Archive for the ‘Gene Therapy Research’ Category
Gene Therapy Cure Claims Are Premature, Advocates Say – POZ
Journalists received not one but three announcements this week from American Gene Technologies (AGT) touting the most promising potential cure for HIV in the world. But such claims amount to unjustified hype, advocates say. The experimental therapy has not yet been tested in humans andif it worksit could be years before its ready for clinical use.
AGT just received clearance from the Food and Drug Administration (FDA) to start the first Phase I human clinical trial of its genetically modified T-cell product, dubbed AGT103-T, which the company is developing in collaboration with researchers at the National Institute of Allergy and Infectious Disease.
From its research, AGT believes a cure is attainable and is now taking the significant step of testing in humans, the company announced in a press release. Added AGT founder and CEOJeff Galvin, I am confidentAGT103-Twill be an important step toward an eventual cure for HIV.
But advocates say such claims are not only premature, but also harmful because they give people with HIV the false impression that a cure is around the corner.
AGTs public relations strategy preys on the emotions of people living with HIV and has a deleterious effect on the understanding of the cure field overall, Seattle advocate Michael Louella told POZ. They make their outrageous comments, and these are then picked up and believed to be certain truth. Any attempt to promote a more nuanced and better-grounded understanding of gene therapy or the clinical process becomes impossible.
Although HIV can be suppressed indefinitely with combination antiretroviral therapy, it has proved exceedingly difficult to cure because a so-called reservoir of latent virus can remain hidden from the drugs in resting immune cells. Only two people appear to have been cured after bone marrow transplants from donors with HIV-resistant stem cellsa procedure far too dangerous for people who dont have life-threatening blood cancer.
Nonetheless, researchers are exploring numerous cure strategies, ranging from flushing HIV out of resting cells to genetically engineering immune cells to make them resistant to the virus. Most experts expect that a combination approach will likely be needed to maintain durable control of HIV after stopping antiretroviral therapythe definition of a functional cure.
AGTs process involves collecting immune cells from a patient and selecting those cells that target HIV antigens. A harmless lentivirus vector is then used to insert genes into the HIV-specific CD4 T cells that disable CCR5 receptorswhich most strains of HIV use to enter cellsas well as genes involved in HIV replication. The genetically modified CD4 cells are then reinfused back into the same patient in a single dose. The entire process takes 11 days.
The company said it expects the approach will provide durable control of genetically diverse strains of HIV, including those that use a different receptor (known as CXCR4) to enter cells. The experimental therapy should work to remove infected cells from the body and decrease or eliminate the need for lifelong antiretroviral treatment, AGT claims.
Another company, Sangamo BioSciences, previously reported promising results from early studies using a different gene therapy technique (a zinc finger nuclease) to edit out CCR5 receptors from T cells. Although it did not cure HIV, some study participants saw a reduction in the size of their viral reservoir and a long-term increase in CD4 counts. More recently, Chinese researcher He Jiankui used yet another technique (CRISPR-Cas9) to disable the CCR5 gene in human embryos in an effort to protect them from HIV.
AGTs approach not only uses a different gene-editing method to disable CCR5, but it also selects CD4 T cells that target HIV and protects them from destruction by the virus, thereby helping the selected cells survive and avoiding the wasted effort of modifying cells that wont attack HIV.
A recent medical journal report described preclinical studies of the approach, which showed that it is feasible to manufacture the modified HIV-specific CD4 T cells. AGT claims that in laboratory studies, the product demonstrates the ability to clear itself of HIV when challenged with the virus and HIV-infected human cells. The company has not yet reported results from studies of the experimental therapy in animals.
These findings were used to support AGTs investigational new drug application to the FDA to allow the company to proceed with a Phase I study in human volunteers, which will be conducted in Baltimore and Washington, DC. Eligible participants must have been on antiretroviral therapy for one to three years, have an undetectable viral load, have a stable CD4 count above 500 and may not have any AIDS-defining conditions.
AGT expects to enroll the first participant in September, with the first infusion of genetically modified T cells to be administered in December. The company said it expects initial data by the end of the year.
But this will be far too soon to determine whether the altered T cells persist in the body or whether they can maintain long-term viral suppression after antiretroviral therapy is discontinued.
Saying AGT believes there is a high likelihood that participants in the upcoming trial will be cured is beyond outrageous and completely undermines informed consent because its an unethical inducement to participate [in trials], Richard Jefferys of the Treatment Action Group told POZ.
And its not based on a shred of evidence. To my knowledge, theres no humanized mouse data, no macaque dataits all theory, he continued. "I would hope that they pause to reconsider their PR strategy and broaden their consultation with stakeholders, including community-based advocates.
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Gene Therapy Cure Claims Are Premature, Advocates Say - POZ
Applied DNA Positions LinearDNA Platform as the Next Generation Manufacturing Platform for Adeno-Associated Virus-based Gene Therapies Through New…
Aug. 17, 2020 10:00 UTC
STONY BROOK, N.Y.--(BUSINESS WIRE)-- Applied DNA Sciences, Inc. (NASDAQ: APDN) (Applied DNA or the "Company"), a leader in Polymerase Chain Reaction (PCR)-based DNA manufacturing that enables in vitro diagnostics, pre-clinical nucleic acid-based therapeutic drug candidates, supply chain security, anti-counterfeiting and anti-theft technology, today announced that it has filed a nonprovisional patent application with the United States Patent and Trademark Office (USPTO) entitled Methods and Systems of PCR-Based Recombinant Adeno-Associated Virus (AAV) Manufacture (the Patent). The Patent claims priority to a previously filed provisional patent application filed with the USPTO in August of 2019.
AAV is utilized to deliver a therapeutic gene of interest to a patient or a patients cells to cause the expression of the necessary protein to address a targeted disease. Reflective of its potential as one of the most promising delivery vehicles for genetic medicines, there are approximately 80 active or enrolling clinical trials that utilize AAV1. With its new patent application, Applied DNA seeks to leverage its LinearDNA platform to give gene and redirected cell therapy developers the ability to greatly improve the manufacture and quality of their AAV-vectored medicines that have the potential to address many diseases, including COVID-19.
AAV is generally accepted as the preferred vector for gene therapy, and so, has an enormous breadth as a therapeutic gene delivery system. AAV vectors are manufactured utilizing multiple plasmid DNAs as starting materials, and as the number of gene therapy programs increase, and with improving levels of clinical success and progression of therapies into late-phase clinical studies, the field is being challenged by the need for larger manufacturing scales, said Dr. James A. Hayward, president and CEO of Applied DNA Sciences. At Applied DNA, we believe we have solved the linear DNA production challenge and can make the DNA sequences at scale for gene therapies. Utilizing our LinearDNA platform to manufacture amplicons, we can deliver gene therapy constructs that potentially lower the risks that come with the use of plasmid-based AAV manufacture. As one of the only companies in the marketplace commercializing a scaled linear DNA manufacturing platform, we believe we are uniquely positioned to deliver linear DNA as an alternative to plasmids, and with this patent filing, we expand our addressable market to including AAVs.
AAV is manufactured by the triple transfection of three plasmid DNA constructs into packaging cell lines to produce recombinant adeno-associated virus. This triple transfection requires large amounts of DNAs that are currently manufactured via plasmids circular DNA constructs which are propagated in bacteria. The three DNA constructs necessary for successful AAV production are: (i) AAV Rep and Cap; (ii) AAV Helper; and (iii) the therapeutic cargo (transgene) flanked on either side by inverted terminal repeat sequences (ITR). The manufacture of plasmid DNA for the production of AAV, however, presents a number of significant challenges, including scalability, fidelity, mis-incorporation of plasmid-derived DNA sequences, high costs, and long lead times for cGMP production.
Concluded Dr. Hayward, The ITRs serve as the viral origins of replication and for packaging signals to assemble AAV in the host cell. But the triple transfection of the required plasmids, and in the strict stoichiometries required for AAV assembly, we believe adds enormously to the cost of traditional AAV manufacturing, limiting the availability of many gene therapies. Our success in simplifying the production of the critical plasmid genes for AAV production we believe enable AAV production with minimal or absent plasmids, potentially greatly enhancing production and lowering costs. The use of LinearDNA potentially lowers the risks that come with plasmids, including off-target DNA, contamination by the genes for antimicrobial resistance or other bacterial DNA, endotoxin contamination and the use of antibiotics.
The Patent claims methods for the use of specialized LinearDNATM amplicons, instead of plasmids, to manufacture AAV. The patent also claims methods and systems for the PCR-based manufacturing of AAV transgene constructs flanked on either side by the necessary ITR sequences via the Companys LinearDNA platform. Due to their complex secondary structures, the ITR sequences necessary for AAV production have historically been very challenging to amplify via PCR. Leveraging the Companys PCR expertise, the methods and systems claimed in the Patent utilize specialized PCR techniques and primer designs to mitigate the challenges caused by the ITR secondary structures, allowing for the high yield and high-fidelity manufacture of transgene ITR amplicons.
About Applied DNA Sciences
Applied DNA is a provider of molecular technologies that enable supply chain security, anti-counterfeiting and anti-theft technology, product genotyping and pre-clinical nucleic acid-based therapeutic drug candidates.
Visit adnas.com for more information. Follow us on Twitter and LinkedIn. Join our mailing list.
The Companys common stock is listed on NASDAQ under ticker symbol APDN, and its publicly-traded warrants are listed on OTC under ticker symbol APPDW.
Applied DNA is a member of the Russell Microcap Index.
Forward-Looking Statements
The statements made by Applied DNA in this press release may be forward-looking in nature within the meaning of Section 27A of the Securities Act of 1933, Section 21E of the Securities Exchange Act of 1934 and the Private Securities Litigation Reform Act of 1995. Forward-looking statements describe Applied DNAs future plans, projections, strategies and expectations, and are based on assumptions and involve a number of risks and uncertainties, many of which are beyond the control of Applied DNA. Actual results could differ materially from those projected due to the possibility of a failure to make timely payment on its outstanding secured convertible notes and resulting enforcement by noteholders of remedies on collateral which includes substantially all of Applied DNAs assets, its history of net losses, limited financial resources, limited market acceptance, the uncertainties inherent in research and development, future clinical data and analysis, including whether any of Applied DNAs or its partners diagnostic candidates will advance further in the preclinical research or clinical trial process, including receiving clearance from the U.S. Food and Drug Administration or equivalent foreign regulatory agencies to conduct clinical trials and whether and when, if at all, they will receive final approval from the U.S. FDA or equivalent foreign regulatory agencies, the unknown outcome of any applications or requests to U.S. FDA, equivalent foreign regulatory agencies and/or the New York State Department of Health, the fact that there has never been a commercial drug or viral vector product utilizing PCR-produced DNA technology approved for therapeutic use, disruptions in the supply of raw materials and supplies, the unknown outcome of the patent application, and various other factors detailed from time to time in Applied DNAs SEC reports and filings, including our Annual Report on Form 10-K filed on December 12, 2019 and our subsequent quarterly reports on Form 10-Q filed on February 6, 2020, May 14, 2020 and August 6, 2020, and other reports we file with the SEC, which are available at http://www.sec.gov. Applied DNA undertakes no obligation to update publicly any forward-looking statements to reflect new information, events or circumstances after the date hereof or to reflect the occurrence of unanticipated events, unless otherwise required by law.
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Applied DNA Positions LinearDNA Platform as the Next Generation Manufacturing Platform for Adeno-Associated Virus-based Gene Therapies Through New...
Global Gene Therapy for Age-related Macular Degeneration Market 2020 to Witness Huge Growth by 2026 | RetroSense Therapeutics, REGENXBIO, AGTC – Owned
The study on the Global Gene Therapy for Age-related Macular Degeneration Market strives to offer prominent and profound insights into the present market scenario and the emerging growth dynamics. The report on Gene Therapy for Age-related Macular Degeneration market also provides the market players as well as the new contenders a complete view of the market landscape. The detailed research will empower the well-established as well as the emerging players to organizetheir business planning and achieve their short-term and long-term goals.
Global financial markets are in crisis as the covid-19 coronavirus spreads worldwide. The coronavirus epidemic is relevant and has far-reaching implications for the market. Many industries are facing a rising number of critical concerns such as supply chain disruption, rising risk of recession, and a possible reduction in consumer spending. These scenarios will run in different regions and sectors, so that correct and timely market research is more important than ever. By studying all aspects, the report provides up to date market intelligence on Gene Therapy for Age-related Macular Degeneration market.
Note: Our analysts monitoring the situation across the globe explains that the market will generate remunerative prospects for producers post COVID-19 crisis. The report aims to provide an additional illustration of the latest scenario, economic slowdown, and COVID-19 impact on the overall industry.
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The Major players profiled in this report include:
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This report offers insights into a dynamic competitive environment. It also offers a progressive viewpoint on various factors driving or restricting the market growth. The report gives an overall view of the global Gene Therapy for Age-related Macular Degeneration market by categorizing it in terms of type, application and region. These segments are analyzed by current and future trends. Regional segmentation includes current and future demand for them in North America, Asia Pacific, Europe, and the Middle East. The report collectively covers specific application segments of the market in each region.
Global Gene Therapy for Age-related Macular Degeneration Market Split by Product Type and Applications:
Types of Global Gene Therapy for Age-related Macular Degeneration Market:
Applications of Global Gene Therapy for Age-related Macular Degeneration Market:
Geographically, the detailed analysis of consumption, revenue, Gene Therapy for Age-related Macular Degeneration market share and growth rate, historic and forecast (2015-2026) of the following regions are covered-
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The growth of this market globally is subjected to different reasons, including consumer ace Gene Therapy for Age-related Macular Degeneration of a lot of Gene Therapy for Age-related Macular Degeneration products, inorganic company growth models, price volatility of raw materials, product innovation along with economic prospects in both producer and consumer countries.
The Report Highlights the Following Key Factors: Detailed information of the companys operations and business segments. List of key products, services, and brands of the company. List of major competitors to the company. Contact details of key locations and subsidiaries of the company. Progression of key events associated with the company. Analysts summarization of the companys business strategy. An extensive analysis of the companys strengths, weakness, opportunities, and threats.
Looking to expand beyond diabetes, Novo Nordisk enlists Evotec in CKD pact worth up to $179M per program – Endpoints News
Most of Novo Nordisks kidney work has come from diabetes, but eyeing an R&D expansion, the Danish giant is putting money down on a pact to target chronic kidney disease directly.
The new deal comes with an old partner: the German drug discovery company Evotec. Under the agreement, Evotec and Novo will collaborate on discovering and developing pre-clinical candidates across different modalities for CKD. Novo will then be in charge of clinical and commercial development for any promising approaches.
The companies were loose on specifics, but between upfront payments, research funding and milestones, Evotec can earn up to 150 million ($179 million) per project.
Building on adjacencies within cardio-renal and metabolism, this collaboration will allow us to strengthen our efforts within kidney disease, Novos cardio-renal and diabetes chief Karin Conde-Knape said in a statement, adding the work will be on the basis of human relevant biology.
Although the overwhelming majority of Novos roughly $20 billion in annual sales come from insulin and other diabetes and obesity drugs, the company has talked openly about its plans to expand into other areas over the next five years, including NASH, cardiovascular disease and CKD.
In 2018, Novo also licensed a CKD drug from San Diegos Epigen for up to $200 million, although they signaled they would also develop the drug in diabetes-related conditions.
Novo and Evotec have collaborated since 2017, when Novo took a 90.3 million stake in the biotech. Its proven to be a prudent investment, in euros and cents alone. Buoyed by a string of big-name partnerships and a few milestone payments, Evotecs revenues have nearly tripled over that time period, from around 150 million in 2017 to 446 million last year. And their stock has risen from 6.87 to 22.09 today.
In 2018, Novo turned to Evotec to boost their early-stage research in the Big Pharmas bread-and-butter areas: diabetes and obesity. That deal was built solely around small molecules, but the company has since invested heavily in both biologics and gene therapy, and the new collaboration is modality agnostic.
With its partners, Evotec has worked on a long list of disease areas including anti-infective research with Bayer and the Bill & Melinda Gates Foundation, neuroscience work with Bristol Myers Squibb, and immunology work with Galapagos and Pfizer and kidney disease has been no exception.
The company has worked with Bayer on drugs for diabetics with kidney disease since 2016, as part of an up to $300 million deal. In 2017, Evotec started working with Cambridge University, the University of Bristol, and the Mario Negri Institute in Italy on what they called the NEPLEX project. It involves using micro-fluidics and iPSC stem to create a nephron-on-a-chip that can be used to test preclinical drugs.
Perhaps, most notably, the company last year teamed with Vifor Biopharma to launch NephThera, a joint venture that uses data from the massive UK kidney biobank studyand Evotecs bioinformatics platform to find new drugs.
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Looking to expand beyond diabetes, Novo Nordisk enlists Evotec in CKD pact worth up to $179M per program - Endpoints News
Transfection Reagents And Equipment Market Share to Witness Steady Rise in the Coming Decade – Scientect
Transfection is a process that involves production of genetically modified cells with utilization of foreign nucleic acid (DNA and RNA). This technology helps the cells in mutation of cancer cells, protein metabolism by affecting the nuclear genes and regulation of gene therapy. Transfection is an integral equipment used in investigation studies for gene function and the modulation of gene expression. Thus, it contributes in the advancement of basic cellular research, drug discovery, and target validation. The transfection reagent and equipment market is driven by rising prevalence of infectious disease, utilization of biopharmaceuticals in the production of proteins, growing obese population, and increasing prevalence of cancer. Various government initiative accentuated the growth of transfection reagent and equipment market. However, high cost of transfection reagents and equipment, risk factors during insertion of the reagents and cytotoxic effect associated with transfection technology are the major factors restraining the transfection reagents and equipment market.
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The transfection reagent and equipment market can be segmented on the basis of various methods such as physical methods and biochemical methods. The biochemical method accounts for the largest share in the overall transfection market. The biochemical based method is further segmented as calcium phosphate, DEAE-dextran, lipid mediated transfection (Lipofection), catonic polymers, activated dendrimers and magnetic beads. The physical based method includes electroporation, biolistic technology, microinjection, laserfection and others (gene gun, sonoporation). Electroporation technique is likely to account for the largest share in the equipment based transfection. The transfection reagent market, by application is segmented into biomedical research, protein product, and therapeutic delivery. The biomedical research segment was observed as one of the largest segment of the transfection reagent market.
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Geographically, North America is the largest transfection reagents and equipment market in the world due to rising prevalence of various cancers (such as cervical cancer, breast cancer, colon cancer, and prostate cancer). Moreover, rising demand for proteomics and genomics technology and upfront initiatives taken by government related to preventive healthcare have supported the growth of transfection reagents and equipment market in this region. Europe was the second largest market due to rising trend of utilization of targeted drug delivery, nanomedicine in diagnostics, clinical trials and drug development studies drive the demand of transfection reagents and equipment market. Asia-Pacific is observed to be an emerging market in transfection reagents and equipment market and is still in the initial stage. One of the important factors driving the growth of transfection reagent and equipment in the Asia-Pacific market is outsourcing of clinical trials to Asian countries by majority of the drug development companies. Moreover, development of in transfection technology, rise in demand of protein therapeutics, developing healthcare infrastructure in emerging markets such as India and China, and increasing demand from applied markets. Latin American countries such as Brazil and Mexico are the regions that have significant potential for growth due to emerging medical infrastructure, high disposable income and rising prevalence of infectious diseases. Transfection equipment and reagents market is in introductory stage especially in Latin American and African countries.
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Some of the major players in the global transfection reagent and equipment market include
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Transfection Reagents And Equipment Market Share to Witness Steady Rise in the Coming Decade - Scientect
Viral Vectors and Plasmid DNA Marketplace 2020-2030: Breakdown of Shares for Gene & Cancer Therapies, Viral Infections, Immunotherapy, Formulation…
Dublin, Aug. 17, 2020 (GLOBE NEWSWIRE) -- The "Global Viral Vectors & Plasmid DNA Market 2020-2030: COVID-19 Implications and Growth" report has been added to ResearchAndMarkets.com's offering.
The global viral vectors & plasmid DNA market is expected to grow from $0.46 billion in 2019 to $0.60 billion in 2020, at a compound annual growth rate (CAGR) of 31.3%.
The growth is mainly due to the outbreak of COVID-19 and requires targeted delivery of the drug to treat affected patients. The number of cases has been increasing resulting in a surge in demand for viral vectors & plasmid DNA. The market is expected to reach $1.05 billion in 2023 at a CAGR of 20.4%.
The rise in the global incidence of cancer is driving the viral vector & plasmid DNA market. According to Cancer Research UK, in 2018, around 17 million new cases of cancer were recorded with deaths of around 9.6 million, and the estimated cancer incidence rate is 62% between 2018-2040 worldwide. The rise in the global incidence of cancer has boosted the demand for the viral vectors and plasmid DNA market.
The unaffordable cost of gene therapies is a major challenge in the viral vectors & plasmid DNA market. The prices of gene therapies range between $0.2 million to $2.1 million. For instance, Gilead company's Yescarta is a gene therapy that costs around $0.3 million and Bluebird Bio's Letiglobin is another gene therapy costing around $2.1 million. The high price is due to various factors like government regulations, production costs, and so on. Therefore, the unaffordable cost of gene therapies is limiting the viral vector & plasmid DNA market growth.
In January 2020, Cognate Bioservices Inc, a US-based company skilled in cell and cell-mediated gene therapy products, acquired Cobra Biologics, specialized in providing manufacturing services for plasmid DNA and viral vector, for an undisclosed amount. The acquisition is expected to create a platform for the life cycle management of cell and gene therapy products. Cobra biologics is well established in the development and manufacture of a variety of viral vectors and DNA. The combined Cognate and Cobra expertise is expected to push both the businesses to a better position to respond to current and future market demand.
North America was the largest region in the viral vectors and plasmid DNA market in 2019. Asia-Pacific is expected to be the fastest-growing region in the forecast period.
Major players in the viral vectors & plasmid DNA market are FUJIFILM Diosynth Biotechnologies, FinVector Vision Therapies, Lonza, Cobra Biologics and Pharmaceutical Services, Brammer Bio, Cell and Gene Therapy Catapult, VGXI, and MassBiologics.
Report Scope
The report covers market characteristics, size and growth, segmentation, regional and country breakdowns, competitive landscape, market shares, trends and strategies for this market. It traces the market's historic and forecast market growth by geography. It places the market within the context of the wider viral vectors & plasmid dna market, and compares it with other markets.
Key Topics Covered
1. Executive Summary
2. Viral Vectors & Plasmid DNA Market Characteristics
3. Viral Vectors & Plasmid DNA Market Size and Growth 3.1. Global Viral Vectors & Plasmid DNA Historic Market, 2015-2019, $ Billion 3.1.1. Drivers of the Market 3.1.2. Restraints on the Market 3.2. Global Viral Vectors & Plasmid DNA Forecast Market, 2019-2023F, 2025F, 2030F, $ Billion 3.2.1. Drivers of the Market 3.2.2. Restraints on the Market
4. Viral Vectors & Plasmid DNA Market Segmentation 4.1. Global Viral Vectors & Plasmid DNA Market, Segmentation by Product, Historic and Forecast, 2015-2019, 2023F, 2025F, 2030F, $ Billion
4.2. Global Viral Vectors & Plasmid DNA Market, Segmentation by Applications, Historic and Forecast, 2015-2019, 2023F, 2025F, 2030F, $ Billion
5. Viral Vectors & Plasmid DNA Market Regional and Country Analysis 5.1. Global Viral Vectors & Plasmid DNA Market, Split by Region, Historic and Forecast, 2015-2019, 2023F, 2025F, 2030F, $ Billion 5.2. Global Viral Vectors & Plasmid DNA Market, Split by Country, Historic and Forecast, 2015-2019, 2023F, 2025F, 2030F, $ Billion
Companies Mentioned
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Viral Vectors and Plasmid DNA Marketplace 2020-2030: Breakdown of Shares for Gene & Cancer Therapies, Viral Infections, Immunotherapy, Formulation...
FDA Approves Targeted Treatment for Rare Duchenne Muscular Dystrophy Mutation – FDA.gov
For Immediate Release: August 12, 2020
Today, the U.S. Food and Drug Administration granted accelerated approval to Viltepso (viltolarsen) injection for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping. This is the second FDA-approved targeted treatment for patients with this type of mutation. Approximately 8% of patients with DMD have a mutation that is amenable to exon 53 skipping.
The FDA is committed to fostering drug development for serious neurological disorders like Duchenne muscular dystrophy, said Billy Dunn, M.D., director of the Office of Neuroscience in the FDAs Center for Drug Evaluation and Research. Todays approval of Viltepso provides an important treatment option for Duchenne muscular dystrophy patients with this confirmed mutation.
DMD is a rare genetic disorder characterized by progressive muscle deterioration and weakness. It is the most common type of muscular dystrophy. DMD is caused by mutations in the DMD gene that results in an absence of dystrophin, a protein that helps keep muscle cells intact. The first symptoms are usually seen between three and five years of age and worsen over time. DMD occurs in approximately one out of every 3,600 male infants worldwide; in rare cases, it can affect females.
Viltepso was evaluated in two clinical studies with a total of 32 patients, all of whom were male and had genetically confirmed DMD. The increase in dystrophin production was established in one of those two studies, a study that included 16 DMD patients, with 8 patients receiving Viltepso at the recommended dose. In the study, dystrophin levels increased, on average, from 0.6% of normal at baseline to 5.9% of normal at week 25.
The FDA concluded that the applicants data demonstrated an increase in dystrophin production that is reasonably likely to predict clinical benefit in patients with DMD who have a confirmed mutation of the dystrophin gene amenable to exon 53 skipping. A clinical benefit of the drug has not been established. In making this decision, the FDA considered the potential risks associated with the drug, the life-threatening and debilitating nature of the disease, and the lack of available therapies.
As part of the accelerated approval process, the FDA is requiring the company to conduct a clinical trial to confirm the drugs clinical benefit. The ongoing study is designed to assess whether Viltepso improves the time to stand for DMD patients with this confirmed mutation. If the trial fails to verify clinical benefit, the FDA may initiate proceedings to withdraw approval of the drug.
The most common side effects observed in DMD patients (pooled from the two studies) treated with 80 mg/kg once a week were: Upper respiratory tract infection, injection site reaction, cough and fever.
Although kidney toxicity was not observed in the Viltepso clinical studies, the clinical experience with Viltepso is limited, and kidney toxicity, including potentially fatal glomerulonephritis, has been observed after administration of some antisense oligonucleotides. Kidney function should be monitored in patients taking Viltepso.
Viltepso was approved under the FDAs accelerated approval pathway, which provides for the approval of drugs that treat serious or life-threatening diseases and generally offer a meaningful advantage over existing treatments. Approval under this pathway can be based on adequate and well-controlled studies showing the drug has an effect on a surrogate endpoint that is reasonably likely to predict clinical benefit to patients (i.e., how patients feel or function or whether they survive). This pathway provides earlier patient access to promising new drugs while the company conducts clinical trials to verify the predicted clinical benefit.
The FDA granted this application Priority Review designation.
The FDA is granting the approval to NS Pharma, Inc.
The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nations food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.
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FDA Approves Targeted Treatment for Rare Duchenne Muscular Dystrophy Mutation - FDA.gov
Next-Generation Sequencing Market Size to Reach US$ 31,411.3 Mn, Introduction of Rapid Sequencing Technologies Drives the Market: Fortune Business…
Pune, Aug. 17, 2020 (GLOBE NEWSWIRE) -- The global next-generation sequencing market is forecast to exhibit a remarkable CAGR as the next-generation sequencing platform allows effective sequencing of millions of DNA molecules. Fortune Business Insights in a new report, titled Next-Generation Sequencing: Global Market Analysis, Insights and Forecast, 2019-2026 offers a detailed analysis of the competitive landscape and market dynamics.
The demand for cost-effective, accurate, and fast DNA sequencing technologies is increasing and this is further giving rise to the dominance of next-generation sequencing platforms. This will further give rise to advanced sequencing technologies for clinical purposes, which is anticipated to drive the market. Increasing advancements in bioinformatics are likely to increase the adoption of next-generation sequencing platforms to facilitate the diagnosis of rare diseases.
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As per the report, the global market is expected to rise at a ferocious CAGR of 22.2% between 2018 and 2026. In 2018, the market was worth US$ 6,335.2 Mn and the market will be a howling success as it is projected to reach US$ 31,411.3 Mn by 2026. The demand for next-generation sequencing methodologies is increasing rapidly across several applications such as diagnostics, clinical research among others.
The global economy is in absolute turmoil because of the COVID-19 pandemic outbreak. Some industries remain largely unaffected by the outbreak, some are thriving, but most are in doldrums. Although the healthcare industry is flourishing, certain markets within the industry are experiencing staggered growth. Wading through these troubled times is a difficult task and Fortune Business Insights aims at equipping your business with the most comprehensive market insights, collated and analyzed by our expert and experienced research team.
Click here to get the short-term and long-term impact of COVID-19 on this market.
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Demand for Next-Generation Sequencing Services to Rise at a High Pace
Next-generation sequencing products encompass instruments, consumables, and software. According to the findings from the report, the next-generation sequencing product segment is anticipated to lead the market over the forecast period. This is ascribable to some primary factors such as new product launches, high usage of consumables, and easy availability.
Apart from next-generation sequencing products, next-generation sequencing services are expected to grow at a higher CAGR during the forecast period. The need for novel platforms regarding advancements in bioinformatics and fast DNA data interpretation are paving way for start-ups, especially for next-generation sequencing services. With the introduction of next-generation sequencing, the cost of sequencing has reduced over the years.
This is positively impacting the growth of this segment. Furthermore, the adoption of next-generation sequencing-based testing is increasing across the world. This, coupled with attractive healthcare reimbursement policies, is expected to drive the segments growth in the projected period.
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North America Leads the Way Owing to Active Government Support
Among regions, North America is anticipated to lead the global next-generation sequencing market through the forecast duration. Rapid developments in precision medicine, next-generation DNA sequencing, and diagnostics are creating growth opportunities for the market. The market is likely to expand as companies in the biotechnology sector are continuously involving in research and development (R&D) activities, which fuels the market demand. Adapative Technologies developed a next-generation sequencing diagnostic test called ClonoSEQ for patients suffering from acute lymphoblastic leukemia or ALL and minimal residual disease or MRD. The test received FDA approval in September 2018, which in turn, contributes to the growth of the market in North America.
Increasing investments in precision medicine and a growing number of strategic collaborations are driving the market in Europe. Driven by these factors, the market in this region is expected to expand in the forecast period. Apart from these two regions, Asia Pacific is expected to expand at a remarkable growth on account of rising geriatric population and growing awareness about reproductive health.
Saphetor SA and Swift Biosciences Inc.s Partnership Aims to Target Next-Generation Sequencing Panels
Strategic partnerships and new product launches are increasingly adopted by companies as these help them to maintain their position in the market. Some of the industry developments are mentioned below:
Companies such as Illumina, Inc., Thermo Fisher Scientific Inc., and F. Hoffmann-La Roche Ltd are likely to lead in the global next-generation sequencing market. This is mainly on account of their strong geographical presence and diversified product portfolio. Moreover, Illumina was leading in the global market owing to its rising product sales. New product launches also helped the company to strengthen its market position.
Some of the other players functioning in the global market are Agilent Technologies, Inc., BGI, PerkinElmer Inc., QIAGEN, Eurofins Scientific, PierianDx, Macrogen, Inc. among others.
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Next-Generation Sequencing Market Segmentation:
By Type
Products
Instruments & Software
Consumables
Services
By Application
Diagnostics
Research
Others
By End User
Research Institutes
Healthcare Facilities & Diagnostic Centers
Pharmaceutical & Biotechnological Companies
Contract Research Organization (CROs)
By Geography
North America (the USA and Canada)
Europe (UK, Germany, France, Italy, Spain, and Rest of Europe)
Asia Pacific (Japan, China, India, Australia, Southeast Asia, and Rest of Asia Pacific)
Latin America (Brazil, Mexico, and Rest of Latin America)
Middle East & Africa (South Africa, GCC, and Rest of the Middle East & Africa)
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Next-Generation Sequencing Market Size to Reach US$ 31,411.3 Mn, Introduction of Rapid Sequencing Technologies Drives the Market: Fortune Business...
Gyroscope Therapeutics Announces Initiation of Phase II Programme Evaluating its Investigational Gene Therapy, GT005, for Dry Age-Related Macular…
Gyroscope Therapeutics Limited, a clinical-stage retinal gene therapy company, today announced the initiation of its Phase II programme evaluating its investigational gene therapy, GT005, for the treatment of geographic atrophy (GA) secondary to dry age-related macular degeneration (AMD). Dry AMD is a leading cause of permanent vision loss in people over the age of 50. GT005 is a one-time AAV-based gene therapy that is delivered under the retina. The goal of the Phase II clinical trial programme is to determine if GT005 has the potential to slow the progression of GA.
Gyroscope plans to conduct two Phase II trials evaluating GT005 in people with GA. The first, called EXPLORE, is enrolling people who have a mutation in their Complement Factor I (CFI) gene [NCT04437368]. The first patient to receive GT005 in EXPLORE was enrolled and dosed by Dr. Arshad M. Khanani at Sierra Eye Associates in Reno, Nev., USA.
Geographic atrophy is a devastating diagnosis, as there are no approved treatments for this gradual and irreversible loss of vision, said Arshad M. Khanani, M.D., M.A., Director of Clinical Research at Sierra Eye Associates and Clinical Associate Professor at the University of Nevada, Reno School of Medicine, and an investigator in the EXPLORE trial. We are excited to participate in this trial evaluating GT005 for the potential to slow progression of geographic atrophy. We believe one-time gene therapies could be a major advancement in the field of retinal disease.
GT005 is designed to restore balance to an overactive complement system, a part of the immune system, by increasing production of the CFI protein. An overactive complement system has been implicated in the development of AMD. The CFI protein regulates the activity of the complement system. It is believed that increasing CFI production will dampen the systems overactivity and reduce inflammation, with the goal of preserving a persons eyesight.
Research has found that approximately 3% of people with dry AMD have certain CFI mutations that correlate with low CFI levels in the blood and a higher risk of developing AMD.1 Gyroscope estimates that more than 100,000 people with GA in the United States and EU5 European countries may have these mutations.1,2,3,4 The EXPLORE trial will evaluate GT005 in this group of people with mutations in their CFI gene.
Research has also shown that a small supplementation of CFI can normalise complement activity in the blood,5 suggesting GT005 may also be applicable for a broader group of people with GA. It is estimated that approximately one million people in the United States alone have GA.4 Gyroscope therefore plans to initiate a second Phase II trial in 2020 that will evaluate GT005 in a broader GA population.
We are excited about the potential of GT005 for people with dry AMD. Research suggests GT005 may be best suited for people with certain mutations in their CFI gene. However, evidence also suggests it may have potential for a broader population of people with geographic atrophy, said Nadia Waheed, M.D., MPH, Chief Medical Officer of Gyroscope. We have designed our clinical programme to evaluate these groups in two distinct Phase II trials, with the goal of determining which patients GT005 may be most appropriate for and to further our understanding of the role of the complement system in AMD.
About the EXPLORE Trial
EXPLORE is a Phase II, multicentre, randomised trial evaluating the safety and efficacy of GT005 administered as a single subretinal injection.
EXPLORE is enrolling people who are aged 55 or older and have a clinical diagnosis of GA secondary to dry AMD and who have a mutation of the CFI gene. People being screened for the trial will be genotyped for the mutations. Trial participants will be randomised to one of three treatment arms: GT005 dose 1, GT005 dose 2 or a control arm. Participants in the control arm will receive current standard of care. The primary endpoint of EXPLORE is progression of GA over 48 weeks. The study will also evaluate GT005 for various safety and tolerability measures. Gyroscope plans to enroll approximately 75 patients at approximately 40 centres based in the United States, United Kingdom, Europe and Australia.
Gyroscope will announce details about the second Phase II trial of GT005 at a later date.
About Age-Related Macular Degeneration (AMD) and Geographic Atrophy (GA)
AMD is a leading cause of blindness affecting an estimated 196 million people globally.6 AMD typically affects people aged 50 and older, and causes a gradual and permanent loss of central vision that worsens over time.7 There are no approved treatments for the dry form of AMD, which is the most common, impacting approximately 90% of people with the disease.8 As dry AMD advances it leads to GA, an irreversible degeneration of retinal cells. This vision loss can be devastating, severely impacting a persons daily life as they lose the ability to drive, read and even see the faces of loved ones.
About Gyroscope Therapeutics: Vision for Life
Gyroscope Therapeutics is a clinical-stage retinal gene therapy company developing and delivering gene therapy beyond rare disease to treat a leading cause of blindness, dry AMD. Our lead investigational gene therapy, GT005, is a one-time therapy delivered under the retina. GT005 is designed to restore balance to an overactive complement system by increasing production of the Complement Factor I protein. GT005 is currently being evaluated in a Phase I/II clinical trial called FOCUS and a Phase II clinical trial called EXPLORE.
Syncona Ltd, our lead investor, helped us create the only retinal gene therapy company to combine discovery, research, drug development, a manufacturing platform and surgical delivery capabilities. Headquartered in London with locations in Philadelphia and San Francisco, our mission is to preserve sight and fight the devastating impact of blindness. For more information visit: http://www.gyroscopetx.com and follow us on Twitter (@GyroscopeTx) and on LinkedIn.
References
1 Kavanagh D, Yu Y, Schramm EC, et al. Rare genetic variants in the CFI gene are associated with advanced age-related macular degeneration and commonly result in reduced serum factor I levels. Hum Mol Genet. 2015;24(13):3861-3870.
2 Data on File.
3 Friedman DS, OColmain BJ, Muoz B, et al. Prevalence of age-related macular degeneration in the United States [published correction appears in Arch Ophthalmol. 2011 Sep;129(9):1188]. Arch Ophthalmol. 2004;122(4):564-572.
4 Rudnicka AR, Kapetanakis VV et al. Incidence of late-stage age-related macular degeneration in American whites: systematic review and meta-analysis. Am J Ophthalmol 2015;160:85-93.
5 Lachmann PJ, Lay E, Seilly DJ, Buchberger A, Schwaeble W, Khadake J. Further studies of the down-regulation by Factor I of the C3b feedback cycle using endotoxin as a soluble activator and red cells as a source of CR1 on sera of different complotype. Clin Exp Immunol. 2016;183(1):150-156.
6 Wong WL, Su X, Li X, et al. Global prevalence of age-related macular degeneration and disease burden projection for 2020 and 2040: a systematic review and meta-analysis. Lancet Glob Health 2014;2:e106116.
7 National Eye Institute. Age-Related Macular Degeneration. https://www.nei.nih.gov/learn-about-eye-health/eye-conditions-and-diseases/age-related-macular-degeneration. Accessed July 16, 2020.
8 Centers for Disease Control and Prevention. Age-Related Macular Degeneration. https://www.cdc.gov/visionhealth/basics/ced/. Page last reviewed June 3, 2020. Accessed August 4, 2020.
View source version on businesswire.com: https://www.businesswire.com/news/home/20200812005751/en/
Charlotte Arnold
VP, Corporate Affairs
Gyroscope Therapeutics
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Gyroscope Therapeutics Announces Initiation of Phase II Programme Evaluating its Investigational Gene Therapy, GT005, for Dry Age-Related Macular...
Global Biologics Market and Curative Oncology and Rare Disease Therapies to 2024 – COVID-19 Impact Analysis – ResearchAndMarkets.com – Business Wire
DUBLIN--(BUSINESS WIRE)--The "Curative Oncology and Rare Disease Therapies Transforming the Global Biologics Market, 2020-2024" report has been added to ResearchAndMarkets.com's offering.
With this research service, the publisher's Transformational Health team provides critical insights into the global biologics industry highlighting the growth opportunities, market revenue segmentation, and technology trends influencing its growth. The global biologics market covered in this study includes 4 major segments - antibody therapies, recombinant proteins, vaccines, and regenerative medicine. The forecast for the global biologics market until 2024 reveals an interesting trend and paradigm shifts that are set to take place in the industry. Importantly, the study also identifies actionable growth opportunities for industry participants to profit upon.
This research service identifies the largest revenue-generating segments and key therapeutic areas for the global biologics industry. Accelerated regulatory approvals for biologic products and the emergence of next-generation technologies are highlighted, in addition to their influence on market trends during the forecast period. The study highlights the dynamics of the biologics industry and the sub-segments of the biologics market.
Growth opportunities in antibody-drug conjugate (ADC), RNA therapy, and gene therapy platforms are also explored. Further, the study discusses the impact (such as the accelerated development of vaccines) of the COVID-19 pandemic on the biologics industry and the market. Current challenges facing the biologics industry such as the sluggish growth rate of vaccines, and recombinant proteins are discussed.
Most importantly, this research service discusses possible future market trends such as the accelerated development of mRNA-based vaccines and the increasing importance of speciality bio-CDMOs for the development and commercialization of regenerative medicine. Based on the market trends and revenue forecasts, the group of analysts highlight key growth opportunities in ADC, RNA, and gene therapy platforms through successful drug launches such as Belantamab Malfodotin, Inclisiran, and Zolgensma.
Research Highlights
Highlights of the report include:
Key Issues Addressed
Key Topics Covered:
1. Executive Dashboard
2. Market Overview - Biologics
3. Market Dynamics
4. COVID-19 Impact Analysis of the Biologics Market
5. Revenue Forecast - Total Biologics Market
6. Antibody Therapies Segment Analysis
7. Recombinant Proteins Segment Analysis
8. Vaccines Segment Analysis
9. Cell & Gene Therapy Segment Analysis
10. Companies to Action - Pipeline Analysis
11. Growth Opportunities
12. The Last Word
13. Appendix
For more information about this report visit https://www.researchandmarkets.com/r/g4tkkr
Gene Therapy Market Applications, Types and Future Outlook Report 2020-2025 – CueReport
The Gene Therapy market report covers the overview of the market and presents the information on business development, market size, and share scenario. The report also emphasizes on the growth prospects of the global Gene Therapy market for the period 2020-2025. The report covers market characteristics for each segmentation across the key regions and country and traces the historic and forecast analysis of the industry. The report covers key vendors their revenue, breakdown by regions, and product demand. Other key details include volume, installed capacity and value chain.
Executive Summary:
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The research report on Gene Therapy market delivers vital data regarding this industry landscape while elaborating on the major growth drivers, limitations & challenges, opportunities, and future prospects impacting the business outlook.
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According to the study, the Gene Therapy market is expected to register a CAGR of XX% during the study period.
Crucial insights pertaining to the regional scope, competitive scenario and other factors influencing the growth pattern of each market segment are encompassed in the document. In addition, the report gauges the impact of COVID-19 outbreak on the revenue graph of this market.
Market Rundown:
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AgeX Therapeutics Reports Second Quarter 2020 Financial Results and Provides Business Update – Business Wire
ALAMEDA, Calif.--(BUSINESS WIRE)--AgeX Therapeutics, Inc. (AgeX; NYSE American: AGE), a biotechnology company developing therapeutics for human aging and regeneration, reported financial and operating results for the second quarter ended June 30, 2020.
AgeX made strides with respect to its newly established licensing and collaboration model, which aims to embed its technology platforms across the cell therapy industry. AgeX has entered into a research license for the use of its immunotolerance UniverCyteTM technology by Sernova Corp. (Sernova), a publicly-listed Canadian regenerative medicine therapeutics company. It also entered a Manufacturing, Marketing, and Distribution Agreement with Pluristyx, Inc. (Pluristyx), an advanced therapy tools and services company serving customers in the fields of regenerative medicine and cellular and gene therapies. In addition, AgeX signed a letter of intent with ImStem Biotechnology (ImStem), for ImStem to utilize AgeXs ESI-brand pluripotent stem cells to derive a cell therapy product for potential use in the treatment of COVID-19 and as well acute respiratory distress syndrome (ARDS) from non-COVID-19 causes.
We are working diligently to position our subsidiary Reverse Bioengineering to optimize the potential of induced tissue regeneration (iTR) technology, said Michael West, CEO of AgeX. We believe this technology offers a powerful new modality to treat age-related degenerative diseases by reversing developmental aging in a tissue, thereby unlocking an innate capacity of tissues to regenerate scarlessly.
AgeX completed its restructuring to streamline its operations to allow efficient usage of capital in the current pandemic environment as well to meet near-term strategic company priorities of deriving value and generating preclinical and ultimately clinical data from our technology platforms through external licensing and collaboration agreements. In the longer-term, AgeX remains committed to in-house product development of AgeX-BAT1 and AgeX-VASC1. AgeX is considering options to bring capital into the company.
AgeX has made excellent progress in terms of its collaboration and licensing model, closing five deals since the beginning of the year so far, said Greg Bailey M.D., Chairman of AgeX. The deals have spanned all three of our technology platforms of UniverCyte for the generation of universal cells, PureStem for the derivation and manufacturing of therapeutic cells, and AgeX ESI pluripotent stem cells to act as a source material for cellular therapies. All these deals show the value industry and academia see in our offerings.
Q2 Highlights
Liquidity and Capital Resources
AgeX is in need of additional capital to finance its operations. On March 30, 2020, AgeX entered into a Secured Convertible Facility Agreement (the New Loan Agreement) with Juvenescence Limited pursuant to which AgeX may borrow funds from time to time. As of August 14, 2020, AgeX has borrowed $3.5 million and may draw additional funds from time to time subject to Juvenescences discretion, prior to the contractual repayment date on March 30, 2023. AgeX may not draw down more than $1.0 million in any single draw. More information about the New Loan Agreement can be found in AgeXs Annual Report on Form 10-K and Quarterly Reports on Form 10-Q for the periods ended March 31, 2020 and June 30, 2020 filed with the Securities and Exchange Commission on March 30, 2020, May 14, 2020, and August 14, 2020 respectively.
On April 13, 2020, AgeX obtained a loan in the amount of $432,952 from Axos Bank under the Paycheck Protection Program (the PPP Loan). The PPP Loan bears interest at a rate of 1% per annum. No payments will be due on the PPP Loan during a six month deferral period commencing on the date of the promissory note. Commencing one month after the expiration of the deferral period, and continuing on the same day of each month thereafter until the maturity date of the PPP Loan, monthly payments of principal and interest will be due, in an amount required to fully amortize the principal amount outstanding on the PPP Loan by the maturity date. The maturity date is April 13, 2022. The principal amount of the PPP Loan is subject to forgiveness under the Paycheck Protection Program (PPP) to the extent that PPP Loan proceeds are used to pay expense permitted by the PPP, including payroll, rent, and utilities (collectively, Qualifying Expenses), during the time frame permitted by the PPP. AgeX believes that it has used the PPP Loan amount for Qualifying Expenses. However, no assurance is provided that AgeX will obtain forgiveness of the PPP Loan in whole or in part.
Staff Reductions
In May 2020, AgeX laid off 11 research and development personnel and consequently paid approximately $105,000 in accrued payroll and unused paid time off and other benefits and recognized approximately $194,800 in restructuring charges in connection with the reduction in staffing, consisting of contractual severance and other employee termination benefits, substantially all of which have been settled in cash. The staff reductions followed AgeXs strategic review of its operations, giving consideration to the status of its product development programs, human resources, capital needs and resources, and current conditions in the capital markets resulting from the COVID-19 pandemic.
Going Concern Considerations
As required under Accounting Standards Update 2014-15, Presentation of Financial Statements-Going Concern (ASC 205-40), AgeX evaluates whether conditions and/or events raise substantial doubt about its ability to meet its future financial obligations as they become due within one year after the date its financial statements are issued. Based on AgeXs most recent projected cash flows, and considering that loans from Juvenescence under the New Loan Agreement will be subject to Juvenescences discretion, AgeX believes that its cash and cash equivalents, the remaining $5.5 million available under the New Loan Agreement and reduction in staff in May 2020 would not be sufficient to satisfy its anticipated operating and other funding requirements for the twelve months following the filing of AgeXs Quarterly Report on Form 10-Q for the three and six months ended June 30, 2020. These factors raise substantial doubt regarding the ability of AgeX to continue as a going concern.
Second Quarter 2020 Operating Results
Revenues: Total revenues for the second quarter of 2020 were $414,000 as compared with $380,000 for the second quarter of 2019. AgeX revenues are primarily generated from subscription and advertising revenues from the GeneCards online database through its subsidiary LifeMap Sciences, Inc. Revenues in 2020 also included approximately $36,000 of allowable expenses under its research grant from the NIH as compared with $47,000 in the same period in 2019.
Operating expenses: Operating expenses for the three months ended June 30, 2020 were $3.0 million as compared to $3.8 million for the same period in 2019. On an as-adjusted basis, operating expenses for the three months ended June 30, 2020 were $2.5 million as compared to $3.1 million for the same period in 2019.
The reconciliation between GAAP and non-GAAP operating expenses is provided in the financial tables included with this earnings release.
Research and development expenses decreased by $0.3 million to $1.4 million during the three months ended June 30, 2020 from $1.7 million during the same period in 2019. The decrease was primarily attributable to the layoff of 11 research and development personnel in May 2020 and decrease in shared services from Lineage Cell Therapeutics, Inc. (Lineage) with the termination of our Shared Facilities and Services Agreement on September 30, 2019.
General and administrative expenses decreased by $0.4 million to $1.7 million during the three months ended June 30, 2020 from $2.1 million during the same period in 2019 despite an increase in head count with the employment of AgeXs own finance team since October 1, 2019. These increases were offset by a decrease in travel and related expenses with the shelter in place mandates since March 15, 2020 resulting from the COVID-19 pandemic, and the elimination of shared facilities and services fees from Lineage following the termination of the Shared Facilities and Services Agreement on September 30, 2019.
About AgeX Therapeutics
AgeX Therapeutics, Inc. (NYSE American: AGE) is focused on developing and commercializing innovative therapeutics for human aging. Its PureStem and UniverCyte manufacturing and immunotolerance technologies are designed to work together to generate highly defined, universal, allogeneic, off-the-shelf pluripotent stem cell-derived young cells of any type for application in a variety of diseases with a high unmet medical need. AgeX has two preclinical cell therapy programs: AGEX-VASC1 (vascular progenitor cells) for tissue ischemia and AGEX-BAT1 (brown fat cells) for Type II diabetes. AgeXs revolutionary longevity platform induced Tissue Regeneration (iTR) aims to unlock cellular immortality and regenerative capacity to reverse age-related changes within tissues. AGEX-iTR1547 is an iTR-based formulation in preclinical development. HyStem is AgeXs delivery technology to stably engraft PureStem cell therapies in the body. AgeXs core product pipeline is intended to extend human healthspan. AgeX is seeking opportunities to establish licensing and collaboration arrangements around its broad IP estate and proprietary technology platforms and therapy product candidates.
For more information, please visit http://www.agexinc.com or connect with the company on Twitter, LinkedIn, Facebook, and YouTube.
Forward-Looking Statements
Certain statements contained in this release are forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Any statements that are not historical fact including, but not limited to statements that contain words such as will, believes, plans, anticipates, expects, estimates should also be considered forward-looking statements. Forward-looking statements involve risks and uncertainties. Actual results may differ materially from the results anticipated in these forward-looking statements and as such should be evaluated together with the many uncertainties that affect the business of AgeX Therapeutics, Inc. and its subsidiaries, particularly those mentioned in the cautionary statements found in more detail in the Risk Factors section of AgeXs most recent Annual Report on Form 10-K and Quarterly Reports on Form 10-Q filed with the Securities and Exchange Commissions (copies of which may be obtained at http://www.sec.gov). Subsequent events and developments may cause these forward-looking statements to change. In addition, with respect to AgeXs Manufacturing, Marketing and Distribution Agreement with Pluristyx there is no assurance that (i) Pluristyx will generate significant sales of AgeX ESI hESC lines, or (ii) AgeX will derive significant revenue from sales of ESI hESC lines by Pluristyx. AgeX specifically disclaims any obligation or intention to update or revise these forward-looking statements as a result of changed events or circumstances that occur after the date of this release, except as required by applicable law.
AGEX THERAPEUTICS, INC. AND SUBSIDIARIES
CONDENSED CONSOLIDATED BALANCE SHEETS
(IN THOUSANDS, EXCEPT PAR VALUE AMOUNTS)
June 30,2020
December 31,2019
(Unaudited)
ASSETS
CURRENT ASSETS
Cash and cash equivalents
$
1,033
$
2,352
Accounts and grants receivable, net
235
363
Prepaid expenses and other current assets
836
1,339
Total current assets
2,104
4,054
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AgeX Therapeutics Reports Second Quarter 2020 Financial Results and Provides Business Update - Business Wire
Viral Vector Purification / Virus Purification Products Market (kit, prepacked column, resin, cassette, filter plate, capsule, reagent, and others),…
NEW YORK, Aug. 12, 2020 /PRNewswire/ --
INTRODUCTION Since the approval and launch of cell and gene therapy products, such as Zolgensma (2019), ZYNTEGLO (2019), Luxturna (2017), KYMRIAH (2017) and YESCARTA (2017), there has been a significant increase in demand for viral vectors. Experts believe that the number of such therapies is likely to double over the next couple of years. It is also worth mentioning that this particular field of medical research received close to USD 10 billion in funding in 2019. Currently, over 1,000 cell and gene therapy-related clinical trials are being conducted, worldwide. Genetic manipulation is a basic requirement of cell and gene therapy development, and, as a result, gene delivery vectors are considered to be of utmost importance in this domain. So far, viral vectors (such as those based on adeno-associated viruses (AAV), adenoviruses, lentivirus, retroviruses and other viruses) have been shown to be the most versatile gene delivery tools available, having demonstrated high transfection efficiencies in both preclinical and clinical settings. Further, taking into account the therapeutic efficacy and low side effects profiles of cell and gene therapies, the demand for such interventions is anticipated to grow at a rapid pace, resulting in a proportional increase in need for appropriate vector systems, as well. However, viral vector development and manufacturing is a complex and cost intensive process. One of the primary concerns associated with viral vector production is related to yield; in fact, a singular batch run is estimated to incur losses of up to 70% during the purification step alone.
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A number of techniques are presently used for viral vector purification. Over the years, size-based viral purification strategies, such as density-gradient ultracentrifugation, ultrafiltration, precipitation and size-exclusion chromatography (SEC), have become part of the accepted industry standard. However, recently, stakeholders have begun relying more on affinity chromatography-based purification regimens, given its robustness and high selectivity. Presently, several companies claim to offer a diverse range of virus purification solutions, including, filter plates, prepacked chromatography columns and resins, and consolidated kits, for viral vector (virus) purification. As indicated earlier, downstream processing of viral vector products is challenging. Existing separation and purification techniques are not efficient when it comes to purifying viral vectors at large scales. Moreover, these techniques are often unable to separate complete viruses from empty capsids, thereby, compromising product recovery. Therefore, it has become essential for stakeholders to develop innovative ways to optimize the purification process, in order to further improve virus recovery and facilitate effective removal of contaminants / impurities. Currently, the viral vector purification products market is driven by abovementioned surge in demand for viral vectors. In this context, it is worth mentioning that there are several virus based vaccines under development against the novel SARS-CoV-2 coronavirus strain. In fact, according to the WHO, there are over 20 viral vaccines currently being investigated against the novel coronavirus. Developing the means to better purify viruses is, therefore, likely to significantly impact viable vaccine yield. The current crisis is definitely going to have a strong impact on the virus purification products market.
SCOPE OF THE REPORT The 'Viral Vector Purification / Virus Purification Products Market (kit, prepacked column, resin, cassette, filter plate, capsule, reagent and others), 2020-2030' report features an extensive study of various products available for viral vector purification, in addition to the current market landscape and future potential of product developers.
Amongst other elements, the report features: - An overview of the current market landscape of companies providing products for purification of viruses / viral vectors, using different techniques, such as chromatography, centrifugation and filtration. It features information on the type of product (kit, prepacked column, resin, cassette, filter plate, capsule and reagent), type of purification technique (chromatography, centrifugation and filtration), scale of operation (lab-scale, clinical and commercial), type of viral vector (AAV, adenovirus, lentivirus, retrovirus and others) and details on other physical and operational parameters of the product (such as matrix, pore size, volume of bed, flow rate, operating pressure, working temperature, pH, filtration area and process time). In addition, the chapter includes information on the purification product developers, including details on the year of establishment, company size and location of headquarters. - Elaborate profiles of key players, including an overview of the company, product portfolio (viral vector purification products), recent developments and an informed future outlook. - An analysis evaluating the potential strategic partners (comprising of viral vector-based therapy developers and viral vector manufacturers) for viral vector purification product developers, based on several parameters, such as type of viral vector, developer strength, operational strength, therapeutic area, strength of clinical pipeline and strength of preclinical pipeline. - A clinical trial analysis of completed, ongoing and planned studies of various viral vector-based cell therapies, gene therapies and vaccines (approved / under development). It features detailed analyses of clinical studies of different viral-vector based therapies on the basis of their registration year, phase of development, trial status, type of therapy, therapeutic area, type of sponsor / collaborator, geographical location, number of patients enrolled and key players. - An informed estimate of the annual clinical and commercial demand (in terms of number of patients) for viral vectors, taking into account the marketed gene-based therapies and clinical studies evaluating vector-based therapies; the analysis also takes into consideration various relevant parameters, such as target patient population, dosing frequency and dose strength. Further, the demand has been segregated on the basis of type of viral vector, type of therapy, therapeutic are and geographical location. - A case study on tangential flow filtration (TFF), highlighting the role, advantages and disadvantages of the technique for purification of viral vectors; the chapter features details of products used for TFF, including product type, scale of operation, membrane material, flow rate and filtration area. - A case study featuring the viral vector manufacturers providing commercial scale production, highlighting details on their year of establishment, company size, type of viral vector (AAV, adenovirus, lentivirus, retrovirus and others), purpose of production (in-house and contract-basis), and location of headquarters and manufacturing facilities.
One of the key objectives of the report was to estimate the existing market size and identify potential growth opportunities for viral vector purification product developers, over the coming decade. Based on various parameters, such as the likely increase in number of clinical studies related to viral vector-based therapies, anticipated growth in target patient population, existing price variations across different purification techniques, and the success of cell and gene therapy products (considering both approved and late-stage clinical candidates), we have provided an informed estimate of the likely evolution of the market in the short to mid-term and long term, for the period 2020-2030. In order to provide a detailed future outlook, our projections have been segmented on the basis of [A] type of viral vector (AAV, adenovirus, lentivirus, retrovirus and others), [B] type of purification technique (chromatography, centrifugation and filtration), [C] type of therapy (gene therapy, cell therapy and viral vaccines) [D] therapeutic area (oncological disorders, cardiovascular disorders, ophthalmic disorders, metabolic disorders, inflammation & immunological diseases and others), [E] scale of operation (preclinical / clinical and commercial) and [F] key geographical regions (North America, Europe, Asia Pacific and rest of the world).
The research, analysis and insights presented in this report are backed by a deep understanding of key insights gathered from both secondary and primary research. The opinions and insights presented in the report were influenced by discussions held with several players in this industry. The study includes detailed transcripts of discussions held with the following individuals: - Haifeng Chen (Chief Executive Officer, Virovek) - Jeffrey Hung (Chief Commercial Officer, Vigene Biosciences) - Kai Lipinski (Chief Scientific Officer, Vibalogics)
All actual figures have been sourced and analyzed from publicly available information forums and primary research discussions. Financial figures mentioned in this report are in USD, unless otherwise specified.
RESEARCH METHODOLOGY The data presented in this report has been gathered via secondary and primary research. For all our projects, we conduct interviews with experts in the area (academia, industry, medical practice and other associations) to solicit their opinions on emerging trends in the market. This is primarily useful for us to draw out our own opinion on how the market will evolve across different regions and technology segments. Where possible, the available data has been checked for accuracy from multiple sources of information. The secondary sources of information include - Annual reports - Investor presentations - SEC filings - Industry databases - News releases from company websites - Government policy documents - Industry analysts' views While the focus has been on forecasting the market till 2030, the report also provides our independent view on various non-commercial trends emerging in the industry. This opinion is solely based on our knowledge, research and understanding of the relevant market gathered from various secondary and primary sources of information.
CHAPTER OUTLINES Chapter 2 provides an executive summary of the insights captured in our research. It offers a high-level view on the likely evolution of the viral vector purification product in the short to mid-term, and long term.
Chapter 3 provides a general overview of various types of viral and non-viral vectors. It includes a detailed discussion on the methods of gene transfer, type of viral vectors and applications of viral vectors in various advanced therapies. It provides information on the different stages of the viral vector purification process, type of purification techniques used and the key challenges associated with the purification of viral vectors. Further, it includes a review of the latest trends and innovations in the vector purification market.
Chapter 4 is an overview of the various types of products used for purification techniques based on principles of chromatography, centrifugation and filtration. It highlights product specific information, such as type of product (kit, prepacked column, resin, cassette, filter plate, capsule and reagent), type of purification technique (chromatography, centrifugation and filtration), scale of operation (lab-scale, clinical and commercial), type of viral vector (adenovirus, AAV, lentivirus, retrovirus and others), type of chromatographic technique (affinity chromatography, ion-exchange chromatography, size-exclusion chromatography and hydrophilic interaction chromatography), pack size (wherever specified) and price (wherever specified). In addition, the chapter provides details on certain additional parameters (based on the purification technique, wherever applicable), such as type of matrix, binding capacity, particle size, volume of bed, flow rate, operating pressure, working temperature, process time and pH. The chapter also provides information on the purification product developers, including year of establishment, company size and location of headquarters.
Chapter 5 includes detailed profiles of players offering products for viral vector purification. Each profile features an overview of the company, , information on its product portfolio, recent developments and an informed future outlook. Chapter 6 highlights potential strategic partners (comprising of viral vector-based therapy developers and viral vector manufacturers) for viral vector purification product developers, based on multiple parameters, such as type of viral vector, developer strength, operational strength, therapeutic areas, strength of clinical pipeline and strength of preclinical pipeline. The analysis aims to provide the necessary inputs to the product developers, enabling them to make the right decisions to collaborate with industry stakeholders with relatively more initiatives in the domain.
Chapter 7 features a geographical clinical trial analysis of completed, ongoing and planned studies of viral vector-based cell therapies, gene therapies and vaccines (approved / under development). The analysis provides details related to the different types of viral vectors investigated / being investigated across various geographies, based on the number of clinical trials, registration year, phase of development, trial status, type of therapy, therapeutic area, type of sponsor / collaborator, number of patients enrolled, key players and type of viral vector.
Chapter 8 features an informed estimate of the annual demand for viral vectors, taking into account the viral vector based marketed therapies and clinical studies. For the purpose of estimating the current clinical demand, we considered the active clinical studies of different types of vector-based therapies that have been registered till date. The data was analyzed on the basis of various parameters, such as number of annual clinical doses, trial location, and the enrolled patient population across different geographies. Further, in order to estimate the commercial demand, we considered the marketed vector-based therapies, based on various parameters, such as target patient population, dosing frequency and dose strength. The chapter features analyses demand on the basis of type of viral vector, type of therapy, therapeutic are and geographical location.
Chapter 9 is a detailed case study which provides an overview of the tangential flow filtration, highlighting its role, advantages and disadvantages. It features key details of the products used for tangential flow filtration, such as type of product, scale of operation, membrane material, flow rate and filtration area.
Chapter 10 is a detailed case study which provides an overview of the viral vector manufacturers. It features an in-depth analysis of the commercial scale viral vector manufacturers, highlighting details on their year of establishment, company size, types of vectors (AAV, adenovirus, lentivirus, retrovirus and others), purpose of production (in-house and contract-basis), headquarters and manufacturing facilities.
Chapter 11 presents an insightful market forecast analysis, highlighting the future potential of the virus purification products market till the year 2030. We have segregated the opportunity of the market on the basis of [A] type of viral vector (AAV, adenovirus, lentivirus, retrovirus and others), [B] type of purification technique (chromatography, centrifugation and filtration), [C] type of therapy (gene therapy, cell therapy and viral vaccines) [D] therapeutic area (oncological disorders, cardiovascular disorders, ophthalmic disorders, metabolic disorders, inflammation & immunological diseases and others), [E] scale of operation (preclinical / clinical and commercial) and [F] geographical location (North America, Europe, Asia Pacific and rest of the world).
Chapter 12 summarizes the overall report. In this chapter, we have provided a list of key takeaways from the report, and expressed our independent opinion related to the research and analysis described in the previous chapters.
Chapter 13 is a collection of interview transcripts of the discussions held with key stakeholders in this market. In this chapter, we have presented the details of our conversations with (in alphabetical order) Haifeng Chen (Chief Executive Officer, Virovek), Jeffrey Hung (Chief Commercial Officer, Vigene Biosciences) and Kai Lipinski (Chief Scientific Officer, Vibalogics).
Chapter 14 is an appendix, which provides tabulated data and numbers for all the figures provided in the report.
Chapter 15 is an appendix, which provides a list of companies and organizations mentioned in this report.
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Gene Therapy Products Market is Slated To Witness Tremendous Growth in Coming Years | Major Players GlaxoSmithKline plc., Merck KGaA, Novartis AG,…
The persuasive Gene Therapy Products Market research report provides explanation about market trends, future prospects, market restraints, leading market drivers, market segments, key developments, key players in the market, and competitor strategies. A thorough analysis has been performed in this report to know the potential of the market in the present and the future prospects from various angles. This Gene Therapy Products Market document also contains a precise investment analysis which forecasts forthcoming opportunities for the market players in the Healthcare industry. Gene Therapy Products Market research analysis is one of the finest options to resolve business challenges quickly by saving lot of time.
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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.
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.
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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.
Segmentation: Global Gene Therapy Products Market
Gene Therapy Products Market : By Product
Gene Therapy Products Market : By Application
Gene Therapy Products Market : ByGeography
Key Developments in the Gene Therapy Products Market:
Gene Therapy Products Market Drivers
Gene Therapy Products Market Restraints
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Gene Therapy Products Market is Slated To Witness Tremendous Growth in Coming Years | Major Players GlaxoSmithKline plc., Merck KGaA, Novartis AG,...
Cancer Gene Therapy Market to Enable the Stakeholders to Capitalize on the Prevailing Industry Opportunities with Investment Analysis By Top Players -…
The report is an all-inclusive research study of the global Cancer Gene Therapy market taking into account the growth factors, recent trends, developments, opportunities, and competitive landscape. The market analysts and researchers have done extensive analysis of the global Cancer Gene Therapy market with the help of research methodologies such as PESTLE and Porters Five Forces analysis. They have provided accurate and reliable market data and useful recommendations with an aim to help the players gain an insight into the overall present and future market scenario. The Cancer Gene Therapy report comprises in-depth study of the potential segments including product type, application, and end user and their contribution to the overall market size.
The Cancer Gene Therapy Market carries out financial changes that occur year by years in market, with information about upcoming opportunities and risk to keeps you ahead of competitors. The report also describes top company profiles that present in market with trends worldwide. This research guided you for extending business.
The Cancer Gene Therapy Market research report presents a comprehensive assessment of the market and contains thoughtful insights, facts, historical data and statistically-supported and industry-validated market data and projections with a suitable set of assumptions and methodology. It provides analysis and information by categories such as market segments, regions, and product type and distribution channels.
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The key players covered in this studyAdaptimmuneBluebird bioCelgeneShanghai Sunway BiotechShenzhen SiBiono GeneTechSynerGene TherapeuticsAltor BioScienceAmgenArgenxBioCancellGlaxoSmithKlineMerckOncoGenex PharmaceuticalsTransgene
Market segment by Type, the product can be split intoOncolytic VirotherapyGene TransferGene-Induced Immunotherapy
Market segment by Application, split intoHospitalsDiagnostics CentersResearch Institutes
Market segment by Regions/Countries, this report coversUnited StatesEuropeChinaJapanSoutheast AsiaIndiaCentral & South America
A proper understanding of the Cancer Gene Therapy Market dynamics and their inter-relations helps in gauging the performance of the industry. The growth and revenue patterns can be revised and new strategic decisions taken by companies to avoid obstacles and roadblocks. It could also help in changing the patterns using which the market will generate revenues. The analysis includes an assessment of the production chain, supply chain, end user preferences, associated industries, proper availability of resources, and other indexes to help boost revenues.
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Market Segmentation based On Type, Application and Region:
The global Cancer Gene Therapy is analyzed for different segments to arrive at an insightful analysis. Such segmentation has been done based on type, application and Region.
Global Cancer Gene Therapy market is presented to the readers as a holistic snapshot of the competitive landscape within the given forecast period. It presents a comparative detailed analysis of the all regional and player segments, offering readers a better knowledge of where areas in which they can place their existing resources and gauging the priority of a particular region in order to boost their standing in the global market.
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For the future period, sound forecasts on market value and volume are offered for each type and application. In the same period, the report also provides a detailed analysis of market value and consumption for each region. These insights are helpful in devising strategies for the future and take necessary steps. New project investment feasibility analysis and SWOT analysis are offered along with insights on industry barriers. Research findings and conclusions are mentioned at the end.
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Curative Oncology and Rare Disease Therapies Transforming the Global Biologics Market, 20202024 – Yahoo Finance
With this research service, our Transformational Health team provides critical insights into the global biologics industry highlighting the growth opportunities, market revenue segmentation, and technology trends influencing its growth.
New York, Aug. 11, 2020 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Curative Oncology and Rare Disease Therapies Transforming the Global Biologics Market, 20202024" - https://www.reportlinker.com/p05950884/?utm_source=GNW The global biologics market covered in this study includes 4 major segments antibody therapies, recombinant proteins, vaccines, and regenerative medicine.
The forecast for the global biologics market till 2024 reveals interesting trend and paradigm shifts that are set to take place in the industry. Importantly, the study also identifies actionable growth opportunities for industry participants to profit upon.This research service identifies the largest revenue-generating segments and key therapeutic areas for the global biologics industry. Accelerated regulatory approvals for biologic products and the emergence of next-generation technologies are highlighted, in addition to their influence on market trends during the forecast period. The study highlights the dynamics of the biologics industry and the sub-segments of the biologics market. Growth opportunities in antibody-drug conjugate (ADC), RNA therapy, and gene therapy platforms are also explored. Further, the study discusses the impact (such as the accelerated development of vaccines) of the COVID-19 pandemic on the biologics industry and the market. Current challenges facing the biologics industry such as the sluggish growth rate of vaccines, and recombinant proteins are discussed. Most importantly, this research service discusses possible future market trends such as the accelerated development of mRNA-based vaccines and the increasing importance of specialty bio-CDMOs for the development and commercialization of regenerative medicine. Based on the market trends and revenue forecasts, our group of analysts highlight key growth opportunities in ADC, RNA, and gene therapy platforms through successful drug launches such as Belantamab Malfodotin, Inclisiran, and Zolgensma.Read the full report: https://www.reportlinker.com/p05950884/?utm_source=GNW
About ReportlinkerReportLinker is an award-winning market research solution. Reportlinker finds and organizes the latest industry data so you get all the market research you need - instantly, in one place.
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Curative Oncology and Rare Disease Therapies Transforming the Global Biologics Market, 20202024 - Yahoo Finance
CAR T cell therapy makes strides in tackling Hodgkin lymphoma – Baylor College of Medicine News
Most patients with Hodgkin lymphoma (HL), a type of cancer of immune cells called B lymphocytes, are cured with first-line therapy, but approximately 15 percent have primary refractory disease or relapse after an initial response to treatment.
Dr. Carlos Ramos at Baylor College of Medicine and his colleagues at Baylor and the University of North Carolina Lineberger Comprehensive Cancer Center have tackled relapsing or refractory HL with a novel treatment combination that produced promising results in a clinical trial.
In a 2017 pilot study in seven refractory/relapsing Hodgkin lymphoma patients, Baylor and UNC researchers found that a CAR-T cell therapy targeting Hodgkin cell-associated protein CD30 appeared safe but brought about only modest responses, said Ramos, co-first author of the study and professor in the Margaret M. and Albert B. Alkek Department of Medicine and the Center for Cell and Gene Therapy at Baylor, Houston Methodist Hospital and Texas Childrens Hospital.
In the new study, which included 41 patients treated at Baylor and UNC, the researchers used the same anti-CD30 CAR-T cell strategy, but added a preparative regimen in which patients existing lymphocytes a broad family of white blood cells including T cells were greatly depleted with chemotherapy drugs prior to the addition of the CAR-T cells.
The combination of lymphodepletion with anti-CD30 CAR-T led to the complete disappearance of tumor in the majority of patients treated at the highest dose level of therapy, with almost all patients having clinical benefit after treatment, said Ramos, a member of the Dan L Duncan Comprehensive Cancer Center at Baylor.
Lymphodepletion prior to CAR-T cell infusion seems to produce a more favorable environment for the CAR-T cells to proliferate and attack their cancerous targets, said Dr. Natalie Grover, study co-first author, assistant professor in the UNC Department of Medicine and a UNC Lineberger member.
These results are particularly exciting because the majority of these patients had lymphomas that had not responded well to other powerful new therapies, said study senior author Dr. Barbara Savoldo, professor in the UNC Department of Microbiology and Immunology at the UNC School of Medicine and a UNC Lineberger member. Everyone worked tirelessly on the study, and I am proud of the collaborative work it fueled between UNC Lineberger and Baylor.
I am very pleased to see this strategy, developed by Dr. Savoldo when she was at Baylor and initially translated to the clinic with Dr. Ramos, has now been validated in larger clinical trials at two centers, said co-author Dr. Helen Heslop, director of the Center for Cell and Gene Therapy and a Dan L Duncan Chair at Baylor.
Side effects of the lymphodepletion plus CAR-T cell treatment were common and included flu-like symptoms due to an immune chemical storm called cytokine release syndrome, but these events were generally modest. None of the patients experienced the more serious, life-threatening complications, such as brain swelling, that have been seen in CAR-T cell trials against other blood cancers.
As the trial progressed, the researchers settled on fludarabine as a key element of the pre-therapy lymphodepletion regimen, since patient outcomes seemed better when it was used. The researchers found that among the 32 patients with active cancer who received fludarabine for lymphodepletion before their CAR-T cells, 19 patients (59 percent) had a complete response.
Of the patients in the study who had a complete response, 61 percent still had no evidence of recurrence a year later. Overall, 94 percent of the treated patients were still alive a year after their treatment.
One patients remarkable recovery is detailed in the Dan L Duncan Comprehensive Cancer Center Annual Report. The researchers hope to do further studies of the CAR-T cell therapy alone and in combination with other new immune-modulating anticancer drugs.
Read the complete study in the Journal of Clinical Oncology
Other authors who contributed to this work include Dr. Anne W. Beaven, Dr. Premal D. Lulla, Meng-Fen Wu, Dr. Anastasia Ivanova, Dr. Tao Wang, Dr. Thomas C. Shea, Dr. Cliona M. Rooney, Christopher Dittus, Steven I. Park, Dr. Adrian P. Gee, Dr. Paul W. Eldridge, Kathryn L. McKay, Birju Mehta, Catherine J. Cheng, Faith B. Buchanan, Bambi J. Grilley, Dr. Kaitlin Morrison, Dr. Malcolm K. Brenner, Dr. Jonathan S. Serody and Dr. Gianpietro Dotti. The authors are affiliated with Baylor College of Medicine and the University of North Carolina at Chapel Hill.
The research was supported by the National Institutes of Health (RO1HL114564, P50CA126752, P30 CA125123, K12CA120780), the University Cancer Research Fund, and the Leukemia Lymphoma Society.
By Molly Chiu
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CAR T cell therapy makes strides in tackling Hodgkin lymphoma - Baylor College of Medicine News
Humanigen Expands Phase III Study of Lenzilumab in COVID-19 to Brazil – Business Wire
BURLINGAME, Calif.--(BUSINESS WIRE)--Humanigen, Inc., (HGEN) (Humanigen), a clinical stage biopharmaceutical company focused on preventing and treating cytokine storm with lenzilumab, the companys proprietary Humaneered anti-human granulocyte macrophage-colony stimulating factor (GM-CSF) monoclonal antibody, announced that the Brazilian regulatory agency, Anvisa, has granted permission to commence a Phase III study of lenzilumab in patients with COVID-19 in Brazil.
This study, now set to begin recruiting patients in Brazil, follows the same protocol approved by the US Food and Drug Administration in April a multicenter, randomized, placebo-controlled, double-blinded clinical trial focused on hospitalized severe and critical adult COVID-19 patients at high risk of disease progression. Humanigen is working with Clinical Trial & Consulting (CTI), recently named the top global contract research organization, to conduct this trial in Brazil.
Cameron Durrant, MD, MBA, chief executive officer of Humanigen, said, COVID-19 is a global crisis and we are committed to offering assistance to patients across the world that are impacted by the pandemic. We hope that expanding the study of lenzilumab to research centers in Brazil, a country with surging rates of COVID-19, will offer patients much needed access to a leading COVID-19 therapeutic candidate.
Currently, Brazil has the second highest reported rates of COVID-19 infection in the world, second only to the US.
Access to clinical trials is critical for healthcare providers in the fight against COVID-19, remarked Timothy Schroeder, chief executive officer of CTI. We are proud to collaborate with our colleagues at Humanigen to extend the reach of the Phase III study of lenzilumab and bring a potential treatment option to those in need.
More details on Humanigens programs in COVID-19 can be found on the companys website at http://www.humanigen.com under the COVID-19 tab, and details of the US Phase III potential registration study can be found at clinicaltrials.gov using ClinicalTrials.gov Identifier NCT04351152.
About Humanigen, Inc.
Humanigen, Inc. is developing its portfolio of clinical and pre-clinical therapies for the treatment of cancers and infectious diseases via its novel, cutting-edge GM-CSF neutralization and gene-knockout platforms. We believe that our GM-CSF neutralization and gene-editing platform technologies have the potential to reduce the inflammatory cascade associated with coronavirus infection. The companys immediate focus is to prevent or minimize the cytokine release syndrome that precedes severe lung dysfunction and ARDS in serious cases of SARS-CoV-2 infection. The company is also focused on creating next-generation combinatory gene-edited CAR-T therapies using strategies to improve efficacy while employing GM-CSF gene knockout technologies to control toxicity. In addition, the company is developing its own portfolio of proprietary first-in-class EphA3-CAR-T for various solid cancers and EMR1-CAR-T for various eosinophilic disorders. The company is also exploring the effectiveness of its GM-CSF neutralization technologies (either through the use of lenzilumab as a neutralizing antibody or through GM-CSF gene knockout) in combination with other CAR-T, bispecific or natural killer (NK) T cell engaging immunotherapy treatments to break the efficacy/toxicity linkage, including to prevent and/or treat graft-versus-host disease (GvHD) in patients undergoing allogeneic hematopoietic stem cell transplantation (HSCT). Additionally, Humanigen and Kite, a Gilead Company, are evaluating lenzilumab in combination with Yescarta (axicabtagene ciloleucel) in patients with relapsed or refractory large B-cell lymphoma in a clinical collaboration. For more information, visit http://www.humanigen.com.
About CTI Clinical Trial and Consulting Services
CTI Clinical Trial and Consulting Services is a global, privately held, full-service contract research organization (CRO) recently named #1 CRO in the world for quality, delivering a complete spectrum of clinical trial and consulting services throughout the lifecycle of development, from concept to commercialization. CTIs focused therapeutic approach provides pharmaceutical, biotechnology, and medical device firms with clinical and disease area expertise in rare diseases, regenerative medicine/gene therapy, immunology, transplantation, nephrology, hematology/oncology, neurology, infectious diseases, hepatology, cardiopulmonary, and pediatric populations. CTI also offers a fully integrated multi-specialty clinical research site that conducts phase I-IV trials. CTI has a passion for helping life-changing therapies succeed in chronically and critically ill patient populations. With clinical trial experience across 6 continents, CTI partners with research sites, patients, and sponsors to fulfill unmet medical needs. CTI is headquartered in the Greater Cincinnati, OH area, with operations across North America, Europe, Latin America, and Asia-Pacific. For more information visit http://www.ctifacts.com.
Forward-Looking Statements
This release contains forward-looking statements. Forward-looking statements reflect management's current knowledge, assumptions, judgment and expectations regarding future performance or events. Although management believes that the expectations reflected in such statements are reasonable, they give no assurance that such expectations will prove to be correct and you should be aware that actual events or results may differ materially from those contained in the forward-looking statements. Words such as "will," "expect," "intend," "plan," "potential," "possible," "goals," "accelerate," "continue," and similar expressions identify forward-looking statements, including, without limitation, statements regarding our expectations surrounding our ongoing and anticipated clinical trial activities in the United States and Brazil and our operational, research, development or commercialization activities for lenzilumab and the other product candidates in our current pipeline. Forward-looking statements are subject to a number of risks and uncertainties including, but not limited to, the risks inherent in our lack of profitability and need for additional capital to conduct the Phase III study and grow our business; our dependence on partners to further the development of our product candidates; the uncertainties inherent in the development and launch of any new pharmaceutical product; the outcome of pending or future litigation; and the various risks and uncertainties described in the "Risk Factors" sections and elsewhere in the Company's periodic and other filings with the Securities and Exchange Commission.
All forward-looking statements are expressly qualified in their entirety by this cautionary notice. You should not place undue reliance on any forward-looking statements, which speak only as of the date of this release. We undertake no obligation to revise or update any forward-looking statements made in this press release to reflect events or circumstances after the date hereof or to reflect new information or the occurrence of unanticipated events, except as required by law.
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Humanigen Expands Phase III Study of Lenzilumab in COVID-19 to Brazil - Business Wire
Cancer Gene Therapy Market Predicted to Witness Sustainable Evolution in Years to : Industry Outlook: Post COVID Investors Eye Bigger-Than-Expected…
Global Cancer Gene Therapy Market Size, Share, Growth Trends, Industry Report from DBMR highlights deep analysis on market characteristics, sizing, estimates and growth by segmentation, regional breakdowns & country along with competitive landscape, players market shares, and strategies that are key in the market. Cancer Gene Therapy Market research report provides key analysis on the market status of the Cancer Gene Therapy manufacturers with market size, growth, share, demand, revenue, trends as well as industry cost structure. The exploration provides a 360 view and insights, highlighting major outcomes of the industry. These insights help the business decision-makers to formulate better business plans and make informed decisions to improved profitability. In addition, the study helps venture or private players in understanding the companies in more detail to make better informed decisions.
According to DBMR, the GlobalCancer Gene Therapy Marketis expected to see a growth rate of 32.54%. The high success rate of cancer gene therapy along with clinical trial and the preclinical trial is gaining popularity among the patient which is leading towards the market.
Download Sample (350 Pages PDF) Report: To Know the Impact of COVID-19 on this[emailprotected]https://www.databridgemarketresearch.com/request-a-sample/?dbmr=global-cancer-gene-therapy-market
Market Overview of Global Cancer Gene Therapy
Increase in funding of research and development in the activities of cancer gene therapy along with rise in prevalence of cancer is likely to accelerate the growth of the cancer gene therapy market in the forecast period of 2020-2027. On the other hand, the favourable government regulations for therapy is further going to boost various opportunities that will lead to the growth of the cancer gene therapy market in the above mentioned forecast period. High cost involved in gene therapy along with unwanted immune responses wills likely to hamper the growth of the cancer gene therapy market in the above mentioned forecast period.
Global Cancer Gene Therapy Market Research Report also provides the latest companies data and industry future trends, allowing you to identify the products and end users driving profits growth and productivity. The Market report lists the most important competitors and provides the insights strategic industry Analysis of the key factors influencing the market. The report includes the forecasts, investigation and discussion of significant industry trends, market volume, market share estimates and profiles of the leading industry Players. Global Cancer Gene Therapy Industry Market Research Report is providing exclusive vital statistics, information, data, trends and competitive landscape details.
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The titled segments and sub-section of the market are illuminated below
By Therapy (Oncolytic Virotherapy, Gene Induced Immunotherapy, Gene Transfer)
By End User (Hospitals, Oncology Institutes, Biotechnological Companies, Clinical Research Laboratories)
Top Key Manufactures or Players (this may not be a complete list and extra companies can be added upon request):
Adaptimmune
GlaxoSmithKline plc
bluebird bio, Inc
Merck & Co., Inc
CELGENE CORPORATION
Anchiano Therapeutics
Achieve Life Sciences
..
Complete Report is Available (Including Full TOC, List of Tables & Figures, Graphs, and Chart)@https://www.databridgemarketresearch.com/toc/?dbmr=global-cancer-gene-therapy-market
The report also focuses on global major leading industry players of Global Cancer Gene Therapy Market providing information such as company profiles, product picture and specification, price, capacity, cost, production, revenue and contact information. Upstream raw materials and equipment and downstream demand analysis is also carried out. The Global Cancer Gene Therapy market development trends and marketing channels are analyzed. Finally, the feasibility of new investment projects is assessed and overall research conclusions offered.
Global Cancer Gene Therapy Market Scope and Market Size
Cancer gene therapy market is segmented on the basis of therapy and end user. The growth amongst these segments will help you analyse meagre growth segments in the industries, and provide the users with valuable market overview and market insights to help them in making strategic decisions for identification of core market applications.
Based on therapy, the cancer gene therapy market is segmented into oncolytic virotherapy, gene induced immunotherapy and gene transfer. The oncolytic virotherapy is further sub-segmented into adenovirus, lentivirus, retro virus, adeno associated virus, herpes simplex virus, alpha virus, vaccinia virus, simian virus and others. The gene induced immunotherapy is further sub-segmented into delivery of cytokines gene and delivery of tumor antigen gene. The gene transfer is further sub-segmented into naked/plasmid vectors, electroporation, sonoporation, magnetofection and gene gun.
Cancer gene therapy market has also been segmented based on the end user into hospitals, oncology institutes, biotechnological companies and clinical research laboratories.
According to the Regional Segmentation the Cancer Gene Therapy Market provides the Information covers following regions:
North America
South America
Asia & Pacific
Europe
MEA (Middle East and Africa)
The key countries in each region are taken into consideration as well, such as United States, Canada, Mexico, Brazil, Argentina, Colombia, Chile, South Africa, Nigeria, Tunisia, Morocco, Germany, United Kingdom (UK), the Netherlands, Spain, Italy, Belgium, Austria, Turkey, Russia, France, Poland, Israel, United Arab Emirates, Qatar, Saudi Arabia, China, Japan, Taiwan, South Korea, Singapore, India, Australia and New Zealand etc.
North America dominates the cancer gene therapy market due to the advanced healthcare infrastructure along with rise in R & D expenditure, while Asia-Pacific is expected to grow with the highest growth rate in the forecast period of 2020 to 2027 due to the improving healthcare infrastructure and government initiatives.
For More Information or Query or Customization Before Buying,[emailprotected]https://www.databridgemarketresearch.com/inquire-before-buying/?dbmr=global-cancer-gene-therapy-market
Scope of Report:
The Market report lists the most important competitors and provides the insights strategic industry Analysis of the key factors influencing the market. This report will help you to establish a landscape of industrial development and characteristics of the Cancer Gene Therapy market. The Global Cancer Gene Therapy market analysis is provided for the international markets including development trends, competitive landscape analysis, and key regions development status. Development policies and plans are discussed as well as manufacturing processes and cost structures are also analyzed. This report also states import/export consumption, supply and demand Figures, price, cost, revenue and gross margins.
Important Features that are under offering & key highlights of the report:
Market Data Segmentation with production, consumption, revenue (million USD), and Price Analysis
Detailed overview of Cancer Gene Therapy market
Changing market dynamics of the industry and Impact of Influencing Factors
In-depth market segmentation by Type, Application and other major segments etc.
To analyse and forecast the Global Cancer Gene Therapy market, in terms of value and volume.
Which segment has the potential to gain the highest market share?
To help decision maker from new offer perspective and benchmark existing marketing strategy.
Correlate cost structure historical data with key business segments.
Analyse marketing contribution and customer acquisition by up-selling and cross selling.
Identifying Influencing factors keeping Global Cancer Gene Therapy Market Intense, factored with periodic analysis of CR4 & CR8 concentration ratio & HHI Index.
Order a Copy of Cancer Gene Therapy Market[emailprotected]https://www.databridgemarketresearch.com/checkout/buy/singleuser/global-cancer-gene-therapy-market
With tables and figures helping analyze worldwide Global Cancer Gene Therapy market, this research provides key statistics on the state of the industry and is a valuable source of guidance and direction for companies and individuals interested in the market.
Major Highlights of TOC:
Chapter One: Market Overview
1.1. Introduction
1.2. Scope/Objective of the Study
Chapter Two: Executive Summary
2.1. Introduction
Chapter Three: Market Dynamics
3.1. Introduction
3.2. Market Drivers, Trends, Challenges, Opportunities
Chapter Four: Market Factor Analysis
4.1. Porters Five Forces
4.2. Supply/Value Chain
4.3. PESTEL analysis
4.4. Market Entropy
4.5. Impact Analysis Post COVID-19
Continued !!!
Chapter Nine: Methodology and Data Source
Thanks for reading this article; you can also get individual chapter wise section or region wise report version like North America, Europe or Asia.
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Mission Bio Lands $70M to Expand Cancer Therapies With Single-Cell Multi-Omics Platform – HIT Consultant
What You Should Know:
Mission Bio raises $70 million in Series C funding led byNovo Growth to accelerate the development of cancer therapies with single-cell multi-omics.
Mission Bios Tapestri Platform, the first single-cell platform capable of detecting DNA and protein changes simultaneously, gives a profound insight into the mutations that drive disease to accelerate the development of novel treatments and save patient lives.
Mission Bio,Inc., a South San Francisco, CA-based pioneer in high-throughputsingle-cell DNA and multi-omic analysis, today announced $70 million in itsSeries C financing led by Novo Growth. Soleus Capital also joins the round, alongwith earlier investors Mayfield, Cota, and Agilent, bringing the companys totalfunding to more than $110 million.
Lack of Understanding at the Single-Cell Level
Cancer is a disease of single cells, and it is driven by DNAmutations. Until now, weve been using outdated tech thats misled us to focuson the wrong parts of these cancer cells. Due to this, our currentunderstanding of the disease at the single-cell level has been incomplete.
Only 3% of cancer drugs tested in clinical trials between2000 and 2015 have been approved to treat patients, and even those that havebeen approved are not as effective as wed hope. Even those aimed at specificbiomarkers like DNA mutations arent effective in every patient with thatmutation, resulting in high rates of relapse. Ultimately, the complexity ofdisease evolution and therapy response requires comprehensive tools tocharacterize how DNA mutations drive disease at the cellular level, includingfunctional changes in the protein. To develop durable therapies, we require adeeper understanding of all analytes of the cell.
One Platform. Genotype + Phenotype
Founded in 2014, Mission Bio Is the first and onlysingle-cell multi-omics platform developed to accelerate the discovery,development, and delivery of treatments for cancer. The Tapestri Platform isthe first-ever single-cell multi-omics platform capable of detecting DNA andprotein changes simultaneously from the same cell a capability necessary forthe development of impactful precision therapies.
The award-winning technology has been adopted by Agios, LabCorp, Onconova Therapeutics, andother companies to optimize clinical trials and improve the likelihood ofsuccess. Leading cancer centers, including Stanford, MD Anderson Cancer Center,the University of California at San Francisco, and Memorial Sloan KetteringCancer Center, have leveraged the Tapestri Platform to support their work intherapeutic resistance and treatment response monitoring.
Over the past year, Mission Bio has advanced its efforts tosupport drug development through clinical trials and the translation ofresearch into novel biomarkers with its growing install base in NCI cancercenters and key biopharma companies.
Expansion Plans
To support Mission Bios rapid growth, the funds will scaleits single-cell multi-omics technology, the Tapestri Platform, to expand thecompanys reach in more effective clinical trials for novel cancer treatments,along with expanding in cell and gene therapy.
Were on a mission to help our customers eliminate cancer. With access to multiple layers of the cellular profile, the Tapestri Platform can help identify novel biomarkers in diseased cells, monitor therapy resistance and response, and accelerate novel therapies through clinical trials, said Charlie Silver, CEO, and Founder of Mission Bio. We are delighted to partner with Novo Growth to advance our impact in drug development and cell and gene therapy.
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Mission Bio Lands $70M to Expand Cancer Therapies With Single-Cell Multi-Omics Platform - HIT Consultant
Cell Therapy Manufacturing: Here are the Key Innovations | Roots Analysis – Bulletin Line
Last week, during our weekly industry update sessions, our prime focus was to understand the recent innovations in the field of cell therapy manufacturing. We were also looking out for players that are driving these innovations.
To start with, the first news that caught our attention was Novartis decision to sidestep Europe travel ban to provide CAR-T drug Kymriah to patients. Amid the growing concerns regarding the rapid rise in COVID-19 cases, this decision by Novartis certainly comes as a much needed good news.
In this article, we will discuss some of the key innovations in cell therapy manufacturing that have caught the industrys attention in the last few weeks.
For More Insights Click Here
Cell Therapy Supply Chain
In one of our recent articles, we talked about the strategies that cell therapy companies can adopt to improve the supply chain. Some of the players, such as (illustrative list) Trakcel, Veeva, Be the Match BioTherapies have already deployed solutions efficiently manage and streamline various aspects of the complex supply chains of cell and advanced therapies. In fact, more than 160 innovative software-enabled systems have been developed for managing the cell and advanced therapies supply chain. The key supply chain challenges in cell therapies are:
Also, across the social media platforms, there has been an immense rise in the popularity of the cell therapy supply chain solutions.
Last week, TrakCel, and Ori Biotech inked a non-exclusive strategic partnership to achieve supply chain coordination within closed-system manufacturing.
Earlier last year, Lonza announced a collaboration with Cryoport to Strengthens its vein-to-vein delivery network in cell therapy manufacturing. This partnership incorporated Cryoports Cryoportal Logistics Management Platform, SmartPak II Condition Monitoring System and Cryoports unique Chain of Compliance for the regulatory solutions.
Vector Manufacturing
One industry that has benefitted the most with the rise of cell and gene therapies is the vector manufacturing space. As a result of the growing demand for such therapies, there has been a significant increase in demand for preclinical and clinical-grade gene delivery vectors.
One other trend that has been observed is the prominent role that the contract manufacturers (CMOs) have played. In fact, existing manufacturing concerns in this field have led many players to outsource vector to different CMOs. The recent deal signed between Lonza and Prevail Therapeutics for AAV based gene therapies is a perfect example of this. Here is a quick snapshot of various CMOs that offer vector manufacturing services.
Last month, we saw an investment of over 50 million by Gamma Biosciences to push to market Univercells manufacturing technology. The technology has been designed to reduce the costs of viral vector production for gene therapies. Several key opinion leaders have highlighted limited capacity and high cost as key concerns with vector manufacturing. In addition, the high vector manufacturing cost has also pushed several players to look beyond viral vectors.
Closed Automated Processing Platform
Lonza recently announced a partnership with three research institutes. It included the transfer of their preclinical immunotherapies to Lonzas Cocoon closed automated processing platform. The collaborators include Stanford University School of Medicine, Fred Hutchinson Cancer Research Center and Parker Institute for Cancer Immunotherapy. The Cocoon system is based on an automated GMP-in-a-box concept for patient-scale cell therapy manufacturing.
This was our take on the key innovations. Keen to know your thoughts on what other innovations that we may have missed.
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Cell Therapy Manufacturing: Here are the Key Innovations | Roots Analysis - Bulletin Line
Global Cell and Gene Therapy Market, Forecast to 2025 by Product, Disease, End-user and Region – COVID-19 Updated – PRNewswire
DUBLIN, Aug. 10, 2020 /PRNewswire/ -- The "Cell & Gene Therapy Market - Global Outlook and Forecast 2020-2025" report has been added to ResearchAndMarkets.com's offering.
In-depth Analysis and Data-driven Insights on the Impact of COVID-19 Included
The study considers the present scenario of the cell and gene therapy market and its market dynamics for the period 2019-2025. It covers a detailed overview of several market growth enablers, restraints, and trends. The report offers both the demand and supply aspects of the market. It profiles and examines leading companies and other prominent ones operating in the market.
Key Questions Answered
1. What is the cell and gene therapy market size and growth rate during the forecast period?2. What are the factors impacting the growth of the cell and gene therapy market share?3. How is the growth of the healthcare segment affecting the growth of the cell and gene therapy market?4. Who are the leading vendors in the cell and gene therapy market, and what are their market shares?5. Which product type/ end-user type/region is generating the largest revenue in the Asia-Pacific region?
The global cell and gene therapy market by revenue is expected to grow at a CAGR of over 30.9% during the period 2019-2025
The global cell and gene therapy market is one of the fastest-growing segments in the regenerative medicine market. The market is expected to grow at a faster pace during the forecast period. The demand can be attributed to the growing prevalence of several chronic diseases such as cancer, cartilage related problems, wounds, diabetic foot ulcer, genetic disorders, and other rare diseases across the globe.
The prevalence of cancer and diabetes is increasing in the global population, which is influencing the growth of the market. There is a large unmet need in the treatment available, which is filled by cell and gene therapies. The market is growing due to the increased availability of funding from various public and private institutions. Besides, there is increased support from regulatory bodies for product approval. Several governments are creating awareness of cell and gene therapies in the population.
Cell and Gene Therapy Market Segmentation
The global cell and gene therapy market research report includes a detailed segmentation by product, disease, end-user, and geography.
In 2019, the cell therapy segment accounted for a market share of over 53% in the global cell and gene therapy market. The segment is expected to grow at a steady rate during the forecast period due to the increase in the target population and the rise in the number of countries preferring cell therapies in their patients. Increased therapeutic benefits are attracting several countries to invest in this technology and conduct a high number of clinical trials. However, the lack of advanced infrastructure in developing countries is hindering the growth of the segment.
In 2019, the oncology segment accounted for a share of over 40% in the global cell and gene therapy market. Oncology has been one of the targets of intense research for the gene therapy procedures & approach. More than 60% of on-going gene therapy clinical trials are targeting cancer. The segment is expected to grow at a promising rate on account of the high prevalence of cancer diseases, especially in low and middle-come countries. The market is growing at a double-digit CAGR, which is expected to help the segment as many cell and gene therapy for cancer are commercially available.
The dermatology application segment in the cell and gene therapy includes wound care management among patients. Vendors are focusing on the development and commercialization of advanced wound care products for the treatment of chronic and acute wounds, thereby increasing the growth of the wound care market. The increased pervasiveness of diabetics is increasing acute and chronic wounds, including surgical wounds, pressure ulcers, diabetic foot ulcers, and other wounds.
In 2019, the oncology segment accounted for a share of over 40% in the global cell and gene therapy market. Oncology has been one of the targets of intense research for the gene therapy procedures & approach. More than 60% of on-going gene therapy clinical trials are targeting cancer. The segment is expected to grow at a promising rate on account of the high prevalence of cancer diseases, especially in low and middle-come countries. The market is growing at a double-digit CAGR, which is expected to help the segment as many cell and gene therapy for cancer are commercially available.
The dermatology application segment in the cell and gene therapy includes wound care management among patients. Vendors are focusing on the development and commercialization of advanced wound care products for the treatment of chronic and acute wounds, thereby increasing the growth of the wound care market. The increased pervasiveness of diabetics is increasing acute and chronic wounds, including surgical wounds, pressure ulcers, diabetic foot ulcers, and other wounds.
Segmentation by Product
Segmentation by Disease
Segmentation by End-user
Insights by Geography
In 2019, North America accounted for a share of over 60% of the global cell and gene therapy market. There are more than 530 regenerative medicine companies, including cell and gene therapy manufacturing developers. The number of products approved in North America grew significantly in 2019, with developers filed for marketing authorization for 10+ regenerative medicines, many of which we expect to be approved in 2020. Within the next 1-2 years, the number of approved gene therapies is expected to double.
The US and Canada are the major contributors to the cell and gene therapy market in North America. Regulatory bodies are supporting several investigational products, fast track approvals, RMAT designation for the faster approval of the product into the market. The alliance for regenerative medicine and Medicare and Medicaid is working together to bring the structured reimbursement channels for cell and gene therapies.
Segmentation by Geography
Insights by Vendors
The global cell and gene therapy market is highly dynamic and characterized by the presence of several global, regional, and local vendors offering a wide range of therapies. Dendreon, Gilead Sciences, Novartis, Organogenesis, Osiris Therapeutics, Vericel, Amgen, and Spark Therapeutics are the leading players in the market with significant shares.
Vendors such as NuVasive, APAC Biotech, Nipro, Orthocell, bluebird bio, J-TEC, and Terumo are the other prominent players in the market with a presence, especially in the cell therapy market. Most leading players are focusing on implementing strategies such as product launches and approvals, marketing and promotional activities, acquisitions, increased R&D investments, and strengthening their distribution networks to enhance their share and presence in the market.
Prominent Vendors
Other Prominent Vendors
Market Dynamics
Opportunities & Trends
Growth Enablers
Growth Restraints
For more information about this report visit https://www.researchandmarkets.com/r/is5t27
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Global Cell and Gene Therapy Market, Forecast to 2025 by Product, Disease, End-user and Region - COVID-19 Updated - PRNewswire
112M Fund Launched to Commercialize UCLs Gene Therapy… – Labiotech.eu
The leading UK institution University College London and the firm AlbionVC have made the first closing of a 112M (100M) fund with the aim of investing in UCL research and spinouts with a focus on gene and cell therapy.
This is the second so-called UCL Technology Fund and could double the size of the first, which raised around 56M (50M) in 2016. The fund is managed by AlbionVC in collaboration with UCLs commercialization company UCL Business (UCLB). Investors in the second fund include the firm British Patient Capital and UCL itself.
While the value of the first closing was not disclosed, Anne Lane, CEO of UCLB, told me that investments from this fund have already begun, and the second close is expected within the next 12 months. The larger size of the fund than the first could also give the team more flexibility on how many investments it makes and how big they are.
We have a focus in terms of UCLs cell & gene therapy research and that is reflected in the portfolio of the fund, but no specific disease areas as such, Lane said.
Examples of investments in the pipeline for this fund include a gene therapy for an undisclosed neurometabolic disorder, a gene therapy for epilepsy, and a cell therapy for glaucoma.
One of the biggest success stories from the first fund is the gene therapy company Orchard Therapeutics, whose Nasdaq IPO raised around 290M in 2018. Another UCL gene therapy spinout, Freeline Therapeutics, is also geared to launch a Nasdaq IPO in the coming weeks.
UCL has a strong entrepreneurial scene comparable to the biotech hubs in Oxford and Cambridge. For example, UCL spinouts reportedly raised 644M (579M) in external investment between 2018 and 2019, the largest amount of any university in the country.
The European startup scene has been shaken by the Covid-19 pandemic this year, with the eurozone economy going into a deep recession. Furthermore, gene therapy developers have experienced disruption with their programs due to clinical trial delays and changes in strategy.
Regarding the effect of Covid-19 on their fundraising, Lane said that the pandemic definitely made it more challenging and took more time than expected. But UCLs research continues and we look to its research base in overcoming the challenges posed by the global pandemic.
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112M Fund Launched to Commercialize UCLs Gene Therapy... - Labiotech.eu
Spark Therapeutics Deepens Drug Development Expertise in Hematology and Rare Disease with Appointment of Gallia G. Levy, MD, Ph.D., as Chief Medical…
PHILADELPHIA, Aug. 10, 2020 (GLOBE NEWSWIRE) -- Spark Therapeutics, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY) and a fully integrated, commercial gene therapy company dedicated to challenging the inevitability of genetic disease, today announced the appointment of Gallia Levy, M.D., Ph.D., as chief medical officer. Dr. Levy will be responsible for strategic and operational leadership across all functions in the product development lifecycle, including setting the global development strategy for current and future pipeline programs.
We are thrilled to welcome Dr. Gallia Levy to our growing gene therapy company striving to create a world where no life is limited by genetic disease, said Jeffrey D. Marrazzo, chief executive officer, Spark Therapeutics. Dr. Levys passion for hematology and gene therapy research is immediately evident and exactly the perspective needed to achieve our goal of unlocking the full potential of gene therapy. Especially during this pivotal time in hemophilia research, Dr. Levys deep understanding of rare blood disorders and the community will help accelerate our ability to deliver potentially transformative gene therapies for hemophilia, while progressing potential gene therapies for other genetic disease across our pipeline.
Dr. Levy joins Spark Therapeutics from Genentech, also a member of the Roche Group, where she served as the Vice President and Global Head of the Rare Blood Disorders franchise in Product Development. In this role, she was responsible for the clinical development of HEMLIBRA for hemophilia A as well as treatments for other rare blood disorders such as paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS). She played a key role in the evolution of gene therapy as a new modality within the Roche Group.
Ive spent my career working to find new, innovative treatment approaches for patients affected by rare, life-altering disorders, and it is with great pride that I join the Spark team to help advance novel gene therapy programs and create next-generation solutions for patients, said Dr. Levy. Spark Therapeutics shares the same affinity for breaking barriers and putting the patient first, and I look forward to what we will achieve together.
Dr. Levy first joined Genentech in 2009, where she worked in both early and late-stage clinical development. She later moved to Portola Pharmaceuticals, where she led the clinical development program for hematology and oncology indications and returned to Genentech in 2014 to lead the hemophilia program.
Dr. Levy is board-certified in hematology and holds an M.D. and Ph.D. in Molecular and Cellular Biology from the University of Michigan. She completed her residency in internal medicine at Stanford University and a fellowship in hematology and oncology at the University of California, San Francisco. She also holds an M.S. in of Molecular and Cellular Biology from the University of Paris, VI and a B.A. from the University of California, Berkeley.
About Spark Therapeutics AtSpark Therapeutics, a fully integrated, commercial company committed to discovering, developing and delivering gene therapies, we challengethe inevitability of genetic diseases,includingblindness, hemophilia, lysosomal storage disorders and neurodegenerative diseases.We currently have four programs in clinical trials.At Spark, a member of the Roche Group, we see the path to a world where no life is limited by genetic disease. For more information, visit http://www.sparktx.com, and follow us on Twitter and LinkedIn.
Media Contact:Kevin Giordanokevin.giordano@sparktx.com(215) 294-9942
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Spark Therapeutics Deepens Drug Development Expertise in Hematology and Rare Disease with Appointment of Gallia G. Levy, MD, Ph.D., as Chief Medical...
LogicBio Therapeutics Reports Second Quarter 2020 Financial Results and Provides Business UpdatesFDA Clears IND Application for LB-001 for the…
LEXINGTON, Mass., Aug. 10, 2020 (GLOBE NEWSWIRE) -- LogicBio Therapeutics, Inc. (Nasdaq:LOGC) (LogicBio or the Company), a company dedicated to extending the reach of genetic medicine with pioneering targeted delivery platforms, today reported financial results for the quarter ended June 30, 2020, provided a business update and announced the U.S. Food and Drug Administration (FDA) has cleared the Companys Investigational New Drug (IND) application for LB-001 for the treatment of methylmalonic acidemia in pediatric patients. LogicBio released a separate press release this morning providing further details on the planned Phase 1/2 clinical design for LB-001.
We are thrilled to have received clearance to move forward with this first-in-human clinical trial with our lead product candidate, LB-001, for the treatment of methylmalonic acidemia, a life-threatening congenital genetic disease with no current therapeutic treatment options. This represents a significant milestone in our goal of bringing a treatment to MMA patients as well as for our GeneRide platform. We have maintained continuous dialogue with the centers of excellence that are planned to participate in the Phase 1/2 clinical trial, and we look forward to activating these sites as quickly as possible, said Fred Chereau, CEO of LogicBio. We have instituted systems attempting to mitigate COVID-19 dynamics on our study start-up process and, based on our best estimates, we plan to enroll our first patient in early 2021.
Commenting on the Next Generation Capsid Program, Mr. Chereau said, We are very excited about the recent advances in our novel capsid program, which has generated liver-tropic capsids intended for use in gene editing technologies such as GeneRide and other gene therapy approaches. We are focused on executing across all of our programs and look forward to sharing further details on our novel capsids in early 2021.
Appointment of Daniel Gruskin, M.D. to SVP, Head of Clinical Development
Daniel Gruskin, M.D. was appointed as SVP, head of clinical development in August 2020. Dr. Gruskin has served as interim head of clinical development of LogicBio since June 2020. In April 2020, Dr. Gruskin started consulting with the Company as a special advisor. Previously, Dr. Gruskin served in roles of increasing responsibility at Sanofi Genzyme, most recently as vice president, head of global medical affairs, rare disease, in which capacity he oversaw medical affairs, life cycle management, scientific affairs and other medical and development activities related to metabolic, rare and/or genetic diseases. Prior to his role at Sanofi Genzyme, Dr. Gruskin served as assistant professor, human genetics and pediatrics at Emory University School of Medicine, where he was also the chief of the genetics section at Childrens Healthcare of Atlanta.
Daniel has been instrumental in leading LB-001 clinical development efforts including getting the IND cleared. His deep experience in genetic medicines and metabolic diseases will serve LogicBio well as we look to execute on our goals for both the GeneRide and Next Generation Capsid platforms in search of transformative medicines, said Mr. Chereau.
Anticipated Milestones for 2020 and 2021:
Second Quarter 2020 Financial Results
Three Months Ended June 30, 2020 and 2019
About LogicBio Therapeutics
LogicBio Therapeuticsis dedicated to extending the reach of genetic medicine with pioneering targeted delivery platforms.
LogicBios proprietary genome editing technology platform, GeneRide, enables the site-specific integration of a therapeutic transgene without nucleases or exogenous promoters by harnessing the native process of homologous recombination. LogicBio has received FDA clearance for the first-in-human clinical trial of LB-001, a wholly owned genome editing program leveraging GeneRide for the treatment of methylmalonic acidemia. Patient enrollment is expected to begin in early 2021. In addition, LogicBio has a collaboration with Takeda to research and develop LB-301, an investigational therapy leveraging GeneRide for the treatment of the rare pediatric disease Crigler-Najjar syndrome.
LogicBio is also developing a Next Generation Capsid platform for use in gene editing and gene therapies. Data presented have shown that the capsids deliver highly efficient functional transduction of human hepatocytes with improved manufacturability with low levels of pre-existing neutralizing antibodies in human samples. Top-tier capsid candidates from this effort demonstrated significant improvements over benchmark AAVs currently in clinical development. LogicBio is developing these highly potent vectors for internal development candidates and potentially for business development collaborations.
LogicBio is headquartered inLexington, Mass. For more information, please visitwww.logicbio.com.
Forward Looking Statements
This press release contains forward-looking statements within the meaning of the federal securities laws, including those related to the Companys plans to initiate, advance and complete its planned SUNRISE Phase 1/2 clinical trial of LB-001 in MMA; the timing, progress and results of the Companys research and development activities, including those related to the GeneRide technology platform and Next Generation Capsid Program; its plans for LB-301 in Crigler-Najjar; and the sufficiency of its cash and cash equivalents to fund operating expenses and capital expenditure requirements. These are not statements of historical facts and are based on managements beliefs and assumptions and on information currently available. They are subject to risks and uncertainties that could cause the actual results and the implementation of the Companys plans to vary materially, including the risks associated with the initiation, cost, timing, progress and results of the Companys current and future research and development activities and preclinical studies and potential future clinical trials. In particular, the impact of the COVID-19 pandemic on the Companys ability to progress with its research, development, manufacturing and regulatory efforts, including the Companys plans to initiate, advance and complete its Phase 1/2 clinical trial for LB-001 in MMA, and the value of and market for the Companys common stock, will depend on future developments that are highly uncertain and cannot be predicted with confidence at this time, such as the ultimate duration of the pandemic, travel restrictions, quarantines, social distancing and business closure requirements in the United States and in other countries, and the effectiveness of actions taken globally to contain and treat the disease. These risks are discussed in the Companys filings with the U.S. Securities and Exchange Commission (SEC), including, without limitation, the Companys Annual Report on Form 10-K filed on March 16, 2020 with the SEC, the Companys Quarterly Report on Form 10-Q filed on May 11, 2020, and the Companys subsequent Quarterly Reports on Form 10-Q and other filings with the SEC. Except as required by law, the Company assumes no obligation to update these forward-looking statements publicly, even if new information becomes available in the future.
Contacts:
Investors:Brian LuqueAssociate Director, Investor Relationsbluque@logicbio.com951-206-1200
Media:Stephanie SimonTen Bridge CommunicationsStephanie@tenbridgecommunications.com617-581-9333
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LogicBio Therapeutics Reports Second Quarter 2020 Financial Results and Provides Business UpdatesFDA Clears IND Application for LB-001 for the...