she was fighting the epidemic at an international hospital in Shanghai. "We try to comfort each other by phone or video chat," she said. "We are not able to see each other since my job is high risk."

In 2015, when Weihong Tan was up for election to the Chinese National Academy of Sciences, his chemistry chairman at the University of Florida recommended him and lauded his ongoing research in China.

"We are very happy to see his great success at Hunan University in research and education," William Dolbier wrote in the letter provided by Tan. "We are very supportive of his research and educational activities there."

Tan's positions were also publicly listed on the web before NIH notified the University of Florida that there might be an issue.

The English language website of Hunan University, beginning in at least March 2018, listed Tan as a vice president and director of a chemistry lab. According to the site, Tan had run the lab since 2010 and had been a vice president of the school since August 2017. The school also indicated Tan was a full professor there and supervised doctoral students. Tan appeared in an English-language video in 2017 to promote a textbook he edited and described himself as a distinguished professor of chemistry at both Florida and Hunan.

On several occasions, Hunan University publicly lauded Tan. In 2017, when he was named an associate editor of the Journal of American Chemical Society, both the University of Florida and Hunan University put out press releases announcing the appointment. Florida officials at the time were apparently unaware of Tan's positions in China, and the school's release makes no mention of them. Hunan, on the other hand, lists his position in Florida.

Tan was also named an "honored professor" in 2017 at the East China University of Science and Technology. A story about a ceremony marking the appointment on that university's website includes photographs of Tan touring school labs and meeting with faculty. It lists him as holding several academic posts in China as well as his University of Florida professorship.

After NIH notified Florida at the beginning of 2019 about a potential problem with Tan, the university's office of research began reviewing Tan's emails. In correspondence, Tan acknowledged his Hunan jobs, according to the notes. He also allegedly used his Florida email account to conduct Hunan business.

The investigators found evidence that Tan had significant ties to Chinese government-sponsored talent programs and helped recruit U.S. researchers to those programs. The emails also indicated Tan received at least four research grants from Chinese government programs and didn't tell the NIH about them. Of all of Tan's extensive university and government ties with China, the only item he appears to have disclosed to the NIH and Florida was an adjunct teaching position at Hunan.

When Tan suddenly resigned his position in Florida last year, he told colleagues he was going to work full time in China but was vague about the reasons for leaving after almost a quarter century on campus. Administrators scrambled to find new mentors for the more than dozen graduate and postgraduate students working in his two labs on campus. The move was so abrupt that Tan's wife stayed behind in Gainesville, according to colleagues.

Tan didn't answer questions sent to him by email, although he did acknowledge receiving them. A federal investigation of Tan's relationships in China is ongoing, according to the investigative summary provided by the university to state legislators.

The University of Florida said in a statement that it has taken steps to prevent other professors from joining Thousand Talents and concealing foreign positions. As a result of a new risk assessment process for detecting foreign influence that it introduced in 2018, it said, Florida is denying most requests from faculty to participate in foreign talent programs.

The university said it "maintains a robust and vigilant program to safeguard our technology and intellectual property from undue foreign influence, and to extend appropriate oversight to UF activities (and those of its faculty members) in connection with foreign organizations." A spokesman declined to answer questions about individual professors, citing ongoing investigations.

William Dolbier, the former chemistry chairman at Florida and now an emeritus professor, said Tan's departure could have been avoided if he had disclosed all of his work in China. "He was not a money guy," Dolbier said. "He was not out to steal from the United States. The development of these drugs was his primary focus and goal." Dolbier added that Tan told him he would be glad to try to make his COVID-19 test available in the U.S.

Jeff Kao and Doris Burke contributed reporting.

ProPublica is an independent, non-profit newsroom that produces investigative journalism in the public interest.

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The Trump Administration Drove Him Back to China, Where He Invented a Fast Coronavirus Test - HealthLeaders Media

Gene therapy canoffer best results in Danon disease treatment, according to new research. Inthis rare health condition, human body is not able to perform the basicbiological work of removal and reuse of proteins. Thus, this situation badly affectsindividuals various body parts. It includes liver, skeletal muscles, eyes, neurologicsystem, and heart. In addition, many patients die or have to go through hearttransplants in their 30s.

Finding Therapies That Help in Addressing Root Cause of Disease

Scientists fromthe University Of California San Diego School Of Medicine revealed the outcomesof their latest research. They stated that they have discovered an advancedtechnique for the treatment of Danondisease.Basically, thistechnique uses gene therapy. The latest study is open for access inthejournal Science Translational Medicine.

Eric Adler isthe key investigator of the research. He stated, Heart transplant cannotbe useful while treating the other organs affected in Danon disease. Further,it is not always available for all patients. This specifies the need to discover new therapiesparticularly aimed at addressing the root cause of this disease. In the latestresearch, we utilized mice that were used as a model for Danon disease. Thesemice were missing this particular LAMP gene. We applied gene therapy to this micegroup. Further, the results were compared to mice that did not get treatment.

In the outcomesof this research, mice receiving gene therapy offered positive results. Thefunctioning of liver, heart, and muscle in these mice was improved. Inaddition, there was improvement in the hearts overall function of ejectingblood and relaxing. There was improvement in the bodys ability of proteins degradationand metabolism.

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Tapping Gene Therapy Technique in Danon Disease Treatment - TMR Research Blog

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Global PharmaSphere: Emerging Biotechnologies-Gene Therapy Market 2025 Forecasts And Analysis with Top Key Players like - Advantagene, Amarna...

NEW YORK, March 19, 2020 /PRNewswire/ --Hoth Therapeutics, Inc. (NASDAQ:HOTH) (the "Company' or "Hoth"), a biopharmaceutical company focused on unique targeted therapeutics for patients suffering from dermatological indications ranging from atopic dermatitis, psoriasis and acne as well as gene therapy treatment for asthmatics, today announced results from a preclinical study demonstrating the potential effectiveness of WEG232, a topical treatment with specific substance P-receptor inhibitor for Erlotinib-induced facial dermatitis and hair loss in cancer patients.

Erlotinib, an EGFR1-tyrosine kinase inhibitor, is an effective anti-tumorigenesis agent, which combats several cancers including lung, colon, head and neck. Typically, significant and often severe cutaneous toxicities are serious side effects of Erlotinib, limiting its full potential use to prolong patient survival. Previous studies suggest that neurogenic inflammation plays a serious role in causing EGFR-TKI induced off-target toxicity. This trial was designed to assess if topical application of WEG232, a specific Substance P-receptor inhibitor, would be protective against erlotinib-induced facial rash and/or hair loss.

The research, which took place at the George Washington University and supported by Hoth, suggests the topical application of WEG232 could be very effective in suppressing erlotinib induced-facial rash/hair loss with approximate 71% reduction. It concluded that WEG232 may be used as an effective intervention to prevent EGFR-TKI-induced cutaneous toxicity.

Mr.Robb Knie, Chief Executive Officer of Hoth, added, "This 12-week study with 25 SD-rats showed overwhelming compelling results of a 71% facial rash and hair loss reduction. Our next steps will be to further our preclinical work while also preparing for a pre-Investigational New Drug (Pre-IND) meeting request with the U.S. FDA. We look forward to publishing complete results of the aforementioned study in poster form prior to our pre-IND."

About Hoth Therapeutics, Inc.Hoth Therapeutics, Inc. isa clinical-stage biopharmaceutical company focused on developing new generation therapies for dermatological disorders. Hoth's pipeline has the potential to improve the quality of life for patients suffering from indications including atopic dermatitis, chronic wounds, psoriasis, asthma and acne. To learn more, please visitwww.hoththerapeutics.com.

Forward Looking StatementsThis press release includes "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements in this press release include, but are not limited to, statements that relate to the advancement and development of the BioLexa Platform, the commencement of clinical trials, the availability of data from clinical trials and other information that is not historical information. When used herein, words such as "anticipate", "being", "will", "plan", "may", "continue", and similar expressions are intended to identify forward-looking statements. In addition, any statements or information that refer to expectations, beliefs, plans, projections, objectives, performance or other characterizations of future events or circumstances, including any underlying assumptions, are forward-looking. All forward-looking statements are based upon Hoth's current expectations and various assumptions. Hoth believes there is a reasonable basis for its expectations and beliefs, but they are inherently uncertain. Hoth may not realize its expectations, and its beliefs may not prove correct. Actual results could differ materially from those described or implied by such forward-looking statements as a result of various important factors, including, without limitation, market conditions and the factors described under the caption "Risk Factors" in Hoth's Form 10K for the period endingDecember 31, 2019, and Hoth's other filings made with the Securities and Exchange Commission. Consequently, forward-looking statements should be regarded solely as Hoth's current plans, estimates and beliefs. Investors should not place undue reliance on forward-looking statements. Hoth cannot guarantee future results, events, levels of activity, performance or achievements. Hoth does not undertake and specifically declines any obligation to update, republish, or revise any forward-looking statements to reflect new information, future events or circumstances or to reflect the occurrences of unanticipated events, except as may be required by law.

ContactsInvestor Relations Contact:Phone: (646) 756-2997Email:investorrelations@hoththerapeutics.comwww.hoththerapeutics.com

KCSA Strategic CommunicationsValter Pinto, Managing Director(212) 896-1254Hoth@kcsa.com

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Hoth Therapeutics Announces Positive Preclinical Data of WEG232 Treatment, Developed for Cancer Patients Suffering from Erlotinib-Induced Facial...

The latest research analysis titledGlobalGene TherapyMarketgives a detailed assessment of the market where each factor, components, segments, and other sections of the market are comprehensively described. The report forecasts theGene Therapymarket to portray prominent growth during the forthcoming years from 2019 to2025. The report delivers geological study into several regions with market growth, production, consumption, and revenue. The research report focuses on critical data that makes it a very important tool for research, analysts, experts, and managers. It examines data and estimates on the market structure, dynamics, and trends.

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The report offers you an in-depth insight into the globalGene Therapyindustry along with estimates of market size, in value terms, estimated at USD million/billion for the period. A comprehensive and systematic framework of the market is displayed. The potential of the market has been assessed. The report looks at the growth strategies employed by key players as well as how these strategies are poised to change the competitive dynamics in the market over the projected period. The company profiles covered along with their market size, key product launches, revenue, products, key segments, mergers, acquisitions, recent developments, R&D initiatives, new product launches, and SWOT analysis,

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The globalGene Therapymarket is analyzed across key geographies namely:North America, Europe, Asia Pacific, South America, and the Middle East and Africa.

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Company Profiles:In the competitive landscape, the trends and outlook of the report are given which highlights a clear insight about the market share analysis of major industry players includingSpark Therapeutics LLC, Bluebird Bio, UniQure N.V., Juno Therapeutics, GlaxoSmithKline, Chiesi Farmaceutici S.p.A., Bristol Myers Squibb, Celgene Corporation, Human Stem Cell Institute, Voyager Therapeutics, Shire Plc, Sangamo Biosciences, Dimension Therapeutics and others.These players are identified through secondary research, their market shares have been determined through primary and secondary research. However, all percentage shares breakdowns have been demonstrated through secondary sources and verified primary sources.

Production Market: Production, volume utilization rate, revenue, capacity, cost, gross, price, gross margin analysis, market share, major manufacturers performance and regional market performance, regional production market analysis.

Market Forecast: The report provides revenue forecast and then continues with sales, sales growth rate, and revenue growth rate forecasts of the globalGene Therapymarket. Additionally, the forecasts are given with respect to consideration product, application, and regional segments of the global market till2025.

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Global Gene Therapy Market 2019 Outlook, Comprehensive Insights, Growth and Forecast 2025 - Packaging News 24

CAMBRIDGE, Mass., March 18, 2020 (GLOBE NEWSWIRE) -- Anchiano Therapeutics Ltd. (Nasdaq: ANCN) (Anchiano), a biopharmaceutical company focused on discovery and development of novel therapies to treat cancer, today reported financial results forthe year ended December 31, 2019.

Key Developments

Year Ended December 31, 2019 Financial Results:

On December 31, 2019, Anchiano had total cash and cash equivalents of approximately $17.6 million. Financial resources are expected to suffice through the end of 2020.

Research and development expenses for the year ended December 31, 2019 were approximately $13.3 million, compared to approximately $7.5 million for the same period in 2018. This increase was mainly due to an increase in clinical trial expenses, manufacturing expenses and manpower expenses, as well as additional startup and initial ongoing expenses in connection with the Collaboration Agreement with ADT.

General and administrative expenses for the year ended December 31, 2019 were approximately $6.2 million, compared to expenses of approximately $5.5 million for the same period in 2018. The increase was mainly due to increases in professional fees, insurance and manpower expenses, offset by a decrease in share-based payment.

Financing expenses, net, in the year ended December 31, 2019 were approximately $4.2 million, compared to approximately $457 thousand for the same period in 2018. This change was mainly due to a revaluation of investor warrants at fair value during a period where these could not be classified within shareholders equity.

Restructuring expenses in the year ended December 31, 2019 were approximately $3.4 million, and were comprised principally of contract termination costs and employee severance and associated termination costs related to the reduction of workforce resulting from Anchianos decision to discontinue its Phase 2 Codex as described above.

Net loss for the year ended December 31, 2019 was approximately $27.1 million compared to approximately $13.8 million for the same period in 2018.

About Anchiano

Anchiano is a biopharmaceutical company dedicated to the discovery, development, and commercialization of novel targeted therapies to treat cancer in areas of significant clinical need, with its headquarters in Cambridge, MA. Anchiano is developing small-molecule pan-RAS inhibitors and inhibitors of PDE10 and the -catenin pathway. For more information on Anchiano, please visit http://www.anchiano.com.

Forward-Looking Statements

This press release contains forward-looking statements that are subject to risks and uncertainties. Words such as believes, intends, expects, projects, anticipates and future or similar expressions are intended to identify forward-looking statements. These forward-looking statements are subject to the inherent uncertainties in predicting future results and conditions, many of which are beyond the control of Anchiano, including, without limitation, the risk factors and other matters set forth in its filings with the Securities and Exchange Commission, including its Annual Report on Form 10-K for the year ended December 31, 2019. Anchiano undertakes no obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as may be required by law.

Company Contact:Frank Haluska, M.D., Ph.D.President and Chief Executive Officerinfo@anchiano.com

Investor Contact:Ashley R. RobinsonManaging DirectorLifeSci Advisors, LLC617-430-7577arr@lifesciadvisors.com

RESULTS OF OPERATIONS (unaudited)

U.S. dollars in thousands

STATEMENTS OF FINANCIAL POSITION (unaudited)

U.S. dollars in thousands

CASH FLOWS (unaudited)

U.S. dollars in thousands

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Anchiano Therapeutics Reports Year-End 2019 Financial Results - GlobeNewswire

Orian research published a detailed study of Gene Therapy Market share, size, growth, trends, regional scope, technology innovation, key players and 2020-2024 forecast analysis. This report also presents the Gene Therapy industry scope and valuable guidance of new companies want to grow business.

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Development policies, investment plans, cost structures, capacity are discussed as well as growth rate, manufacturing processes, economic growth are analyzed. This research report also states import/export data, industry supply and consumption figures as well as cost structure, price, industry revenue (Million USD) and gross margin by regions like (North America, Europe and Asia-Pacific) and the main countries (United States, Germany, united Kingdom, Japan, South Korea and China etc.)

The major players profiled in this report include:

Gene Therapy Industry research report is a meticulous investigation of the current scenario of the Gene Therapy global and regional market, which covers several industry dynamics. The Gene Therapy market research report is a resource, which provides current as well as upcoming technical and financial details with market risk, growing demand and raw materials. The thorough analysis in this report enables investors, CEOs, regional traders, suppliers, top vendors to understand the market in a better way and based on that knowledge make well-informed decisions.

Inquire more or share questions if any before the purchase on this report https://www.orianresearch.com/enquiry-before-buying/1529811

The end users/applications and product categories analysis:On the basis of product, this report displays the sales volume, revenue (Million USD), product price, market share and growth rate of each type, primarily split into-General Type

On the basis on the end users/applications, this report focuses on the status and outlook for major applications/end users, sales volume, market share and growth rate of Gene Therapy for each application, including-Medical

The research study is a highly acclaimed resource that investors, market contestants, and other people interested in this Gene Therapy report can use to intensely position themselves in the global Gene Therapy market. It mentions the recent developments structures, future growth plans, and other significant aspects of the business key participants that define their growth in the global Gene Therapy market.

The report includes six parts, dealing with:

1) Basic Information;

2) Asia Gene Therapy Market;

3) North American Gene Therapy Market;

4) European Gene Therapy Market;

5) Market Entry and Investment Feasibility;

6) Report Conclusion.

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The report firstly introduced the Gene Therapy basics: definitions, classifications, applications and market overview; product specifications; manufacturing processes; cost structures, raw materials and so on. Then it analyzed the worlds main region market conditions, including the product price, profit, capacity, production, supply, demand and market growth rate and forecast etc. In the end, the report introduced new project SWOT analysis, investment feasibility analysis, and investment return analysis.

Gene Therapy Market Report Covered Major 20 Chapters in Table of Contents:

Part I Gene Therapy Industry Overview

Part II Asia Gene Therapy Industry (The Report Company Including the Below Listed But Not All)

Part III North American Gene Therapy Industry (The Report Company Including the Below Listed But Not All)

Part IV Europe Gene Therapy Industry Analysis (The Report Company Including the Below Listed But Not All)

Part V Gene Therapy Marketing Channels and Investment Feasibility

Part VI Global Gene Therapy Industry Conclusions

Note: If you have any special requirements related to Gene Therapy Market Report please let us know and we will offer you the report as you want.

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Gene Therapy Market 2020: Demand Analysis, Key Players, Trends, Sales, Supply, Deployment Model, Segments, Organization Size, Production, Revenue Data...

Yousuf Khan is PhD student in molecular and cellular physiology at Stanford School of Medicine. He is the first author on a recent article published in BMC Genetics which outlines the discovery of a novel overlapping coding sequence in the gene POLG. His work, combined with other research in the field, may suggest that there is an abundance of overlapping open reading frames.Technology Networks recently spoke with Khan to learn more about the research study, how genetics can be likened to cooking up a recipe, and an accidental discovery that could lead to an exciting collaboration in this field.

Molly Campbell (MC): For our readers that may be unfamiliar with genomics, open reading frames and bioinformatics, can you tell us about the background of your research?Yousuf Khan (YK): Lets compare the expression of a genetic sequence with cooking a meal. TheDNAis our motherscookbook, it contains every single possible recipe that we would ever need. But when we want to prepare a meal, we dont want to get our precious cookbook dirty. So instead, we store the cookbook in a different location (genomic DNA in eukaryotes is inside the nucleus). When we want to make something, we make aphotocopyof a page in the cookbook (the cell creates amessenger RNA, a temporary copy, of a portion of DNA).

This process of copying a portion of theDNAintomRNAis called transcription. We then take oursingle sheet of our photocopied recipe(ourmRNA) and take it back into the kitchen (thecytoplasmof the cell).

In thekitchen(cytoplasm), we read thephotocopied recipeone step at a time. By reading thephotocopied recipefrom thefirst step to the last step(the open reading frame), we convert the instructions into our finished,delicious meal. In a similar manner,mRNAis read and translated into aproteinby a machine called a ribosome.In the traditional way we understand biology, cells that want to create different proteinsjust alternatively splice differentmRNAsto be translated. This would be the equivalent of photocopyingrecipesfor scrambled eggs, pancakes, and bacon on Monday morning and then photocopying a different set ofrecipes(e.g. mashed potatoes, steak, and salad) for dinner.

MC: Can you expand on your recently published study? Have you essentially discovery a "gene within a gene"? If so, does this point towards a potentially "hidden" genome?YK: In our article, we found that the gene POLG creates an mRNA that contains a very long overlapping open reading frame. Imagine youre following the instructions of a recipe to make lasagna; you start with step one and you complete every step until you reach the last step. But if you started at step two and then completed steps three, four, five, and six, you would create a hamburger instead. So encoded within a single photocopied recipe, there are multiple meals that can be made.

This study and others that have been published previously may suggest that there are an abundance of these overlapping open reading frames. The real effort is finding them and characterizing them!

MC: What were the key challenges you encountered in this research?YK: The real challenge is finding these overlapping sequences. Theyre tricky to detect and it would not have been possible to do this work without the support of the amazing researchers at Ensembl.

MC: What are your next steps in this space?YK: I think there are more of these "hidden genes" to be found. The two important questions are i) where exactly are they? and ii) how are they read?

MC: On Twitter, you said "This finding was also made by another group at the exact same time, whose manuscript will be up shortly as well" Will your research group be looking to collaborate?YK: This is actually a funny story. I was at a conference in Germany last September and I was sitting in the audience listening to a talk. As my focus began to wane, I started leafing through the abstract book seeing what other research was going to be presented at the conference. Right in front of me on a completely random page I turned to was almost exactly my research. The only difference was that it was done by a completely different group. I panicked. However, after an email of advice from adviser, I decided to approach the group and we ended up agreeing on trying to coordinate our submissions. My work was accepted to a journal faster and hence they uploaded their manuscript to a preprint server after I told them my paper was up. The link for their work is here.

Yousuf Khan, PhD researcher at Stanford School of Medicine, was speaking to Molly Campbell, Science Writer, Technology Networks.

Reference: Khan et al. (2020). Evidence for a novel overlapping coding sequence in POLG initiated at a CUG start codon. BMC Genetics. DOI: https://doi.org/10.1186/s12863-020-0828-7.

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Novel Discovery of "Hidden" Gene Within a Gene in Mammals - Technology Networks

The global stem cell and regenerative medicines market should grow from $21.8 billion in 2019 to reach $55.0 billion by 2024 at a compound annual growth rate (CAGR) of 20.4% for the period of 2019-2024.

Report Scope:

The scope of this report is broad and covers various type of product available in the stem cell and regenerative medicines market and potential application sectors across various industries. The current report offers a detailed analysis of the stem cell and regenerative medicines market.

The report highlights the current and future market potential of stem cell and regenerative medicines and provides a detailed analysis of the competitive environment, recent development, merger and acquisition, drivers, restraints, and technology background in the market. The report also covers market projections through 2024.

Request for Report Sample:https://www.trendsmarketresearch.com/report/sample/11723

The report details market shares of stem cell and regenerative medicines based on products, application, and geography. Based on product the market is segmented into therapeutic products, cell banking, tools and reagents. The therapeutics products segments include cell therapy, tissue engineering and gene therapy. By application, the market is segmented into oncology, cardiovascular disorders, dermatology, orthopedic applications, central nervous system disorders, diabetes, others

The market is segmented by geography into the following regions: North America, Europe, Asia-Pacific, South America, and the Middle East and Africa. The report presents detailed analyses of major countries such as the U.S., Canada, Mexico, Germany, the U.K. France, Japan, China and India. For market estimates, data is provided for 2018 as the base year, with forecasts for 2019 through 2024. Estimated values are based on product manufacturers total revenues. Projected and forecasted revenue values are in constant U.S. dollars, unadjusted for inflation.

Report Includes:

28 data tables An overview of global markets for stem cell and regenerative medicines Analyses of global market trends, with data from 2018, estimates for 2019, and projections of compound annual growth rates (CAGRs) through 2024 Details of historic background and description of embryonic and adult stem cells Information on stem cell banking and stem cell research A look at the growing research & development activities in regenerative medicine Coverage of ethical issues in stem cell research & regulatory constraints on biopharmaceuticals Comprehensive company profiles of key players in the market, including Aldagen Inc., Caladrius Biosciences Inc., Daiichi Sankyo Co. Ltd., Gamida Cell Ltd. and Novartis AG

Summary

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The global market for stem cell and regenerative medicines was valued at REDACTED billion in 2018. The market is expected to grow at a compound annual growth rate (CAGR) of REDACTED to reach approximately REDACTED billion by 2024. Growth of the global market is attributed to the factors such as growingprevalence of cancer, technological advancement in product, growing adoption of novel therapeuticssuch as cell therapy, gene therapy in treatment of chronic diseases and increasing investment fromprivate players in cell-based therapies.

In the global market, North America held the highest market share in 2018. The Asia-Pacific region is anticipated to grow at the highest CAGR during the forecast period. The growing government funding for regenerative medicines in research institutes along with the growing number of clinical trials based on cell-based therapy and investment in R&D activities is expected to supplement the growth of the stem cell and regenerative market in Asia-Pacific region during the forecast period.

Reasons for Doing This Study

Global stem cell and regenerative medicines market comprises of various products for novel therapeutics that are adopted across various applications. New advancement and product launches have influenced the stem cell and regenerative medicines market and it is expected to grow in the near future. The biopharmaceutical companies are investing significantly in cell-based therapeutics. The government organizations are funding research and development activities related to stem cell research. These factors are impacting the stem cell and regenerative medicines market positively and augmenting the demand of stem cell and regenerative therapy among different application segments. The market is impacted through adoption of stem cell therapy. The key players in the market are investing in development of innovative products. The stem cell therapy market is likely to grow during the forecast period owing to growing investment from private companies, increasing in regulatory approval of stem cell-based therapeutics for treatment of chronic diseases and growth in commercial applications of regenerative medicine.

Products based on stem cells do not yet form an established market, but unlike some other potential applications of bioscience, stem cell technology has already produced many significant products in important therapeutic areas. The potential scope of the stem cell market is now becoming clear, and it is appropriate to review the technology, see its current practical applications, evaluate the participating companies and look to its future.

The report provides the reader with a background on stem cell and regenerative therapy, analyzes the current factors influencing the market, provides decision-makers the tools that inform decisions about expansion and penetration in this market.

Request for Report Discount:https://www.trendsmarketresearch.com/report/discount/11723

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Stem Cell And Regenerative Therapy Market Research Insights Global Industry Outlook Shared in Detailed Report, Forecast size 2024 - Daily Science

Abeona Therapeutics Inc.ABEO announced that it has treated the first patient in the pivotal phase III study VIITAL evaluating its lead pipeline candidate, EB-101, in patients with recessive dystrophic epidermolysis bullosa (RDEB). The rare connective tissue disorder, RDEB, is characterized by severe skin wounds and can lead to systemic complications.

The study is being conducted by investigators at Stanford University Medical Center and enrollment in it is ongoing.

Currently, there are no FDA-approved therapies for treating RDEB. A successful development of the gene-corrected cell therapy candidate, EB-101 will be a major boost for the clinical-stage pharma company.

Please note that the company has successfully completed a phase I/II study on EB-101 in RDEB patients. Data from the study showed that treatment with the candidate resulted in sustained and durable wound healing. It also had a favorable safety profile.

Shares of Abeona have lost 37.7% so far this year compared with the industrys decline of 12.7%.

Apart from EB-101, the company has two other clinical-stage pipeline candidates in its portfolio. The candidates ABO-102 and ABO-101 are adeno-associated virus (AAV)-based gene therapies, which are being developed for treating Sanfilippo syndrome type A and Sanfilippo syndrome type B, respectively.

The company is also planning to initiate a phase I/II study to evaluate pre-clinical AAV-based gene therapy candidate, ABO-202 in patients with CLN1 disease soon. An investigational new drug application to support the initiation of the study was approved by the FDA in May 2019.

Abeona Therapeutics Inc. Price

Abeona Therapeutics Inc. Price

Abeona Therapeutics Inc. price | Abeona Therapeutics Inc. Quote

Zacks Rank & Stocks to Consider

Abeona currently has Zacks Rank #3 (Hold) stock.

Some better-ranked stocks from the biotech sector include Regeneron Pharmaceuticals, Inc. REGN, MeiraGTx Holdings PLC MGTX and Verona Pharma PLC VRNA, all sporting a Zacks Rank #1 (Strong Buy). You can seethe complete list of todays Zacks #1 Rank stocks here.

Regenerons earnings estimates for 2020 have gone up from $28.31 to $29.18 and from $28.93 to $30.97 for 2021 over the past 30 days. Regenerons stock has returned 31% so far in 2020.

MeiraGTxs loss estimates for 2020 have narrowed from $2.41 to $2.06 and from $4.10 to $3.40 for 2021 over the past 30 days.

Veronas loss estimates for 2020 have narrowed from $3.95 to $2.65 and from $3.96 to $2.59 for 2021 over the past 30 days.

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Want the latest recommendations from Zacks Investment Research? Today, you can download 7 Best Stocks for the Next 30 Days. Click to get this free reportRegeneron Pharmaceuticals, Inc. (REGN) : Free Stock Analysis ReportAbeona Therapeutics Inc. (ABEO) : Free Stock Analysis ReportVerona Pharma PLC American Depositary Share (VRNA) : Free Stock Analysis ReportMeiraGTx Holdings PLC (MGTX) : Free Stock Analysis ReportTo read this article on Zacks.com click here.

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Abeona Treats First Patient in Pivotal Gene Therapy Study - Yahoo Finance

BOSTON and LONDON, March 18, 2020 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced that company founder and gene therapy pioneer Bobby Gaspar, M.D., Ph.D., has been named chief executive officer, effective immediately. Dr. Gaspar, previously president of research, chief scientific officer, and a member of the Orchard board of directors, succeeds Mark Rothera, who has served as the companys chief executive officer since 2017. As part of this transition process, Frank Thomas, Orchards chief operating officer and chief financial officer, will take on the role of president.

As a world-renowned scientist and physician, and accomplished strategic and organizational leader with more than 25 years of experience in medicine and biotechnology, Bobby Gaspar is uniquely qualified to lead Orchard into the future, said Jim Geraghty, chairman of the Orchard board of directors. In addition, Frank Thomas proven track record of success in leading operations, corporate finance and commercialization at a number of publicly traded life sciences companies will continue to be invaluable in his expanded role. On behalf of the entire Board of Directors, Id like to personally thank Mark for his many contributions to building Orchard into a leading gene therapy company over the last three years and wish him all the best in his future endeavors.

One of the companys principal scientific founders, Dr. Gaspar has served on Orchards board of directors and has driven its research, development and regulatory strategy since its inception. Over the course of his long career he has been a leading force in the development of hematopoietic stem cell (HSC) gene therapy bringing it from some of the first studies in patients to potential regulatory approvals. Dr. Gaspars unparalleled expertise, in addition to his deep relationships with key physicians and treatment centers around the world, will continue to be integral to efforts to identify and treat patients with metachromatic leukodystrophy (MLD) and other diseases through targeted disease education, early diagnosis and comprehensive newborn screening.

Dr. Gaspar commented: I am honored to become Orchards next CEO at a time of such opportunity for the company and for patients with severe genetic disorders. Through the consistent execution of our strategy, our talented team has advanced a leading portfolio of gene therapy candidates, expanding our R&D, manufacturing and commercial capabilities. We will now focus on driving continued innovation and growth, as well as strong commercial preparation and execution. I look forward to providing greater detail around our commercialization plan, pipeline prioritization and how we can realize the full potential of our HSC gene therapy platform, in the coming quarter.

Mr. Thomas commented: Im excited to be part of this next phase of Orchards evolution as a gene therapy leader as we look to refine our strategic priorities, ensure financial strength through improved operating efficiencies and prepare for a new cycle of growth, which includes our anticipated upcoming launch of OTL-200 in Europe. Im confident we will achieve long-term growth and value for our shareholders while turning groundbreaking innovation into potentially transformative therapies for patients suffering from devastating, often-fatal inherited diseases.

Mr. Rothera commented: It has been a great privilege to lead Orchard and this outstanding management team for the past three years. Orchard is poised to make a huge difference to the lives of patients worldwide living with devastating rare genetic conditions. Having worked closely with Bobby for the last several years, I know that he is tremendously talented, extremely passionate about the patient-centric mission, and fully prepared to lead Orchard as it enters its next phase as a company.

About OrchardOrchard Therapeutics is a global gene therapy leader dedicated to transforming the lives of people affected by rare diseases through innovative, potentially curative gene therapies. Our ex vivo autologous gene therapy approach harnesses the power of genetically-modified blood stem cells and seeks to permanently correct the underlying cause of disease in a single administration. The company has one of the deepest gene therapy pipelines in the industry and is advancing seven clinical-stage programs across multiple therapeutic areas where the disease burden on children, families and caregivers is immense and current treatment options are limited or do not exist, including inherited neurometabolic disorders, primary immune deficiencies and blood disorders.

Orchard has its global headquarters in London and U.S. headquarters in Boston. For more information, please visit http://www.orchard-tx.com, and follow us on Twitter and LinkedIn.

Availability of Other Information About Orchard

Investors and others should note that Orchard communicates with its investors and the public using the company website (www.orchard-tx.com), the investor relations website (ir.orchard-tx.com), and on social media (twitter.com/orchard_tx and http://www.linkedin.com/company/orchard-therapeutics), including but not limited to investor presentations and investor fact sheets, U.S. Securities and Exchange Commission filings, press releases, public conference calls and webcasts. The information that Orchard posts on these channels and websites could be deemed to be material information. As a result, Orchard encourages investors, the media, and others interested in Orchard to review the information that is posted on these channels, including the investor relations website, on a regular basis. This list of channels may be updated from time to time on Orchards investor relations website and may include additional social media channels. The contents of Orchards website or these channels, or any other website that may be accessed from its website or these channels, shall not be deemed incorporated by reference in any filing under the Securities Act of 1933.

Forward-Looking Statements

This press release contains certain forward-looking statements about Orchards strategy, future plans and prospects, which are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include express or implied statements relating to, among other things, the companys business strategy and goals, and the therapeutic potential of Orchards product candidates, including the product candidate or candidates referred to in this release. These statements are neither promises nor guarantees and are subject to a variety of risks and uncertainties, many of which are beyond Orchards control, which could cause actual results to differ materially from those contemplated in these forward-looking statements. In particular, the risks and uncertainties include, without limitation: the impact of the COVID-19 virus on Orchards clinical and commercial programs, the risk that any one or more of Orchards product candidates, including the product candidate or candidates referred to in this release, will not be approved, successfully developed or commercialized, the risk of cessation or delay of any of Orchards ongoing or planned clinical trials, the risk that prior results, such as signals of safety, activity or durability of effect, observed from preclinical studies or clinical trials will not be replicated or will not continue in ongoing or future studies or trials involving Orchards product candidates, the delay of any of Orchards regulatory submissions, the failure to obtain marketing approval from the applicable regulatory authorities for any of Orchards product candidates, the receipt of restricted marketing approvals, and the risk of delays in Orchards ability to commercialize its product candidates, if approved. Given these uncertainties, the reader is advised not to place any undue reliance on such forward-looking statements.

Other risks and uncertainties faced by Orchard include those identified under the heading "Risk Factors" in Orchards annual report on Form 10-K for the year ended December 31, 2019, as filed with the U.S. Securities and Exchange Commission (SEC) on February 27, 2020, as well as subsequent filings and reports filed with the SEC. The forward-looking statements contained in this press release reflect Orchards views as of the date hereof, and Orchard does not assume and specifically disclaims any obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as may be required by law.

Contacts

InvestorsRenee T. LeckDirector, Investor Relations+1 862-242-0764Renee.Leck@orchard-tx.com

MediaChristine C. HarrisonVP, Public Affairs & Stakeholder Engagement+1 202-415-0137media@orchard-tx.com

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Orchard Therapeutics Appoints Company Founder and Gene Therapy Pioneer Bobby Gaspar, MD, Ph.D., as New Chief Executive Officer - BioSpace

NEW YORK, March 18, 2020 /PRNewswire/ --

The report on the global zinc finger nuclease technology market provides qualitative and quantitative analysis for the period from 2017 to 2025.

Read the full report: https://www.reportlinker.com/p05874235/?utm_source=PRN

The report predicts the global zinc finger nuclease technology market to grow with a healthy CAGR over the forecast period from 2019-2025. The study on zinc finger nuclease technology market covers the analysis of the leading geographies such as North America, Europe, Asia-Pacific, and RoW for the period of 2017 to 2025.

The report on zinc finger nuclease technology market is a comprehensive study and presentation of drivers, restraints, opportunities, demand factors, market size, forecasts, and trends in the global zinc finger nuclease technology market over the period of 2017 to 2025. Moreover, the report is a collective presentation of primary and secondary research findings.

Porter's five forces model in the report provides insights into the competitive rivalry, supplier and buyer positions in the market and opportunities for the new entrants in the global zinc finger nuclease technology market over the period of 2017 to 2025. Further, IGR- Growth Matrix gave in the report brings an insight into the investment areas that existing or new market players can consider.

Report Findings1) Drivers Rising use of gene therapy and genome therapy Benefits offered by zinc finger nuclease technology such as permanent and heritable mutation and efficient creation of animal models2) Restraints Complexities associated with zinc finger nuclease technology3) Opportunities Application of zinc finger nuclease technology in drug discovery

Research Methodology

A) Primary ResearchOur primary research involves extensive interviews and analysis of the opinions provided by the primary respondents. The primary research starts with identifying and approaching the primary respondents, the primary respondents are approached include1. Key Opinion Leaders associated with Infinium Global Research2. Internal and External subject matter experts3. Professionals and participants from the industry

Our primary research respondents typically include1. Executives working with leading companies in the market under review2. Product/brand/marketing managers3. CXO level executives4. Regional/zonal/ country managers5. Vice President level executives.

B) Secondary ResearchSecondary research involves extensive exploring through the secondary sources of information available in both the public domain and paid sources. At Infinium Global Research, each research study is based on over 500 hours of secondary research accompanied by primary research. The information obtained through the secondary sources is validated through the crosscheck on various data sources.

The secondary sources of the data typically include1. Company reports and publications2. Government/institutional publications3. Trade and associations journals4. Databases such as WTO, OECD, World Bank, and among others.5. Websites and publications by research agencies

Segment CoveredThe global zinc finger nuclease technology market is segmented on the basis of type, and application.

The Global Zinc Finger Nuclease Technology Market by Type Cell Line Engineering Animal Genetic Engineering Plant Genetic Engineering Other

The Global Zinc Finger Nuclease Technology Market by Application Biotechnology Companies Pharmaceutical Companies Hospital Laboratory and Diagnostic Laboratory Academic and Research Institutes

Company Profiles Sigma-Aldrich Corporation Thermo Fisher Scientific Sangamo Therapeutics inc. LabOmics S.A. Gilead Sciences, Inc. OriGene Technologies, Inc Others

What does this report deliver?1. Comprehensive analysis of the global as well as regional markets of the zinc finger nuclease technology market.2. Complete coverage of all the segments in the zinc finger nuclease technology market to analyze the trends, developments in the global market and forecast of market size up to 2025.3. Comprehensive analysis of the companies operating in the global zinc finger nuclease technology market. The company profile includes analysis of product portfolio, revenue, SWOT analysis and latest developments of the company.4. IGR- Growth Matrix presents an analysis of the product segments and geographies that market players should focus to invest, consolidate, expand and/or diversify.

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Global zinc finger nuclease technology market is expected to grow with a healthy CAGR over the forecast period from 2019-2025 - PRNewswire

Barth syndrome is an X-linked metabolic disorder, affecting only males. It has widespread systemic effects presenting with cardiomyopathy, neutropenia, muscle weakness, stunted growth, exercise intolerance and abnormal skeletal structures. In many cases, it results in stillbirth. It is strongly related to mutations in the tafazzin gene, also known as TAZ. Currently only symptomatic treatment exists, and no definite cure has been developed for Barth syndrome.

Read Also: CRISPR Used for the First Time to Treat a Blind Patient

Researchers at Boston Childrens Hospital have proposed gene therapy as a potential treatment method to inhibit processes that lead to heart failure. The researchers conducted the study on mouse models with Barth syndrome.

A research to better understand Barth syndrome was conducted in 2014 by William Pu, MD and colleagues at Boston Childrens Hospital. Together, they created heart on chip models of Barth syndrome by using cardiac myocytes derived from patients with TAZ mutation. This led the researchers to discover the correlation between Barth syndrome and dysfunction. When the defective mutated TAZ myocytes was replaced by healthy TAZ gene myocytes, the cardiac dysfunction was spontaneously corrected.

Pu and colleagues realized that in order to fully understand the effects of Barth syndrome on the system, an animal body was crucial. Attempts at creating a whole body model had previously been done, but had not been successful.

The Beatson Institute for Cancer Research in the U.K has recently been successful in creating mouse models of Barth syndrome. Two categories of these mouse models were created, in the first category TAZ gene was deleted throughout the whole system whereas in the second category of mouse models ha TAZ gene deleted only from the cardiac myocytes.

The mouse models with whole body TAZ deletion died before birth mostly due to hypotonic weak musculature. However, some of the mice survived and developed cardiomyopathy, similar to the dilated cardiomyopathy in humans. The hearts left ventricle had thinner walls and dilated substantially which decreased the systolic pressure resulting in decreased cardiac output.

In those mice with deleted TAZ in heart muscle cells, all subjects survived but had cardiomyopathy issues and reduced cardiac output. Under electron microscope, the heart muscles were found to have abnormal structures and poor organization.

Read Also:UC Berkeley Researchers Restore Vision in Mice Through Gene Insertion

Using gene therapy, the researchers replaced the TAZ gene by administering a gentically engineered virus subcutaneously or intravenously. Whole body TAZ deletion mice survived to an average life span of healthy mice. It successfully prevented cardiac dysfunction in all mice models.

Only when more than 70 percent cardiac myocytes had taken up the modified TAZ gene, significant improvement was seen.

The problem is that neutralizing antibodies to the virus develop after the first dose, said Pu. Getting enough of the muscle cells corrected in humans may be a challenge.

Post introduction of TAZ gene corrected cells, the major problem was seen in sustaining the levels of modified gene cells. In comparison to cardiac myocytes, the number of corrected gene cells in skeletal muscles declined progressively.

https://www.sciencedaily.com/releases/2020/03/200309165231.htm

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Correcting Barth Syndrome With Gene Therapy - Gilmore Health News

BETHESDA, Md., March 18, 2020 /PRNewswire/ -- Internationally acclaimed clinical geneticist and pediatrician Harvey Levy, MD, FACMG, is the recipient of the 2020 ACMG Foundation for Genetic and Genomic Medicine's David L. Rimoin Lifetime Achievement Award in Medical Genetics.

Dr. Levy, senior physician in medicine and genetics at Boston Children's Hospital and professor of pediatrics at Harvard Medical School, is being honored for his many years of groundbreaking work with patients who have genetic metabolic diseases including phenylketonuria (PKU), homocystinuria, cobalamin metabolic disorder, and others; as well as for his training and mentoring of the next generation of genetics service providers; and for his major contributions to the development of newborn screening in the United States and around the world.

Dr. Levy's medical career spans more than 60 years. He hasmentored over 60 medical genetics fellows; published more than 400 research articles, reviews, book chapters, and proceedings from research meetings; written 2 books and created 2 educational videos for patients and clinicians; served on editorial boards and as a reviewer for numerous prominent research journals; and founded and formerly directed both the Maternal PKU Program and the Inborn Errors of Metabolism/PKU Program at Boston Children's Hospital.

"Harvey Levy is a physician scientist who has been instrumental in the development of newborn screening programs for metabolic diseases," said former ACMG Executive Director Dr. Michael S. Watson, FACMG. "Of particular importance has been his melding of knowledge of clinical genetics, population genetics and metabolic diseases to identify critical issues in the transition from a disease-based understanding of particular metabolic diseases to a population-based prevention program that has had enormous impact on hundreds of newborns in the United States."

"We take newborn screening for granted now," said Gerard Berry, MD, director of the Metabolism Program and professor of pediatrics at Harvard Medical School. "There are laws in different states that babies need to be screened for certain diseases. But when Harvey began, this was uncharted territory. People didn't understand the power of newborn screening and how it could change lives by allowing someone to get on a diet or a medication that they need to take for life in order to be healthy. Harvey played a major role in allowing all of this to come to fruition. These same individuals, who might have been institutionalized years ago because of severe intellectual disability, are now students in elite colleges. Harvey possesses insight and super-ability to understand what is really important for healthcare. Newborn screening is one of the major healthcare successes of the previous centurymaybe the most important healthcare success. And Harvey was part of a group of unique individuals who helped to see that through."

The news that he had received the David L. Rimoin Lifetime Achievement Award came to Dr. Levy as a delightful surprise. "This means a great deal to me because of the ACMG, where I've been an active member for a number of years," Levy shared. "It's a wonderful organization full of outstanding individuals, so to be in that company is particularly gratifying. And it's very, very nice to be appreciated."

"The Rimoin family is proud to recognize Dr. Harvey L. Levy, whose outstanding work includes studies that formed the basis for newborn metabolic screening, the discovery of the first human vitamin B12defect and the establishment of cobalamin defects, and the development of Maternal PKU programs," said Dr. Ann Garber, David Rimoin's surviving spouse."Based on his scientific accomplishments, along with his remarkable integrity, empathy and collaboration, our family is pleased to honor Dr. Levy with the David L. Rimoin Lifetime Achievement Award."

Beyond his list of academic achievements and leadership positions, the nominations for Dr. Levy to receive this award stressed his abounding generosity of time, knowledge and skill while working with patients, families and the broad range of clinical providers and researchers who have collaborated with him.

"He's dedicated himself to the study of PKU and metabolic disorders with an energy and intellect and soul that is extraordinary," said neuropsychologist Susan Waisbren, PhD, a professor of psychology at Harvard Medical School and Dr. Levy's long-time collaborator at Boston Children's Hospital Metabolism Clinic. "One of the qualities I've always found striking is the respect he has for professions outside of medicine. In his mind, every member of the clinical team is important. He truly feels this and it shows in his academic as well as clinical work. He has included as co-authors psychologists, dieticians, social workers, genetic counselors, nurses, administrators, secretaries, and parents.

"The patients adore him, always," she added, "and they recognize a certain compassion and ability to see the whole person, not just the metabolic disorder."

"Harvey is one of those special individuals who one may encounteronce in a lifetime," said Dr. Levy's collaborator at Boston Children's Hospital, Dr. Berry. "He's much more than an accomplished geneticistand investigator.First and foremost, he's a very endearing individual with a wonderful bedside manner, and he's beloved by patients and families whom he's cared for over the years. Harvey goes out of his way to make things better for patients and their families."

As an example, Dr. Berry, who has knownDr. Levy for several decades, recalled a case around 15 years ago, when a baby had been born with PKU in a suburban hospital outside of Boston. "Without telling anyone, Harvey drove to the hospital just to say hello to the new parents and to see the baby," Dr. Berry recounted. "He didn't need to do that. Everything was already in place, people were already taking care of what needed to be done, but he felt compelled to drive out there on a Friday evening to say hello."

Harvey L. Levy was born in Augusta, Georgia in 1935, the eldest of three sons. His father owned a one-room mercantile that supplied clothing to families of the surrounding area, which comprised mostly farmland during that period. His mother, who was a homemaker, graduated from Hunter College and served as a technician in a research laboratory in New York before her marriage. He credits her with some of his initial interest in research.

"I was a guy who was looking for answers to things, so I was always interested in science. And I particularly liked chemistry," Dr. Levy recalled. "My mother was a very intelligent person and very interested in education and music and arts, and also interested in science. I talked with my mother quite a bit about science. So, I think she had a feeling that maybe it would be a good idea for me to be a doctor."

Dr. Levy began studying history as an undergraduate student at Emory University and then switched to an early admission program at the Medical College of Georgia. One of his medical school professors, the famous Dr. Victor Vaughan, headed the department of pediatrics and had a profound influence on the direction of Dr. Levy's career. "I was always interested in pediatrics because of its developmental aspects," explained Dr. Levy. "I felt that if I was going to do something in terms of disease, preventing or helping patients in a significant way, I had to start early, and the earlier the better."

After completing his medical degree in 1960, Dr. Levy served an internship in pediatrics at the Boston City Hospital under Dr.Sydney Gellis, a renowned teacher of pediatrics. Following the internship he moved to New York and the Columbia-Presbyterian Medical Center, where he spent a year under Dr. Dorothy Anderson, the discoverer of cystic fibrosis. Then, as world events escalated toward the start of the Vietnam War, he was drafted and served 2 years in the Unites States Navy as a medical officer stationed in the Philippines.

His introduction to genetics came when he returned to his medical training in 1964 as a second-year pediatrics resident at Johns Hopkins University, where he met the pioneering pediatric clinical geneticist Dr. Barton Childs. What he learned from Dr. Childs about DNA triggered memories of an earlier time, and brought forth questions that further defined Dr. Levy's future career.

"If I go back to my childhood, my upbringing, I had three cousins from one of my father's brothers, whose family we were very close to, and all of these cousins were developmentally disabled," Dr. Levy said. " No reason was given for their disability and I always thought if I got into genetics, then maybe I could discover the causes of brain disease, particularly intellectual disability, and maybe I could influence the prevention of it."

Dr. Levy returned to Boston, where he served as Chief Resident in Pediatrics back at the Boston City Hospital. During that year he heard a lecture by Dr. Mary Efron, director of the Amino Acid Laboratory at Massachusetts General Hospital, in which she described her studies on metabolic disorders and their enzymatic defects as well as how newborn screening was helping clinicians to identify infants with these disorders so they could receive immediate preventive treatment.

"I became so fascinated with that. It was just absolutely the thing that I really wanted to do," recalled Dr. Levy. "Here was chemistry, biochemistry, genetics, and the prevention of disease! So I asked Dr. Efron if I could do a fellowship with her, which resulted in an NIH-funded fellowship at Massachusetts General Hospital. And that began the journey that has continued to this day."

One cold, fateful Friday afternoon while he was working in Dr. Efron's lab, a telephone call came from Dr. Robert MacCready, director of the Massachusetts Newborn Screening Program. Dr. MacCready asked if someone could come to the screening lab to look at an unusual screening result. Dr. Efron was ill, so Dr. Levy rode his bicycle seven miles across town to the State Laboratory Institute, where he recognized the unusual spot on the paper chromatogram test as a high level of methionine, the hallmark of a genetic disorder he had recently learned about called homocystinuria.

"I called the baby's doctor and asked if I could see the baby at the Massachusetts General Hospital the following Monday," Dr. Levy recalled. "The family and baby came that Monday and I confirmed that the infant indeed had homocystinuria. I asked if they had other children, and was told, 'Yes, we have a daughter.' And I asked if she was ok, and they said she was fine. I asked to see her and she was brought to the next visit, where I immediately recognized that she was developmentally delayed and had other features of homocystinuria that had only recently been described. She was born before screening for homocystinuria had begun. So that launched me into the field of methionine metabolism and some very interesting new areas of research." Much of this research was in collaboration with the late Dr. Harvey Mudd of the NIH, who was the world's foremost authority on methionine and on sulfur amino acid metabolism in general.

Dr. Efron passed away and Dr. Levy assumed Dr. Efron's position as consultant to the Massachusetts Newborn Screening Program and, in 1972, was appointed Director of the program. Four years later, he became Chief of Biochemical Genetics for the New England Newborn Screening Program, a position he held until 1997. Throughout this period, Dr. Levy collaborated with the famed, late microbiologist Robert Guthrie, MD, PhD, of Buffalo, New York, who had established newborn screening with his invention of the PKU test. During this time, he also continued to conduct research and to diagnose and treat patients with metabolic disorders at the Massachusetts General Hospital. An extraordinary influence for Dr. Levy during this time, and continuing to the present, is the internationally famous Canadian biochemical geneticist Dr. Charles Scriver, with whom Dr. Levy has often collaborated.

Toward the end of the 1970's Dr. Levy moved to Boston Children's Hospital, where he transformed the PKU Clinic it into a larger, comprehensive clinicthe Inborn Errors of Metabolism clinicthat now sees patients and families from around the world who are affected by a range of diseases: PKU, galactosemia, histidinemia, methylmalonic acidemia, problems with vitamin B12 metabolism and many other disorders. The hospital recently named the metabolic program after Dr. Levy.

At Boston Children's Hospital Dr. Levy became concerned about infants born to mothers who have genetic metabolic disease. "Before we began newborn screening girls who had PKU became delayed in their mental development, so very few bore children," Dr. Levy explained. "But now that we were treating them from infancy, they were bearing children. Even though their babies were genetically normal, they would be born with multiple severe problems if the mothers were not strictly treated for PKU during the pregnancies. So, with an extraordinary group of very talented professionals, including psychologists, nutritionists, a nurse, and a social worker, as well as physicians, we organized the New England Maternal PKU Program and followed these women on very strict dietary treatment throughout their pregnancies. We found that this regimen prevented many of these problems that the babies would otherwise have."

Today Dr. Levy is considered one of the foremost proponents worldwide for newborn screening. He led a successful effort in Massachusetts to expand newborn metabolic screening with new technology so that 20 to 30 disorders of amino acid, organic acid and fatty acid metabolism could be included rather than only 5 or 6 disorders previously screened. Within the ACMG, Dr. Levy led the effort to develop "ACT Sheets," one-page synopses of the newborn screened metabolic disorders so that physicians caring for infants can easily read an explanation of the biochemical, clinical and treatment characteristics of the disorders when contacted by a newborn screening program about an abnormality. As part of a contract funded by the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), Dr. Levy began and led the Newborn Screening Translational Research Network of the ACMG.

Though many of his contemporaries have retired, Dr. Levy continues to lead research efforts that examine the long-term outcomes of expanded newborn screening using tandem mass spectrometryincluding the medical, biochemical and neuropsychological outcomes in relation to early treatment. He is also involved with clinical trials to develop new therapies for PKU and homocystinuria. Dr. Levy is driven to continue his work because there is still much work to do. "The fact that we've had to rely on complicated diets that alter the lives of patients so they cannot enjoy a normal meal with their family or their friends, they have to only be able to eat this very difficult diet, and also the fact that we still discover diseases for which we have no treatment, " he explained, "these are the issues that trouble me. There are still individuals we discover during newborn screening or we discover later on because we didn't screen for their disorder, and they have severe disorders for which we have no treatments. There are still metabolic diseases that are not being prevented."

Dr. Levy still spends time communicating face-to-face with patients. "If you have a new baby, in a room with the family, you have to present this very complicated story, and the family has no idea what this is about," he explained. "So, we spend a great deal of time explaining the biochemistry, the genetics, the problems that can occur and the treatments that can prevent these problems. Early on, we just thought about biochemistry. But today we become more involved in talking about the genes, because we think it's important for families to understand the origin of these disorders since at some point we are likely to talk to them about the possibility of gene therapy, actually introducing the normal gene into the child. So, they need to understand where the disorder comes from. It's a complicated and long process. The family will take in as much information as they can, but as you can imagine, a lot of what we tell them will be forgotten or not understood. So, we go over everything with them again, and for as many times as they need."

One of the most pleasing aspects of Dr. Levy's career, he recounted, has been working with wonderful and dedicated individualspsychologists, nutritionists, dieticians, nurses, social workers, coordinators, administratorsand within the community of clinicians and researchers who study metabolic genetic disorders, a "relatively small, cohesive group of delightful, brilliant people" as he describes them. "It's been an extraordinarily wonderful professional life, as gratifying as any professional life I could ever dream of," reflected Dr. Levy. "Little did I know when I started that I would have this kind of life and little did I know that I would be awarded with the awards and certainly nothing comparable to the David L. Rimoin Lifetime Achievement Award."

The David L. Rimoin Lifetime Achievement Award is the most prestigious award given by the ACMG Foundation. A committee of past presidents of the American College of Medical Genetics and Genomics selects the recipient following nominations, which come from the general membership.

About the ACMG Foundation for Genetic and Genomic Medicine

The ACMG Foundation for Genetic and Genomic Medicine, a 501(c)(3) nonprofit organization, is a community of supporters and contributors who understand the importance of medical genetics and genomics in healthcare. Established in 1992, the ACMG Foundation supports the American College of Medical Genetics and Genomics (ACMG) mission to "translate genes into health." Through its work, the ACMG Foundation fosters charitable giving, promotes training opportunities to attract future medical geneticists and genetic counselors to the field, shares information about medical genetics and genomics, and sponsors important research.To learn more and support the ACMG Foundation mission to create "Better Health through Genetics" visit http://www.acmgfoundation.org.

Note to editors: To arrange interviews with experts in medical genetics, contact ACMG Senior Director of Public Relations Kathy Moran, MBA at kmoran@acmg.net.

Kathy Moran, MBAkmoran@acmg.net

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Pediatrician and Geneticist Dr. Harvey Levy Receives 2020 David L. Rimoin Lifetime Achievement Award in Medical Genetics from the ACMG Foundation for...

Global Gene Therapy and Antisense Drugs Market: Snapshot

Since an escalating number of patients are being diagnosed with cancer every day, the global demand for gene therapy and antisense drugs is bound to multiply. The growing incidence of health conditions such as Parkinsons disease and high cholesterol is also boosting the demand for gene therapy and antisense drugs. Several new drugs and therapies have been making their debut in the global gene therapy and antisense drugs market of recent.

For instance, in June 2017, it was announced that a group of scientists at the University of Queensland have developed a new technique that is capable of permanently silencing severe allergies triggered by shellfish, peanuts, and venom. The treatment was found to be successful in animal trials. The technique holds considerable potential for treating asthma completely. During the same month, results of another research study for treating multiple myeloma, a type of blood cancer, were announced at a conference held by the American Society of Clinical Oncology. The new CAR-T therapy consists of filtering the blood of the patients in order to eliminate T cells which are then genetically altered and given back to the patients.

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However, the difficulty pertaining to the delivery of antisense technology to the brain can present key challenges to the expansion of the global market for gene therapy and antisense drugs. Moreover, the unavoidable toxic aftereffects associated with the technology can also inhibit the growth of the gene therapy and antisense drugs market worldwide. However, the present attempts at developing safe and efficient antisense drugs by several market participants including Ionis Pharmaceuticals, which undertook testing of the technology on transgenic mice, can promote the further expansion of the market.

Global Gene Therapy and Antisense Drugs Market: Overview

Antisense gene therapy is emerging as one of the most beneficial therapeutics for various diseases such as tumors, cancer etc. These newer therapies are based on increased knowledge of molecular events that lead to disordered cellular growth. The therapy involves using a gene silencing technique rather than a gene repairing technique for silencing the genes effect.

The research report is a valuable tool for comprehending the progression of the global gene therapy and antisense drugs market between 2017 and 2025.

Global Gene Therapy and Antisense Drugs Market: Treatment Insights

Antisense drugs attach to the mRNA of a target protein, which inhibits the protein production process. Antisense therapeutics can obstruct the expression of oncogenes and other cancer-related genes that express growth factors. Antisense gene therapy involves the utilization of various therapeutic strategies which requires a clear knowledge of the molecular anatomy of cancer related genes. Antisense gene therapy is used to treat various diseases such as hemorrhagic fever, cancer, cystic fibrosis, renal diseases, HIV/AIDS, spinal muscular atrophy, and cardiovascular diseases.

Global Gene Therapy and Antisense Drugs Market: Market Segmentation

On the basis of therapeutic area, the gene therapy and antisense drugs market is segmented into cancer, anemia, rheumatoid arthritis, cardiovascular diseases, HIV/AIDS, cystic fibrosis, diabetes mellitus and obesity, and renal diseases.

By gene transfer method, ex vivo gene transfer and in vivo gene transfer are the segments of the market. The former involves the transfer of cloned genes into cells, i.e., cells are altered outside the body before being implanted into the patient, whereas the latter involves the transfer of cloned genes directly into the patients tissues. The outcome of in vivo gene transfer technology mainly depends on the general efficacy of gene transfer and expression.

Global Gene Therapy and Antisense Drugs Market: Regional Outlook

The global gene therapy and antisense drugs market is segmented into North America, Asia Pacific, Europe, and Rest of the World. Amongst these, North America holds the leading position in the market followed by Europe. The increasing incidence of cancer and other fatal diseases, unhealthy lifestyle practices such as excessive smoking and excessive consumption of high fat content food, and increasing research efforts for treatment against cancer are the major factors driving the gene therapy and antisense drugs market in these regions.

Asia Pacific is expected to emerge as a significant market for gene therapy and antisense drugs. The high population density including a large geriatric population, expeditiously increasing demand for technologically advanced therapeutics, and increasing government support for improved healthcare infrastructure in the region is driving the growth of this regional market. Furthermore, favorable reimbursement policies and tax benefits on newer therapies will further fuel the growth of the Asia Pacific gene therapy and antisense drugs market.

Major Companies Mentioned in Report

Some of the leading companies operating in the global gene therapy and antisense drugs market are GenVec Inc., Avigen Inc., Genome Therapeutics Corp., Tekmira Pharmaceuticals Corporation, Isis Pharmaceuticals, Cell Genesys Inc., and others. These companies are profiled for their key business attributes in the report.

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Gene Therapy and Antisense Drugs Market Growth Trends, Key Players, Competitive Strategies and Forecasts to 2025 - 3rd Watch News

Report Description

A recent market intelligence report that is published by Data Insights Partner onGene Therapy marketmakes an offering of in-depth analysis of segments and sub-segments in the regional and international Gene Therapy market. The research also emphasizes on the impact of restraints, drivers, and macro indicators on the regional and world Gene Therapy market over the short as well as long period of time. A detailed presentation of forecast, trends, and dollar values of international Gene Therapy market is offered. In accordance with the report, the global Gene Therapy market is projected to expand at a CAGR of 30% over the period of forecast.

Market Insight, Drivers, Restraints& Opportunity of the Market:

Gene therapy is a medical procedure which replaces defective genes or introduces new genes n order to prevent or cure genetic disorders. This procedure has become a bench mark in medical industry as there is no requirement of surgery or drugs or other procedure which has side effects on the individuals. Gene therapy was first commercialized in China in 2004 by China based SiBono Gene Tech (product Gendicine).

The global gene therapy market has been expanding due to the rigorous research conducted in the field of genetics. The rising awareness about the capability of cure of several rare genetic diseases by gene therapy is another important driver which leads the global gene therapy market during the forecast period. Gene therapy has capability cure several life threatening diseases such as cancer, cardiac diseases, AIDS, cystic fibrosis, age-related disorders, sickle cell anemia etc. In March 2019, the director of the National Health Institute (NIH), the U.S. announced that the recent clinical trials on the gene therapy for the treatment of sickle cell anemia showed promising result- therefore, increasing prevalence of aforementioned lie threatening diseases would likely to drive the growth of the global gene therapy market during the forecast period.

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On the other hand, treatment cost and stringent regulatory conditions etc. may hamper the growth of the global gene therapy market in the upcoming future. The results of Gendicine clinical trials were published in 2003 and the medicine got approval by the China State Food and Drug Administration in the same year. Although approved in China, Gendicines use is not very promising outside China. There are several concerns among the researchers about the quality of the clinical trials performed and safety and efficacy of the treatment. However, Gendicines equivalent Advexin (company Introgen Therapueitcs) is still waiting for the FDA approval.

Increasing investment to the gene therapy related research (around 10 Bn was invested in 2015 by private and public organizations), new product developments such as (Zolgensma in 2019), strategic alliance among the key players (such as collaboration between Axovant and Yposkesi) would bring the global gene therapy market an opportunity to propel during the forecast period. In May 2019, Avexis (a Novartis company) has got the FDA approval for Zolgensma for treatment of spinal muscular atrophy for the pediatric patients (less than 2 years of age).

Segment Covered:

This market intelligence report on the global gene therapy market encompasses market segments based on product, application, target user and geography. On the basis of product, the sub-markets is segmented into Yescarta, Kymriah, Strimvelis, Gendicine, Zolgensma and others (Advexin). Based on application, the global gene therapy market has been segregated into large B-Cell lymphoma, Car T Cell therapy, ADA-SCID (adenosine deaminase deficiency), muscular atrophy, head and neck squamus cell carcinoma, others (Crigler-Najjar syndrome). By target user, the global gene therapy market is also classified into adult and pediatric. By Geography, the global gene therapy market has been divided into North America (the U.S., Canada), Latin America (Brazil, Mexico, Argentina and other countries), Europe (Germany, France, the U.K., Spain, Italy, Russia, and other countries), Asia Pacific (India, Japan, China, Australia and New Zealand and other countries), Middle East and Africa (GCC, South Africa, Israel and Other countries).

Profiling of Market Players:

This business intelligence report offers profiling of reputed companies that are operating in the market. Companies such as Novartis, Gilead Sciences, Orchard Therapeutics Ltd, SiBiono GeneTech Co, Introgen Therapeutics and among others have been profiled into detail so as to offer a glimpse of the market leaders. Moreover, parameters such as gene therapy market related investment & spending and developments by major players of the market are tracked in this global report.

Report Highlights:

In-depth analysis of the micro and macro indicators, market trends, and forecasts of demand is offered by this business intelligence report. Furthermore, the report offers a vivid picture of the factors that are steering and restraining the growth of this market across all geographical segments. In addition to that, IGR-Growth Matrix analysis is also provided in the report so as to share insight of the investment areas that new or existing market players can take into consideration. Various analytical tools such as DRO analysis, Porters five forces analysis has been used in this report to present a clear picture of the market. The study focuses on the present market trends and provides market forecast from the year 2017-2027. Emerging trends that would shape the market demand in the years to come have been highlighted in this report. A competitive analysis in each of the geographical segments gives an insight into market share of the global players.

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Salient Features:

This study offers comprehensive yet detailed analysis of the Gene Therapy market, size of the market (US$ Mn), and Compound Annual Growth Rate (CAGR (%)) for the period of forecast: 2019 2027, taking into account 2017 as the base year

It explains upcoming revenue opportunities across various market segments and attractive matrix of investment proposition for the said market

This market intelligence report also offers pivotal insights about various market opportunities, restraints, drivers, launch of new products, competitive market strategies of leading market players, emerging market trends, and regional outlook

Profiling of key market players in the world Gene Therapy market is done by taking into account various parameters such as company strategies, distribution strategies, product portfolio, financial performance, key developments, geographical presence, and company overview

Leading market players covered this report comprise names such as. Novartis, Gilead Sciences, Orchard Therapeutics Ltd, SiBiono GeneTech Co, Introgen Therapeutics and among others

The data of this report would allow management authorities and marketers of companies alike to take informed decision when it comes to launch of products, government initiatives, marketing tactics and expansion, and technical up gradation

The world market for Gene Therapy market caters to the needs of various stakeholders pertaining to this industry, namely suppliers, manufacturers, investors, and distributors for Gene Therapy market. The research also caters to the rising needs of consulting and research firms, financial analysts, and new market entrants

Research methodologies that have been adopted for the purpose of this study have been clearly elaborated so as to facilitate better understanding of the reports

Reports have been made based on the guidelines as mandated by General Data Protection Regulation

Ample number of examples and case studies have been taken into consideration before coming to a conclusion

Reasons to buy:

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vIdentification of key factors driving investment opportunities in the Gene Therapy market

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Gene Therapy Market size Witness Growth Acceleration during 2027 - Packaging News 24

Britain is really good at biology. In physics and chemistry, or painting and music, we have often failed to match the Germans, the French or the Italians. But in the bio-sciences, nobody can equal us. Heres an astonishing list of firsts that happened on this damp island: William Harvey and the circulation of the blood. Robert Hooke and the cell. Edward Jenner and vaccines. Charles Darwin and natural selection. Alexander Fleming and antibiotics. Francis Crick and James Watson (and Rosalind Franklin and Maurice Wilkins) and the structure of DNA. Fred Sanger and DNA sequencing. Patrick Steptoe and Robert Edwards and the first test-tube baby. Alec Jeffreys and DNA fingerprinting. Ian Wilmut and Dolly the Sheep. The biggest single contribution to the sequencing of the human genome (the Wellcome Trust).

Annoyingly, the exciting new tool of genome editing is the one that got away. The best of the new tools, known as CRISPR, emerged from the work of a Spaniard, Francisco Mojica, who first spotted some odd sequences in a microbes genome that seemed to be part of a toolkit for defeating viruses. Then a few years ago French, American, Finnish, Dutch and Chinese scientists turned this insight into a device for neatly snipping out specific sequences of DNA from a genome in any species, opening up the prospect of neatly rewriting DNA to prevent disease or alter crops. Two American universities are squabbling over the patents (and Nobel prize hopes). Further improvements are coming thick and fast.

But we are well placed to catch up with superb labs straining at the leash to apply these new tools. The biggest immediate opportunity is in agriculture, and here leaving the European Union is absolutely key. There is no clearer case of a technology in which we will be held back if we do not break free from the EU approach. It would not be a race to the bottom in terms of safety and environmental standards, but the very opposite: a race to the top.

For example, if we allowed the genetically modified blight-resistant potatoes that have been developed at the Sainsbury Laboratory in Norfolk to be grown in fields here in the UK, we would be able to greatly reduce the spraying of fungicides on potato fields, which at present happens up to 15 times a year, harming biodiversity and causing lots of emissions from tractors. That would be a big improvement, not a regression, in environmental terms. But at the moment commercializing the Sainsbury Lab potato is in practice impossible because of onerous EU rules.

Other countries are already dashing ahead with the new technology. Last year a review of the patenting of CRISPR products in agriculture found that, whereas America had taken out 872 patent families and China 858, the European Union had taken out only 194. The gap is growing.

The reason is nothing to do with the quality of research in Europe. It is all about regulation. When genome editing first came along, the European Commission decided to delay for several years making up its mind about how to regulate the release of genome-edited organisms while it waited for the European Court of Justice to decide whether to treat this new technology as if it were like genetic modification (the process invented a generation ago for transferring genes between species) or a form of mutation breeding (the process invented two generations ago for randomly scrambling the genes of plants under gamma rays in the hopes of generating better varieties).

If it was like genetic modification, then it would be subject to draconian rules that amount to a de-facto ban. Nobody even tries to commercialize a GMO crop in Europe any more because you enter a maze of delay, obfuscation, uncertainty, expense and red tape from which you never emerge.

The result is that European agriculture is more dependent on chemical sprays than it would have otherwise been, as shown by research at Gottingen University: on average, GMOs have reduced the application of pesticides to crops wherever they have been grown by 37 per cent. So we have missed out on biological solutions and had to stick with chemical ones instead.

If on the other hand genome editing is like mutation breeding, then you can go ahead and plant a crop straight away here with no restrictions. This is, of course, mad, since mutation breeding is more likely (though still very unlikely) to produce an accidentally harmful result even than GMOs, but its an older technique and has been used for much of the food you eat, including organic food, and for some reason nobody at Greenpeace objects.

Brexit is a fantastic opportunity to do something no European continental competitor is allowed to

Genome editing is an even more precise and predictable technique than GMOs. It involves no transfer of foreign DNA and the incision is made at a specific location in a genome, not at random. It is clearly the safest of all these three techniques, and so said the European Courts advocate general in his advice to the court. But in July 2018 the ECJ, being a political entity, decided otherwise and told the commission what it wanted to hear, that it should treat genome-edited plants and animals as if they were GMOs.

There was fury and dismay throughout the laboratories of Europe. There would have been more in Britain if academics had not feared playing into the hands of Brexiteers while remaining was still a possibility. A Canadian biotech professor tweeted that this was a good day for Canada since it removed a competitor continent from the scene. The absurdity is illustrated by the fact that in some cases it is impossible to distinguish a genome-edited variety from a variety bred by hybridisation or lucky selection with the same trait. Stefan Jansson from Ume University in Sweden put it like this: Common sense and scientific logic says that it is impossible to have two identical plants where growth of one is, in reality, forbidden while the other can be grown with no restrictions; how would a court be able to decide if the cultivation was a crime or not?

Brexit therefore offers a fantastic opportunity to do something no European continental competitor is effectively allowed to do, and that will benefit the environment. We have great laboratories here, in Norwich, Nottingham, Rothamsted and Edinburgh among other places. But the private sector of plant biotechnology is all but extinct in Britain and will take some jump-starting.

Twenty years ago there were 480 full-time equivalent, PhD-level, private sector jobs in agricultural biotechnology in this country. Today there are just ten. That is what has happened to that whole sector in this country as a result of the misinformed and misguided green campaign against GMOs. Until politicians signal a sea change, the private sector will shun the UKs wonderful labs and the breakthroughs will be applied overseas, if at all.

As a new online tool called the Global Gene Editing Regulation Tracker has shown, America, Canada, Argentina, Brazil, Japan and much of the rest of the world are moving towards a nimbler and more rational regulatory approach: namely judging a crop not by the method used to produce it, but by the traits it possesses. If you can make a potato resistant to blight, what matters is whether the potato is safe, not whether it was made by conventional breeding, gamma-ray mutagenesis or genome editing.

[Visit GLPs global gene-editing regulation tracker and index to learn more.]

In the EU, if you made this potato by gamma-ray mutation breeding, scrambling its DNA at random in a nuclear reactor, the regulations would say: No problem. Go ahead and plant it. If you made it by the far more precise method of genome editing, in which you know exactly what you have done and have confined your activities to one tiny bit of DNA, you are plunged into a Kafkaesque labyrinth of regulatory indecision and expense. The House of Lords Science and Technology Committee, on which I sit, recommended we switch to regulation by trait, a few years back but it was not possible before Brexit.

Genome editing can bring not just environmental benefits but animal welfare benefits too. In 2017, scientists at the Roslin Institute near Edinburgh announced that they had genome-edited pigs to protect them against a virus called porcine reproductive and respiratory syndrome, PRRS. They used CRISPR to cut out a short section from the pig gene that made the protein through which the virus gained access to cell. The change therefore denied the virus entry. They did this without altering the function of the protein made by the gene, so the animal grew up to be normal in every way except that it was immune to the disease.

This means less vaccination, less medication and less suffering. What is not to like? (Incredibly, when I mentioned this case in a speech in the House of Lords, a Green Party peer objected that eradicating a disease that causes suffering in pigs might be a bad thing in case it allows a change in pig husbandry techniques. Even Marie Antoinette was never quite that callous.) But commercialising that animal in the UK is currently all but impossible until we change the rules.

Genome-editing technology could revolutionize conservation as well as agriculture. Looking far ahead into much more speculative science, the same scientists at the Roslin who made the virus-resistant pigs are now looking into how to control grey squirrels not by killing them, as we do now, but by using genome editing to spread infertility infectiously through the population, so that the population slowly declines while squirrels live happily into old age.

This technique, called gene drive, could transform the practice of conservation all around the world, especially the control of invasive alien species the single greatest cause of extinction among birds and mammals today. We could eliminate the introduced mosquitos on Hawaii whose malaria is slowly exterminating the native honeycreeper birds. We could get rid of the non-native rats and goats on the Galapagos which are destroying the habitat of tortoises and birds.

We could get rid of the signal crayfish from America that have devastated many British rivers. For those who worry that gene drive might run riot, there is a simple answer: it can and will be designed in each case to last for a certain number of generations, not forever. And it will be wholly species-specific, so it cannot affect, say, the native red squirrel.

Genome editing may one day allow the de-extinction of the great auk

Still more futuristically, genome editing may one day allow the de-extinction of the great auk and the passenger pigeon. To achieve this, we need to take four steps: to sequence the DNA of an extinct species, which we have done in the case of the great auk; to edit the genome of a closely related species inthe lab, which is not yet possible but may not be far off as genome editing techniques improve by leaps and bounds; to turn a cell into an adult animal, which is difficult, but possible through primordial germ cell transfer, again pioneered at the Roslin Institute; and to train the adults for living in the wild, which is hard work but possible.

Genome editing is also going to have implications for human medicine. Here the European Union is less of a problem, and home-grown regulation is already in good shape: cautious and sensibly applied under the Human Fertilization and Embryology Authority. Britain has already licensed the first laboratory experiments, at the Crick Institute, on the use of genome editing in human embryos, but this is for research into infertility, not for making designer babies.

There is universal agreement that germ-line gene editing to produce human beings with new traits must remain off-limits and be considered in future only for the elimination of severe disease, not for the enhancement of normal talents. This view is shared around the world: the Chinese rogue scientist He Jiankui, who claims he used CRISPR to make two babies HIV-resistant from birth, was sentenced to three years in prison last December.

In practice, fears about designer babies are somewhat exaggerated. The same issue comes up about once a decade with every new breakthrough in biotechnology. It was raised about artificial insemination in the 1970s, about in-vitro fertilization in the 1980s, about cloning in the 1990s and about gene sequencing in the 2000s. Indeed, it has been possible to choose or selectively implant sperm, eggs and embryos with particular genes for a long time now and yet demand remains stubbornly low.

Most people do not want to use IVF or sperm donation to have the babies of clever or athletic people, as they easily could, but to have their own babies: the technology has been used almost exclusively as a cure for infertility. Indeed, the more we find out about genomes, the harder it becomes to imagine anybody wanting to, let alone being able to, enhance specific traits in future children by fiddling with genes: there are just too many genes, each with only very small effects, interacting with each other in the creation of any particular behaviour or ability.

Imagine walking into a doctors clinic and being presented with a catalogue of expensive genetic changes that could be made to your future babys genes, each of which might have a tiny and uncertain effect. The truth is most people do not want to have especially clever or sporty offspring: they want children like themselves.

However, in contrast to germ-line gene editing, somatic genome editing will play a large part in medicine. It is already happening, for example in a process known as CAR-T cell therapy, in which an immune cell is genome-edited so that it will attack a specific tumour, then multiplied and injected back into the body as a form of live drug. If we encourage genome editing in Britain we will be in a position to cure some cancers, enhance agricultural yield, improve the nutrient quality of food, protect crops from pests without using chemicals, eradicate animal diseases, enhance animal welfare, encourage biodiversity and maybe bring back the red squirrel. If we do not, then China, America, Japan and Argentina will still push ahead with this technology and will follow their own priorities, leaving us as supplicants to get the technology second-hand.

Matt Ridley is a British journalist and businessman. He is the author of several books, including The Red Queen (1994), Genome (1999), The Rational Optimist (2010) and The Evolution of Everything (2015). Follow him on Twitter @mattwridley

This article originally ran at The Critic and has been republished here with permission.

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Why Brexit could jump start UK GMO, CRISPR researchonce stifled by 'dead hand' of EU regulation - Genetic Literacy Project