Archive for the ‘Gene Therapy Research’ Category

DUBLIN--(BUSINESS WIRE)--The "Global Sickle Cell Anemia Therapeutics Market Analysis 2019" report has been added to ResearchAndMarkets.com's offering.

The Global Sickle Cell Anemia Therapeutics market is expected to reach $5,567.75 million by 2026 growing at a CAGR of 12.5% during the forecast period. Sickle-cell anaemia is a disease which is categorized by the existence of sickle-shaped erythrocytes. Sickle-cell anaemia is an inherited, lifelong blood disorder that impacts haemoglobin causing serious damage to the body.

Some of the key factors driving the growth of sickle-cell anaemia therapeutics market are the unmet medical needs and the increase in sickle-cell diseases. However, the side effects associated with current therapies and low awareness among individuals will surely impede the growth of this market.

Based on the Product Type, the pharmacotherapy segment is estimated to have a lucrative growth due to the potential launch of promising pipeline candidates such as Voxelotor, Crizanlizumab, Rivipansel, and Altemia. It is also estimated to lead the market, owing to a strong pipeline and several promising drug launches throughout the forecast period.

The key vendors mentioned are Addmedica, Baxter, Bioverativ, Bluebird Bio, Bristol-Myers, Squibb, Daiichi Sankyo, Eli Lilly, Emmaus Medical, Gamida Cell, Gilead Sciences, Global Blood Therapeutics, GlycoMimetics, HemaQuest Pharmaceuticals, Imara, Ironwood Pharmaceuticals, Johnson & Johnson, Mast Therapeutics, Merck Sharp & Dohme, Micelle BioPharma, and Modus Therapeutics.

Key Questions Answered in this Report:

Key Topics Covered:

1 Market Synopsis

2 Research Outline

3 Market Dynamics

3.1 Drivers

3.2 Restraints

4 Market Environment

4.1 Bargaining power of suppliers

4.2 Bargaining power of buyers

4.3 Threat of substitutes

4.4 Threat of new entrants

4.5 Competitive rivalry

5 Global Sickle Cell Anemia Therapeutics Market, By Medication

5.1 Introduction

5.2 Antibiotics

5.3 Hydroxyurea

5.4 Pain-Relieving Medications

5.5 Folic Acids

5.6 Antimetabolites

5.7 Vaccines

5.8 Analgesics

5.9 Other Medications

6 Global Sickle Cell Anemia Therapeutics Market, By Therapy

6.1 Introduction

6.2 Hydroxyurea

6.3 L-Glutamine

7 Global Sickle Cell Anemia Therapeutics Market, By Product Type

7.1 Introduction

7.2 Blood Transfusion

7.3 Bone Marrow Transplant

7.4 Gene Therapy

7.5 Pharmacotherapy

8 Global Sickle Cell Anemia Therapeutics Market, By Application

8.1 Introduction

8.2 Adult

8.3 Child

9 Global Sickle Cell Anemia Therapeutics Market, By End User

9.1 Introduction

9.2 Government

9.3 Home Healthcare

9.4 Hospitals

9.5 Infusion Centers

9.6 Private Clinics

9.7 Other End Users

10 Global Sickle Cell Anemia Therapeutics Market, By Geography

10.1 North America

10.2 Europe

10.3 Asia Pacific

10.4 South America

10.5 Middle East & Africa

11 Strategic Benchmarking

12 Vendors Landscape

12.1 Addmedica

12.2 Baxter

12.3 Bioverativ

12.4 Bluebird Bio

12.5 Bristol-Myers Squibb

12.6 Daiichi Sankyo

12.7 Eli Lilly

12.8 Emmaus Medical

12.9 Gamida Cell

12.10 Gilead Sciences

12.11 Global Blood Therapeutics

12.12 GlycoMimetics

12.13 HemaQuest Pharmaceuticals

12.14 Imara

12.15 Ironwood Pharmaceuticals

12.16 Johnson & Johnson

12.17 Mast Therapeutics

12.18 Merck Sharp & Dohme

12.19 Micelle BioPharma

12.20 Modus Therapeutics

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Global Sickle Cell Anemia Therapeutics Industry Analysis 2019 - Market Developments and Financials of Key Players - ResearchAndMarkets.com - Business...

ReportsnReports added a new report on The Gene Therapy in CVMD report that delivers the clean elaborated structure of the Report comprising each and every business-related information of the market at a global level. The in-depth study on the current state which focuses on the major drivers and restraints for the key players. Gene Therapy in CVMD Industry research report provides granular analysis of the market share, segmentation, revenue forecasts, geographic regions of the market and analytical tools such as SWOT analysis to generate a whole set of trade based studies regarding the Gene Therapy in CVMD.

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Gene therapies have been a point of discussion during the last several years as a potential curative option for a variety of disease indications. While mainly still in preclinical stages, gene therapy aims to treat or alleviate a disease by genetically modifying the cells of a patient. This report focuses on gene therapies in development across the 8MM for cardiovascular and metabolic disorders, including coronary artery disease, critical limb ischemia, diabetic foot ulcers, and Pompe Disease. In addition, this report provides an assessment of the pipeline, clinical, and commercial landscape of gene therapies in CVMD supplemented with a variety of KOL and payer perspectives.

Scope of this Report-This report combines KOL and Payer insights along with data from the Pharma Intelligence Center with in-house analyst expertise to provide a competitive assessment of the disease marketplace. Components of the slide deck include Overview of CVMD and Gene Therapies: epidemiology and regulatory oversight Pipeline Assessment: regional breakdown, promising late-stage products, early-stage pipeline by molecule type Clinical Trials Assessment: trial breakdown by phase, leading industry and non-industry sponsors Market Access: considerations for reimbursement, pricing, and unmet needs Market Outlook: competitive assessment and key market events (2018-2025).

Reasons to buy this Report-

Develop and design your in-licensing and out-licensing strategies through a review of pipeline products and technologies, and by identifying the companies with the most robust pipeline. Develop business strategies by understanding the trends shaping and driving the global CVMD gene therapy market. Drive revenues by understanding the key trends, innovative products and technologies, market segments, and companies likely to impact the global CVMD gene therapy market in the future. Formulate effective sales and marketing strategies by understanding the competitive landscape and by analyzing the performance of various competitors. Identify emerging players with potentially strong product portfolios and create effective counter-strategies to gain a competitive advantage. Organize your sales and marketing efforts by identifying the market categories and segments that present maximum opportunities for consolidations, investments, and strategic partnerships.

Single User License: US $ 7995

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Table of Contents in this Report-1. Preface1.1 Related Reports 41.2 Upcoming Related Reports 51.3 Abbreviations 62. Executive Summary 92.1 Key Findings 102.2 KOL and Payer Insight on CVMD Gene Therapy Competitive Landscape 113. Overview of CVMD Gene Therapy 123.1 What is Gene Therapy? 143.2 Gene Transfer Methods and Vectors Used for Gene Therapy 183.3 Viral Vectors vs. Non Viral Vectors 203.4 Therapeutic Gene Therapy Strategies Employed in CVMD 243.5 Gene Therapy in the 8MM 254. Epidemiology Analysis 314.1 Coronary Artery Disease 334.2 Peripheral Artery Disease 344.3 Peripheral Artery Disease with Critical Limb Ischemia 354.4 Systolic Heart Failure 364.5 Diabetic Foot Ulcers 374.6 Diabetic Neuropathy 384.7 Pompe Disease 395. Pipeline Assessment 405.1 CVMD Gene Therapy Pipeline in the 8MM 425.2 Pipeline Products Phase III 435.3 AnGes MGs Collategene 445.4 Angionetics Generx 455.5 ViroMeds Donaperminogene Seltoplasmid 465.6 Renovas RT-100 495.7 Pipeline Products Phase II 505.8 ID Pharmas DVC-10101 515.9 Juventas JVS-100 525.10 UFs Gene Therapy to Activate Acid Alpha Glucosidase for Pompe Disease 546. Clinical Trials Mapping and Design 556.1 Clinical Trial Mapping 576.2 Clinical Trial Design 597. Pricing and Reimbursement Strategies Payer Perspective 607.1 Current CVMD Space 627.2 Challenges Associated with Reimbursement of Novel CVMD Therapies 637.3 Prospective Payer Strategies for CVMD Gene Therapies 648. Market Outlook 658.1 Phase III CVMD Gene Therapy Pipeline 678.2 Key Launch Dates for Phase II and III CVMD Gene Therapy Pipeline 719. Appendix 729.1 Sources 739.1 Methodology 749.2 Primary Research 759.3 About the Authors 76

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Gene Therapy in CVMD Report- Growth Analysis, Future Trends, Size Estimation, Insights and Share Analysis By 2026 - Stock Market Herald

Gene Therapy Products market research report looks at the key aspects of the market including its market improvement, development, position and others. The industry inquiries in this report provide an examination and data as specified by classes. It underlines the global key manufacturers and analyzes the market competition landscape. The report also comprises of a bottomless knowledge on market definition, market drivers and market restraints, classifications, applications, and engagements. What is more, Gene Therapy Products Market report conducts analysis on sales (consumption) of market, focuses on the top players to assess their sales, price, revenue and market share with volume and value for each region.

Global gene therapy products market is set to witness a substantial CAGR in the forecast period of 2019- 2026. The report contains data of the base year 2018 and historic year 2017. Rising cancer cases and unused potential for emerging markets are the major factors for the growth of this market.

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Few of the major competitors currently working in the globalgene therapy products marketareAdaptimmune., Anchiano Therapeutics, bluebird bio, Inc., CELGENE CORPORATION, GlaxoSmithKline plc., Merck KGaA, Novartis AG, Achieve Life Sciences, Inc., Spark Therapeutics, Inc., Abeona Therapeutics, Inc, Adverum, agtc, Arbutus Biopharma, Audentes Therapeutics, AveXis, Inc., CRISPR Therapeutics, Intellia Therapeutics, Inc and Gilead Sciences,Inc. among others.

Market Definition:Global Gene Therapy Products Market

Gene therapy or human gene therapy is a process which is used to modify gene for the treatment of any disease. Plasmid DNA, bacterial vector, human gene editing technology and viral vectors are some of the most common type of gene therapy products. The main aim of the gene therapy is to replace the dysfunctional genes. Somatic and germline are some of the most common type of the gene therapy.

Complete report on Global Gene Therapy Product Market Research Report 2019-2026 spread across 350 Pages, profiling Top companies and supports with tables and figures

Segmentation: Global Gene Therapy Products Market

Gene Therapy Products Market : By Product

Gene Therapy Products Market : By Application

Gene Therapy Products Market : ByGeography

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Key Developments in the Gene Therapy Products Market:

Gene Therapy Products Market Drivers

Gene Therapy Products Market Restraints

Competitive Analysis: Gene Therapy Products Market

Global gene therapy products market is highly fragmented and the major players have used various strategies such as new product launches, expansions, agreements, joint ventures, partnerships, acquisitions, and others to increase their footprints in this market. The report includes market shares of gene therapy products market for Global, Europe, North America, Asia-Pacific, South America and Middle East & Africa.

Key questions answered in the report :-

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Data Bridge Market Researchis a versatile market research and consulting firm with over 500 analysts working in different industries. We have catered more than 40% of the fortune 500 companies globally and have a network of more than 5000+ clientele around the globe. Our coverage of industries include Medical Devices, Pharmaceuticals, Biotechnology, Semiconductors, Machinery, Information and Communication Technology, Automobiles and Automotive, Chemical and Material, Packaging, Food and Beverages, Cosmetics, Specialty Chemicals, Fast Moving Consumer Goods, Robotics, among many others.

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Gene Therapy Products Market : Revenue Overview, Business Analysis 2020-2026 || Leading Players Adverum, agtc, Arbutus Biopharma, Audentes...

Viralym-M recently received EMA PRIME and U.S. FDA RMAT designations

Phase 3 pivotal and Phase 2 proof-of-concept studies to be initiated for Viralym-M in 2020 targeting six devastating and life-threatening viral pathogens in immunocompromised patients

AlloVir, a late-clinical stage allogeneic T-cell immunotherapy company, today announced it has been granted Orphan Drug Designation from the European Medicines Agency (EMA) for Viralym-M (ALVR105) as a potential treatment of viral diseases and infections in patients undergoing hematopoietic stem cell transplantation (HSCT). Viralym-M is the companys lead allogeneic, off-the-shelf, multi-virus specific T-cell therapy, being developed for the treatment and prevention of serious viral diseases caused by six commonly occurring, devastating viral pathogens in immunocompromised individuals: BK virus, cytomegalovirus, human herpes virus-6, Epstein Barr virus, adenovirus, and JC virus. Viral diseases are a primary reason for poor outcomes in transplant patients, resulting in potentially devastating and life-threatening consequences.

In addition to Orphan Drug Designation, Viralym-M has been granted PRIority MEdicines (PRIME) designation from the EMA and Regenerative Medicine Advanced Therapy (RMAT) designation from the U.S. Food and Drug Administration (FDA). Viralym-M is one of only seven investigational therapies, to date, to receive both PRIME and RMAT designations from the EMA and FDA, respectively. AlloVir plans to initiate Phase 3 pivotal and Phase 2 proof-of-concept studies with Viralym-M in 2020 targeting six commonly occurring, devastating and life-threatening viral pathogens.

At AlloVir, we are committed to advancing allogeneic, off-the-shelf novel T-cell therapies with the potential to improve the way we treat and prevent devastating viral diseases, said Agustin Melian, MD, Chief Medical Officer and Head of Global Medical Sciences of AlloVir. The Orphan Drug Designation by the EMA acknowledges the critical need for new treatment options for patients who have undergone stem cell transplant and are at risk of the serious consequences of viral diseases. Also, leveraging PRIME and RMAT designations, we are working to quickly advance Viralym-M through late-stage clinical development to bring, what we believe to be a transformative new therapy, to patients in the U.S., European Union and eventually around the world.

The EMA grants Orphan Drug Designation status for products intended for the treatment, prevention or diagnosis of life-threatening or chronically debilitating conditions that affect no more than five in 10,000 people in the European Union, and where the product represents a significant benefit over existing treatments. Orphan Drug Designation provides companies with certain benefits and incentives in the EU, including a 10-year period of market exclusivity after product approval, reduced regulatory fees and protocol assistance.

About Opportunistic Viral Diseases

In healthy individuals, virus-specific T-cells from the bodys natural defense system provide protection against numerous disease-causing viruses. However, in patients with a weakened immune system these viruses may be uncontrolled. Viral diseases are common, with potentially devastating and life-threatening consequences in immunocompromised patients. For example, up to 90% of patients will reactivate at least one virus following an allogeneic HSCT and two-thirds of these patients reactivate more than one virus, resulting in significant and prolonged morbidity, hospitalization and premature death. Typically, when viruses infect immunocompromised patients, standard antiviral treatment does not address the underlying problem of a weakened immune system and therefore, many patients suffer with life-threatening outcomes such as multi-organ damage and failure, and even death.

About Viralym-M (ALVR105)

AlloVirs lead product Viralym-M (ALVR105) is in late-stage clinical development as an allogeneic, off-the-shelf, multi-virus specific T-cell therapy targeting six common viral pathogens in immunocompromised individuals: BK virus, cytomegalovirus, adenovirus, Epstein-Barr virus, human herpesvirus 6, and JC virus. In a positive Phase 2 proof-of-concept study, published in the Journal of Clinical Oncology (Tzannou, JCO, 2017), greater than 90% of patients who failed conventional treatment and received Viralym-M, demonstrated a predefined criteria for a complete or partial clinical response, most with complete elimination of detectable virus in the blood and resolution of major clinical symptoms. The company plans to initiate pivotal and proof-of-concept studies with Viralym-M in 2020 for treatment and prevention of severe and life-threatening viral diseases.

Viralym-M has received Regenerative Medicine Advanced Therapy (RMAT) designation from the U.S. Food and Drug Administration (FDA) and PRIority MEdicines (PRIME) designation from European Medicines Agency (EMA).

About AlloVir

AlloVir, formerly ViraCyte, is an ElevateBio portfolio company that was founded in 2013 and is the leader in the development of novel cell therapies with a focus on restoring natural immunity against life-threatening viral diseases in patients with severely weakened immune systems. The companys technology platforms deliver commercially scalable solutions by leveraging off-the-shelf, allogeneic, multi-virus specific T cells targeting devastating viral pathogens for immunocompromised patients under viral attack. AlloVirs technology and manufacturing process enables the potential for the treatment and prevention of a spectrum of devastating viruses with each single allogeneic cell therapy. The company is advancing multiple mid- and late-stage clinical trials across its product portfolio.

AlloVirs investors include Fidelity Research and Management Company, Gilead Sciences, F2 Ventures, The Invus Group, Redmile Group, EcoR1, Samsara Biocapital, and Leerink Partners Co-investment Fund, LLC.

AlloVir is an ElevateBio portfolio company. More information can be found at http://www.allovir.com.

About ElevateBio

ElevateBio, LLC, is a Cambridge-based creator and operator of a portfolio of innovative cell and gene therapy companies. It begins with an environment where scientific inventors can transform their visions for cell and gene therapies into reality for patients with devastating diseases. Working with leading academic researchers, medical centers, and corporate partners, ElevateBios team of scientists, drug developers, and company builders are creating a portfolio of therapeutics companies that are changing the face of cell and gene therapy and regenerative medicine. Core to ElevateBios vision is BaseCamp, a centralized state-of-the-art innovation and manufacturing center, providing fully integrated capabilities, including basic and transitional research, process development, clinical development, cGMP manufacturing, and regulatory affairs across multiple cell and gene therapy and regenerative medicine technology platforms. ElevateBio portfolio companies, as well as select strategic partners are supported by ElevateBio BaseCamp in the advancement of novel cell and gene therapies.

ElevateBios investors include F2 Ventures, MPM Capital, EcoR1 Capital, Redmile Group, Samsara BioCapital, Emerson Collective, The Invus Group, Surveyor Capital (A Citadel company), EDBI, and Vertex Ventures.

ElevateBio is headquartered in Cambridge, Mass, with ElevateBio BaseCamp located in Waltham, Mass. For more information, please visit http://www.elevate.bio.

View source version on businesswire.com: https://www.businesswire.com/news/home/20200326005129/en/

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European Medicines Agency Grants Orphan Drug Designation to AlloVir's Viralym-M, an Allogeneic, Off-the-Shelf, Multi-Virus Specific T-Cell Therapy -...

- Leveraging proprietary technology and manufacturing platform to develop transformativegene therapies for multiple rare diseases with high unmet needs -

- Pipeline led by Phase 3 gene therapy candidate for treatment of recessive dystrophic epidermolysis bullosa (RDEB), with a BLA filing targeted for 2021 -

- Backed by world-class group of biotech operators and investors -

EXTON, Pa., March 25, 2020 (GLOBE NEWSWIRE) -- Castle Creek Biosciences, Inc., a privately held, late-stage gene therapy company, announced that it has received a new investment of $75 million to support the advancement of its clinical development pipeline. Castle Creek Biosciences is a portfolio company of Paragon Biosciences, which led the $55 million equity investment from Fidelity Management & Research Company and Valor Equity Partners, along with a $20 million venture loan from Horizon Technology Finance Corporation (HRZN).

Castle Creek Biosciences is leveraging its proprietary technology platform and commercial-scale manufacturing infrastructure to develop personalized gene therapies for rare diseases with high unmet needs. The company plans to use the funding to advance and expand its gene therapy pipeline, led by the Phase 3 clinical development of FCX-007 (NCT04213261), its gene therapy candidate for the treatment of RDEB. It will also use the funding to expand its current good manufacturing practices (cGMP) infrastructure located in the greater Philadelphia region.

Clinical results from the ongoing Phase 1/2 clinical trial for FCX-007 continue to show positive trends in safety and wound healing in RDEB patients. Current data from this clinical trial were presented at the inaugural World Congress on Epidermolysis Bullosa held in London during January of 2020. FCX-007 was administered to 10 non-healing chronic wounds of which eight achieved complete wound closure 12 weeks post-administration (80%) vs. no wound closure in intra-patient, matched non-treated wounds (0%). FCX-007 continues to be well tolerated up to 52 weeks post administration.

We are proud to have the strategic support of world-class investors whose impact enables our efforts to transform the lives of patients and the future of medicine, said John Maslowski, Chief Executive Officer of Castle Creek Biosciences. We are steadfast in our commitment to the epidermolysis bullosa community and will continue to keep patients, caregivers and clinicians informed on the progress of our current programs, including FCX-007 and diacerein topical ointment, while we expand the scope of our gene therapy platform.

Castle Creek Biosciences is led by a strong executive leadership team with a proven record of developing innovative and potentially life-changing treatments for conditions with the greatest medical need, said Jeffery Aronin, Chairman and Chief Executive Officer of Paragon Biosciences. As investors, we are excited by the progress that the team has made and are committed to growing the Castle Creek Biosciences platform to address multiple rare genetic diseases.

About Castle Creek Biosciences, Inc. Castle Creek Biosciences is a privately held company that develops and commercializes gene therapies for patients with rare and serious genetic diseases. The companys lead gene therapy candidate, FCX-007, is being evaluated for the treatment of recessive dystrophic epidermolysis bullosa (RDEB), the most severe and debilitating form of epidermolysis bullosa (EB). The company is also advancing clinical research evaluating a diacerein topical ointment, CCP-020, for the treatment of epidermolysis bullosa simplex (EBS) and other forms of EB. In addition, Castle Creek Biosciences is developing FCX-013, a gene therapy for the treatment of moderate to severe localized scleroderma. Castle Creek Biosciences is a portfolio company of Paragon Biosciences. For more information, visit castlecreekbio.com or follow Castle Creek on Twitter @CastleCreekBio.

About Paragon BiosciencesParagon is a life science innovator that invests in, builds, and advises bioscience companies. Our mission is to serve patients living with severe medical conditions which do not yet have adequate treatments. Paragons portfolio of independently-run bioscience companies focus on biopharmaceuticals, AI-enabled life science products, and advanced treatments such as cell and gene therapies. We help people live longer, healthier lives. For more information, please visit: ParagonBioSci.com.

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Castle Creek Biosciences Announces $75 Million Investment to Advance Development of Multiple Gene Therapy Candidates for Rare Diseases - Yahoo Finance

SEATTLE, March 25, 2020 /PRNewswire/ -- According to Coherent Market Insights, the global viral vector and plasmid DNA manufacturing market is estimated to be valued at US$ 427.2 million in 2019, and is expected to exhibit a CAGR of 22.8% over the forecast period (2019-2027).

Coherent_Market_Insights_Logo

Key Trends and Analysis of the Global Viral Vector and Plasmid DNA Manufacturing Market:

Key trends in the market include increasing incidences of cancer, rising number of product launches, and increasing collaboration and acquisition activities by key market players.

According to World Health Organization (WHO), in 2018, around 9.6 million cancer deaths occurred globally. Over the last decade, development of gene therapy for the treatment of the cancer has increased significantly. Gene therapy treatment for cancer include transfer of foreign genetic material in the targeted cancer cell in the host's body. Various types of viral vectors and plasmid DNA such as retrovirus and HGF plasmidare used in the development of gene therapy.

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Increasing product launches and approvals by regulatory authorities are expected to drive growth of the global viral vector and plasmid DNA manufacturing market over the forecast period. For instance, in December 2017, Spark Therapeutics received the U.S. FDA approval to launch the LUXTURNA in the U.S. market. It is the first FDA approved gene therapy for treatment for an inherited retinal disease (IRD) and the first adeno-associated virus (AAV) vector gene therapy approved in the U.S.

Furthermore, key players operating in the market are focused on adopting acquisition, agreement, and collaboration strategies, in order to expand their product offerings in markets. For instance, in December 2017, Merck KGaA entered into a commercial supply agreement with bluebird bio, Inc., a clinical-stage biopharmaceutical company. According to the agreement, Merck agreed to manufacture viral vectors for bluebird's gene therapy products targeting the rare genetic disorders.

Key Market Takeaways:

Key players operating in the global viral vector and plasmid DNA manufacturing market include

Lonza Group AG, FinVector Vision Therapies, Cobra Biologics and Pharmaceutical Services, Sigma-Aldrich Co. LLC, VGXI, Inc., VIROVEK, SIRION Biotech GmbH, FUJIFILM Diosynth Biotechnologies U.S.A., Inc., Sanofi, Cell and Gene Therapy Catapult, Brammer Bio, and MassBiologics.

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Market Segmentations:

Did not find what you were looking for? Here are some other topics:

DNA AND RNA SAMPLE PREPARATION MARKET

DNA and RNA samples are necessary for variety of applications in drug research and development and cancer studies. High quality DNA and RNA samples are important for a wide variety of research and clinical applications. Biological studies require purified and isolated nucleic acids as the first step and in all recombinant DNA techniques. The extraction of nucleic acids from biological material requires cell lysis, inactivation of cellular nucleases, and separation of the desired nucleic acid from cellular debris.

Read more @ https://www.coherentmarketinsights.com/market-insight/dna-and-rna-sample-preparation-market-3620

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LIFE SCIENCE PRODUCTS MARKET

Life science products include laboratory supplies & accessories, cell culture & fermentation processes, cell therapy technologies, chromatography products, bioprocess filtration, fixed and live cell research through imaging and analysis, sample collection products, recombinant proteins, cell lines, and antibodies. These products are used for drug discovery, tissue engineering, drug screening, forensic testing, and genetic analysis.

Read more @ https://www.coherentmarketinsights.com/market-insight/life-science-products-market-3652

GLYCOBIOLOGY MARKET

Glycobiology involves study of structural aspects, biosynthesis, and biology of polysaccharides and how they function in an organism. Study of glycobiology has variety of application in areas such as drug discovery and development, diagnostic applications, therapeutic application, and industrial applications.Complex structure of glycan's and difficulty in its study, high costs of spectrometry and high performance liquid chromatography are expected to hinder growth of the market.

Read more @ https://www.coherentmarketinsights.com/market-insight/glycobiology-market-3639

Contact Us:RajShahCoherent Market Insights1001 4th Ave.#3200Seattle, WA 98154Tel: +1-206-701-6702Email:sales@coherentmarketinsights.com

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Global Viral Vector and Plasmid DNA Manufacturing Market to Surpass US$ 2,205.6 Million by 2027 - CMI - Yahoo Finance

]]> The companies will work together on three initial programmes for gene therapies to treat rare disorders. Credit: PublicDomainPictures from Pixabay.

Takeda Pharmaceutical has signed a strategic collaboration and licence agreement with biotherapeutics developer Codexis to research and create gene therapies for rare disorders.

Codexis will use its CodeEvolver protein engineering platform to construct gene sequences encoding protein variants that could boost efficacy by improving activity, stability and cellular uptake.

Takeda will combine these transgenes and its gene therapy capabilities to develop candidates for treating various rare genetic diseases, including lysosomal storage disorders and blood factor deficiencies.

Codexis president and CEO John Nicols said: Our CodeEvolver platform technology enables the rapid engineering of novel genetic sequences that encode more efficacious proteins. The prospects of these improved sequences for the development of differentiated gene therapies for patients with rare diseases, therefore, holds great promise.

Takedas expertise in developing novel treatments for patients with rare genetic disorders, and its commitment to developing the best possible gene therapies, makes them an ideal partner for our growing Novel Biotherapeutics business unit.

The companies will work together on three initial programmes. Codexis will generate enzyme sequences that could be progressed as gene therapies into pre-clinical development.

Takeda will carry out the pre-clinical and clinical development, as well as commercialisation.

Apart from the three programmes, Takeda could launch up to four programmes for different target indications.

Codexis will obtain an upfront payment, research and development (R&D) fee reimbursement.

The company is also eligible for development and commercial milestone payments, and sales royalties on any commercial product resulting from the partnership.

Earlier this month, Takeda Pharmaceutical announced plans to develop a drug to treat Covid-19. The company will develop a polyclonal hyperimmune globulin (H-IG), referred to as TAK-888.

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Codexis and Takeda partner on gene therapies for rare diseases - Pharmaceutical Technology

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.

How this report will benefit you

Read on to discover how you can exploit the future business opportunities emerging in this sector.

In this brand new201-page reportyou will receive104 tables and 110 figures all unavailable elsewhere.

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

By ordering and reading our brand-new report today you stay better informed and ready to act.

To request sample pages from this report please contact Sara Peerun at sara.peerun@visiongain.com or refer to our website: https://www.visiongain.com/report/cell-therapy-technologies-market-forecast-2020-2030/#download_sampe_div

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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To request a report overview of this report please contact Sara Peerun at sara.peerun@visiongain.com or refer to our website: https://www.visiongain.com/report/cell-therapy-technologies-market-forecast-2020-2030/

Did you know that we also offer a report add-on service? Email sara.peerun@visiongain.comto discuss any customized research needs you may have.

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

To see a report overview please e-mail Sara Peerun on sara.peerun@visiongain.com

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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.

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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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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.

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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

Continued here:
Anchiano Therapeutics Reports Year-End 2019 Financial Results - GlobeNewswire

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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.

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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

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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.

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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.

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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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Zacks has just released a Special Report on the booming investment opportunities of legal marijuana.

Ignited by new referendums and legislation, this industry is expected to blast from an already robust $6.7 billion to $20.2 billion in 2021. Early investors stand to make a killing, but you have to be ready to act and know just where to look.

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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