StemCell Therapy

New York, Sept. 18, 2020 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Advanced Therapy Medicinal Products Market Research Report by Therapy Type - Global Forecast to 2025 - Cumulative Impact of COVID-19" - https://www.reportlinker.com/p05953100/?utm_source=GNW

The Global Advanced Therapy Medicinal Products Market is expected to grow from USD 2,946.38 Million in 2019 to USD 6,524.94 Million by the end of 2025 at a Compound Annual Growth Rate (CAGR) of 14.16%.

Market Segmentation & Coverage:This research report categorizes the Advanced Therapy Medicinal Products to forecast the revenues and analyze the trends in each of the following sub-markets:

Based on Therapy Type , the Advanced Therapy Medicinal Products Market studied across CAR-T Therapy, Cell Therapy, Gene Therapy, and Tissue Engineered Product. The Cell Therapy further studied across Non-stem Cell Therapy and Stem Cell Therapy.

Based on Geography, the Advanced Therapy Medicinal Products Market studied across Americas, Asia-Pacific, and Europe, Middle East & Africa. The Americas region surveyed across Argentina, Brazil, Canada, Mexico, and United States. The Asia-Pacific region surveyed across Australia, China, India, Indonesia, Japan, Malaysia, Philippines, South Korea, and Thailand. The Europe, Middle East & Africa region surveyed across France, Germany, Italy, Netherlands, Qatar, Russia, Saudi Arabia, South Africa, Spain, United Arab Emirates, and United Kingdom.

Company Usability Profiles:The report deeply explores the recent significant developments by the leading vendors and innovation profiles in the Global Advanced Therapy Medicinal Products Market including AveXis, Inc., Bluebird Bio, Inc., Celgene Corporation, Gilead Lifesciences, Inc., JCR Pharmaceuticals Co., Ltd., Kolon TissueGene, Inc., MEDIPOST, Novartis AG, Organogenesis Inc., PHARMICELL Co., Ltd, Spark Therapeutics, Inc., UniQure N.V., and Vericel Corporation.

FPNV Positioning Matrix:The FPNV Positioning Matrix evaluates and categorizes the vendors in the Advanced Therapy Medicinal Products Market on the basis of Business Strategy (Business Growth, Industry Coverage, Financial Viability, and Channel Support) and Product Satisfaction (Value for Money, Ease of Use, Product Features, and Customer Support) that aids businesses in better decision making and understanding the competitive landscape.

Competitive Strategic Window:The Competitive Strategic Window analyses the competitive landscape in terms of markets, applications, and geographies. The Competitive Strategic Window helps the vendor define an alignment or fit between their capabilities and opportunities for future growth prospects. During a forecast period, it defines the optimal or favorable fit for the vendors to adopt successive merger and acquisition strategies, geography expansion, research & development, and new product introduction strategies to execute further business expansion and growth.

Cumulative Impact of COVID-19:COVID-19 is an incomparable global public health emergency that has affected almost every industry, so for and, the long-term effects projected to impact the industry growth during the forecast period. Our ongoing research amplifies our research framework to ensure the inclusion of underlaying COVID-19 issues and potential paths forward. The report is delivering insights on COVID-19 considering the changes in consumer behavior and demand, purchasing patterns, re-routing of the supply chain, dynamics of current market forces, and the significant interventions of governments. The updated study provides insights, analysis, estimations, and forecast, considering the COVID-19 impact on the market.

The report provides insights on the following pointers:1. Market Penetration: Provides comprehensive information on the market offered by the key players2. Market Development: Provides in-depth information about lucrative emerging markets and analyzes the markets3. Market Diversification: Provides detailed information about new product launches, untapped geographies, recent developments, and investments4. Competitive Assessment & Intelligence: Provides an exhaustive assessment of market shares, strategies, products, and manufacturing capabilities of the leading players5. Product Development & Innovation: Provides intelligent insights on future technologies, R&D activities, and new product developments

The report answers questions such as:1. What is the market size and forecast of the Global Advanced Therapy Medicinal Products Market?2. What are the inhibiting factors and impact of COVID-19 shaping the Global Advanced Therapy Medicinal Products Market during the forecast period?3. Which are the products/segments/applications/areas to invest in over the forecast period in the Global Advanced Therapy Medicinal Products Market?4. What is the competitive strategic window for opportunities in the Global Advanced Therapy Medicinal Products Market?5. What are the technology trends and regulatory frameworks in the Global Advanced Therapy Medicinal Products Market?6. What are the modes and strategic moves considered suitable for entering the Global Advanced Therapy Medicinal Products Market?Read the full report: https://www.reportlinker.com/p05953100/?utm_source=GNW

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Advanced Therapy Medicinal Products Market Research Report by Therapy Type - Global Forecast to 2025 - Cumulative Impact of COVID-19 - GlobeNewswire

The CDC estimates that sickle cell disease affects well over 100,000 Americans, with the disease occurring most often in African Americans. September has been designated as National Sickle Cell Awareness month designed to focus attention on the ongoing research in this field and the need for new treatments. The sector can certainly point to the significant progress that has taken place during the past few years, with new medicines reaching the market and several novel therapeutics with new mechanisms of action advancing in the pipeline.

Ted Love, president and CEO of Global Blood Therapeutics Inc. (GBT), said 2019 was a landmark year with the FDA approval of two new novel therapies to treat sickle cell disease. He was speaking at the virtual annual Sickle Cell Disease (SCD) Therapeutics Conference this week. His company, together with the Sickle Cell Disease Association of America, was hosting the one-day event featuring discussions on the latest advances and future trends.

Approvals

The key manifestation of the inherited blood disorder is that red blood cells (RBCs) are abnormally shaped (crescent), which restricts their flow in blood vessels and limits oxygen delivery to the bodys tissues, leading to severe pain and organ damage. The condition is also characterized by severe chronic inflammation that results in vaso-occlusive crisis (VOC) where patients experience episodes of extreme pain and organ damage.

Late November, GBT gained accelerated approval for its Oxbryta (voxelotor) tablets for the treatment SCD in adults and pediatric patients 12 and older. The agencys green light came less than two weeks after it gave the go-ahead to Novartis AG for Adakveo (crizanlizumab) to reduce the frequency of VOCs in adult and pediatric patients ages 16 and older with SCD.

According to Love, Oxbryta is a new class of therapy binding to hemoglobin and stabilizing RBCs in an oxygenated state and inhibiting deoxygenated sickle hemoglobin polymerization, making cells less likely to bind together and form the distinctive sickle shape.

The launch of the drug has gone well since it was approved, he said in the companys second-quarter financial report and business update, despite the impact of COVID-19. Net sales in the period reached $31.5 million, well ahead of the Streets expectations. Going forward, the company is planning to expand the potential use of Oxbryta for the treatment of SCD in children as young as 4 years old and also seek marketing authorization in Europe for Oxbryta to treat hemolytic anemia in SCD patients ages 12 and older with a marketing authorization application being submitted to the EMA by the middle of next year.

Pipeline progress

Cambridge, Mass.-based Agios Pharmaceuticals Inc. is working on mitapivat (AG-348), an investigational, oral, small-molecule allosteric activator of wild-type and a variety of mutated pyruvate kinase-R (PKR) enzymes, in patients with SCD. The compound has been shown to decrease 2,3-diphosphoglycerate (2,3-DPG) and increase adenosine triphosphate (ATP), and through that mechanism, it may reduce hemoglobin (Hb) S polymerization and red blood cell sickling. In June, the company reported that clinical proof of concept had been established based on a preliminary analysis in a phase I trial being conducted in collaboration with the U.S. NIH as part of a cooperative research and development agreement.

The ongoing study had enrolled nine patients, with eight completing all planned dose levels of mitapivat. Seven of eight patients who completed all dose levels experienced a Hb increase, with five of eight patients (63%) achieving a hemoglobin increase of 1 g/dL from baseline (range 1-2.7 g/dL). All five patients who achieved a hemoglobin increase of 1 g/dL did so at doses of 50 mg BID or lower. Decreases in 2,3-DPG and increases in ATP levels were observed, consistent with the proposed mechanism of action and comparable to that observed in healthy volunteer studies with mitapivat.

The company said it expects to report data from ACTIVATE and ACTIVATE-T, its two global pivotal trials for mitapivat in adults with pyruvate kinase deficiency, between the end of 2020 and mid-2021.

Watertown, Mass.-based Forma Therapeutics Holdings Inc., which made its public debut this year, also has a selective RBC pyruvate kinase-R activator in its pipeline for treating SCD. FT-4202 is being evaluated in a phase I trial in SCD patients ages 12 and older and has been granted fast track, rare pediatric and orphan drug designations. The compound is a potent activator of pyruvate kinase-R designed to improve RBC metabolism, function and survival by decreasing 2,3 DPG and increasing ATP, potentially resulting in both increased hemoglobin levels and reduced VOCs.

Olinciguat, an oral guanylate cyclase (sGC) stimulator, being developed by Cyclerion Therapeutics Inc., has completed the treatment period in its STRONG-SCD study with a total of 70 patients randomized. The placebo-controlled, dose-ranging study is designed to evaluate safety, tolerability and pharmacokinetics, as well as to explore effects on daily symptoms and biomarkers of disease activity when dosed over a 12-week treatment period. Top-line results are expected this year. Olinciguat is a compound that aims to stimulate sGC production, leading to the production of a signaling molecule called cyclic guanosine monophosphate (cGMP). High levels of cGMP help reduce inflammation in blood vessels, decrease adhesion between RBCs, and allow for improved blood flow by increasing the availability of nitric oxide.

Boston-based Imara Inc. is developing IMR-687, a small-molecule inhibitor of PDE9 that degrades cyclic guanosine monophosphate (cGMP), an active signaling molecule that plays a role in vascular biology. The company said that lower levels of cGMP are often found in people with SCD and beta-thalassemia and are associated with impaired blood flow, increased inflammation, greater cell adhesion and reduced nitric oxide-mediated vasodilation. Blocking PDE9 acts to increase cGMP levels, which are associated with reactivation of fetal hemoglobin.

In August, the company dosed the first patient in its Ardent phase IIb trial of IMR-687 for adult patients with SCD. The planned primary efficacy objective is to evaluate the proportion of all patients with fetal hemoglobin (HbF) response, defined as an increase of 3% in HbF from baseline to week 24, compared to placebo.

Gene therapy/gene editing

Since SCD is a monogenic disease condition, researchers believe that it would be a good candidate for gene therapy therapeutic approaches. For example, significant progress is being made by Cambridge, Mass.-based Bluebird Bio Inc. with lentiglobin, its lentiviral-based gene therapy that inserts an anti-sickling beta-globin variant into CD34-positive cells, progenitors of red blood cells.

At the virtual European Hematology Association (EHA) meeting in June, it reported new data from its ongoing phase I/II study involving adult and adolescent patients with SCD that showed a near-complete reduction of serious VOCs and acute chest syndrome. The company expects to submit a BLA to the FDA for the gene therapy next year.

Crispr Therapeutics AG and Vertex Pharmaceuticals Inc. are progressing CTX-001, an investigational, autologous, CRISPR/Cas9 gene-edited hematopoietic stem cell therapy being evaluated for patients suffering from severe hemoglobinopathies. At EHA, the companies reported that in the phase I/II Climb-121 study, at nine months after CTX-001 infusion, the first treated patient was free of VOCs, was transfusion independent and had total hemoglobin levels of 11.8 g/dL, 46.1% fetal hemoglobin and F-cells (erythrocytes expressing fetal hemoglobin) of 99.7%.

Last month, Cambridge, Mass.-based Editas Medicine Inc., a genome editing company, reported that the FDA had granted rare pediatric disease designation for EDIT-301, an experimental, autologous cell medicine, being developed as a potentially best-in-class, durable medicine for SCD. The company plans to file an investigational new drug application for EDIT-301 by the end of this year. EDIT-301 comprises sickle patient CD34+ cells genetically modified using a hCRISPR/Cas12a (also known as Cpf1) ribonucleoprotein to edit the HBG1/2 promoter region in the beta-globin locus. Red blood cells derived from EDIT-301 CD34+ cells demonstrate a sustained increase in fetal hemoglobin (HbF) production.

In its second-quarter financial report and business update, Beam Therapeutics Inc. announced the nomination of its first two adenine base editing development candidates, BEAM-101, targeting patients with hereditary persistence of fetal hemoglobin, and BEAM-102 (Makassar variant), both aimed at correcting SCD.

New Initiative

The NIH, which reports it spends approximately $100 million on sickle cell disease research, announced that is has launched The Cure Sickle Cell Initiative designed to speed the development of cures for the disease. It will take advantage of the latest genetic discoveries and technological advances to progress the most promising genetic-based curative therapies safely into clinical trials within five to 10 years.

Aided by research partners, the initiative will establish a national data warehouse of genetic therapies for sickle cell disease and conduct comparative analyses of therapeutic approaches to assess both clinical and cost effectiveness. National networks will also be created to make it easier for patients and providers to interact with the research, clinical trials, and other activities.

Read this article:
New medicines in the pipeline to treat sickle cell disease - BioWorld Online

With September being Sickle Cell Disease Awareness Month, Loma Linda University Childrens Health wants to help educate the community about SCD one of the most common yet overlooked genetic disorders in the world.

Each year, approximately 1,000 babies in the U.S. and 500,000 worldwide are born with the disease, according to the Sickle Cell Disease Association of America.

Akshat Jain, MD, MPH, a global sickle cell disease expert at Childrens Hospital, is passionate about establishing awareness and proper care for children suffering from SCD and Sickle Cell Trait, especially the diverse patient population in San Bernardino County.

There are many barriers to receiving care for those with SCD in our community, Jain says. One barrier specifically is lack of awareness surrounding the disease coupled with lack of awareness surrounding the treatment options available at Childrens Hospital.

In sickle cell disease, a persons red blood cells have an irregular cell shape, Jain says. Instead of round discs, theyre in a crescent or sickle shape.

Due to their shape, texture and inflexibility, the cells become clumped together. This grouping causes a blockage in a childs blood vessels, hindering blood-flow. This blockage may cause varying levels of pain and potentially organ damage long-term.

Jain says some of the signs and symptoms of SCD include:

Jain says that many children with SCD develop symptoms in their first year of life. SCD is commonly diagnosed during newborn screening tests, which check for the abnormal hemoglobin found in SCD. Additionally, if both parents of a child are known carriers of a SCD trait, their child will have a 25% chance of having the disease, Jain says.

Some of the emergent issues needing immediate medical care in kids with SCD disease include:

Treatments for SCD include pain medicines for pain management, adequate hydration, blood transfusions, vaccines and antibiotics, and some medicines. Currently, stem cell transplant from bone marrow is the recognized cure for SCD.

Childrens Hospital, with Jain working as a lead on the team, performed the institutions first stem cell transplant in 2019, curing a then 11-year-old girl who had suffered from SCD since birth. Since then, the team has successfully performed the transplant on several pediatric patients.

Patients with SCD at Childrens Hospital are placed into a treatment and care program where Jain and his team offer non-traditional services such as individualized patient treatment plans and direct access to the care team in case of an emergent event. Additionally, the program is working toward offering curative gene therapy for both sickle cell and hemophilia patients.

The bottom line is children and families suffering from this disease need to know that theyre not alone, Jain says. Here at Childrens Hospital, we are here to manage and fight this disease alongside of you.

Learn more about our treatments for sickle cell disease at our Specialty Team Centers.

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Here's what to know about Sickle Cell Disease in kids - Loma Linda University Health

NEW YORK--(BUSINESS WIRE)--As part of a two-day virtual Investor Day, Pfizer Inc. (NYSE: PFE) provided an extensive overview of pipeline advances and shared updates on the Companys efforts to battle the COVID-19 pandemic on multiple fronts, including new data on the BNT162b2 vaccine candidate being developed in collaboration with BioNTech SE. The pipeline updates contribute to the Companys expectation of at least a 6% revenue CAGR over the next five years, as well as delivery of longer-term topline growth beyond that period.

Pfizers goal of delivering up to 25 breakthroughs to patients by the year 2025 has 38 such opportunities to draw from as of today, including the companys 20-valent pneumococcal conjugate vaccine candidate (20vPnC). On a non-risk adjusted basis, these opportunities collectively represent more than $15 billion (excluding 20vPnC) in potential incremental revenue for Pfizer from 2020 to 2025, as well as aggregate peak annual sales potential of $35 billion to $40 billion (including 20vPnC). If successful, the Companys COVID-19 programs would be incremental to these estimates.

Pfizers purpose Breakthroughs that change patients lives has never been more relevant, and our R&D pipeline has never been more dynamic, said Dr. Albert Bourla, Pfizer Chairman and CEO. I am proud of the truly transformational science that our research and clinical teams are bringing to the fight against disease, as well as the unprecedented speed with which we are advancing our clinical programs in the battle against COVID-19. In the coming months and years, I look forward to the new Pfizer continuing to demonstrate the agility and innovative spirit of a biotech combined with the scale of Big Pharma. With the depth and breadth of our current portfolio, the tremendous potential of our pipeline and scientific engine, and the power of our culture of innovation, we are poised to continue delivering meaningful value to patients by addressing some of the worlds most difficult health challenges.

UPDATES ON COVID-19 DEVELOPMENT PROGRAMS

Pfizer announced several key advances in its efforts to protect humankind from the COVID-19 pandemic and prepare the pharmaceutical industry to better respond to future global health crises.

BNT162 mRNA-based Vaccine Program

Pfizer and BioNTech shared several updates from their BNT162 mRNA-based vaccine program against SARS-CoV-2, the virus that causes COVID-19 disease, including:

Protease Inhibitor Program

The company announced the initiation of its Phase 1b clinical trial to evaluate the safety of a novel investigational therapeutic for COVID-19, PF-07304814. Of note,

THERAPEUTIC AREAS OF FOCUS

Pfizer shared significant research advances across its various therapeutic areas including candidates with blockbuster potential expected to launch by 2025.

Vaccines

In addition to the COVID-19 vaccine program, Pfizer aims to deliver five innovative vaccines by 2025, subject to clinical success and regulatory approval. Updates on these late-stage clinical development programs include:

Rare Disease

Pfizers Rare Disease late-stage pipeline currently includes three gene therapy programs that, if successful, are expected to gain regulatory approval by the end of 2023, with an additional pipeline of 10 preclinical initiatives that are at various stages of maturity. Key updates include:

Oncology

Pfizers Oncology pipeline has the potential to deliver up to 14 approvals expected by the end of 2025 and the potential for 24 new molecular entities in the clinic by the end of 2021. Key updates included, for the first time, early-stage opportunities obtained from the 2019 acquisition of Array BioPharma:

Inflammation and Immunology

The Inflammation & Immunology pipeline is focused on patients with autoimmune and chronic inflammatory diseases across rheumatology, gastroenterology and dermatology, with five distinct immuno-kinases, in oral and topical formulations, studied for potential treatment of 10 diseases, and three additional novel biologics in Phase 2 studies. Key updates included:

Internal Medicine

The Internal Medicine pipeline addresses the increasing global burden of cardiometabolic disease, with nine investigational medicines in active clinical studies and additional therapies in the pre-clinical pipeline. Key updates included:

To access a replay of the webcast, including audio, video and presentation slides, visit our web site at http://www.pfizer.com/investors.

About Pfizer: Breakthroughs That Change Patients Lives

At Pfizer, we apply science and our global resources to bring therapies to people that extend and significantly improve their lives. We strive to set the standard for quality, safety and value in the discovery, development and manufacture of health care products, including innovative medicines and vaccines. Every day, Pfizer colleagues work across developed and emerging markets to advance wellness, prevention, treatments and cures that challenge the most feared diseases of our time. Consistent with our responsibility as one of the world's premier innovative biopharmaceutical companies, we collaborate with health care providers, governments and local communities to support and expand access to reliable, affordable health care around the world. For more than 150 years, we have worked to make a difference for all who rely on us. We routinely post information that may be important to investors on our website at http://www.Pfizer.com. In addition, to learn more, please visit us on http://www.Pfizer.com and follow us on Twitter at @Pfizer and @Pfizer News, LinkedIn, YouTube and like us on Facebook at Facebook.com/Pfizer.

Disclosure Notice: The information contained in this release is as of September 15, 2020. Pfizer assumes no obligation to update forward-looking statements contained in this release or the webcast as the result of new information or future events or developments.

This release and the webcast contain forward-looking information about Pfizers anticipated operating and financial performance, business plans and prospects, Pfizers pipeline portfolio (including anticipated regulatory submissions, data read-outs, study starts, approvals, revenue contributions and market opportunities), and our efforts to respond to COVID-19, including our investigational vaccine candidate against SARS-CoV-2 and our investigational protease inhibitor, including their potential benefits, among other things, that are subject to substantial risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statements. Risks and uncertainties include, among other things, the uncertainties inherent in research and development, including the ability to meet anticipated clinical endpoints, commencement and/or completion dates for our clinical trials, regulatory submission dates, regulatory approval dates and/or launch dates, as well as the possibility of unfavorable new clinical data and further analyses of existing clinical data; risks associated with interim and preliminary data; the risk that clinical trial data are subject to differing interpretations and assessments by regulatory authorities; whether regulatory authorities will be satisfied with the design of and results from our clinical studies; whether and when any drug applications, biologics license applications and/or emergency use authorization applications may be filed in any jurisdictions for any potential indication for Pfizers product candidates; whether and when any such applications that may be filed for any of Pfizers product candidates may be approved by regulatory authorities, which will depend on myriad factors, including making a determination as to whether the product's benefits outweigh its known risks and determination of the product's efficacy and, if approved, whether any such product candidates will be commercially successful; decisions by regulatory authorities impacting labeling, manufacturing processes, safety and/or other matters that could affect the availability or commercial potential of Pfizers product candidates, including development of products or therapies by other companies; manufacturing capabilities or capacity; uncertainties regarding the ability to obtain recommendations from vaccine technical committees and other public health authorities and uncertainties regarding the commercial impact of any such recommendations; uncertainties regarding the impact of COVID-19 on Pfizers business, operations and financial results; and competitive developments.

A further description of risks and uncertainties can be found in Pfizers Annual Report on Form 10-K for the fiscal year ended December 31, 2019 and in its subsequent reports on Form 10-Q, including in the sections thereof captioned Risk Factors and Forward-Looking Information and Factors That May Affect Future Results, as well as in its subsequent reports on Form 8-K, all of which are filed with the U.S. Securities and Exchange Commission and available at http://www.sec.gov and http://www.pfizer.com.

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Pfizer Investor Day Features Significant Number of Pipeline Advances for COVID-19 Programs and Across Numerous Therapeutic Areas - Business Wire

Here are the top biotech companies you will find in Oxford, a city with an old scientific tradition and an enormous output of biotech applications.

Oxford is well known for its university, one of the oldest in Europe and considered to be one of the best in the world. Recently, the University of Oxford has been the center of attention thanks to an experimental Covid-19 vaccine that the university is developing in partnership with big pharmaceutical companies. If successful, the vaccine, already in phase III testing, could be one of the first to get approval for this new disease.

The University of Oxford has hosted thousands of bright minds over the years. Thanks to its emphasis on technology transfer, the university has also helped a large number of them turn their ideas into successful spinout companies. The environment created around the university has also attracted many talents and businesses to the city, making it the ideal melting pot for new and promising ideas.

Biotech is one of the fortes of the innovation seen in the city of Oxford. So we consulted with local experts to put together a list of the most remarkable companies in the city, be it for their size, innovation, or influence in the sector.

Founded in 2008, Immunocore is one of just a few private biotech companies in Europe that are estimated to be worth over 1B. The company is tackling multiple forms of cancer as well as infectious and autoimmune diseases using T-cell receptor (TCR) technology. TCRs are proteins on the surface of immune T cells that are responsible for identifying a threat that must be destroyed, such as cancerous or infected cells. Immunocore aims to patients with engineered TCRs to circumvent the mechanisms by which these threats evade the immune system, restoring its ability to fight disease.

The company is collaborating on several projects with Genentech, AstraZeneca, Eli Lilly, and GSK. Its most advanced program is a treatment for uveal melanoma that is currently in phase III trials. Other programs target solid tumors, hepatitis, HIV, and type 1 diabetes.

Adaptimmune Therapeutics was founded at the same time as Immunocore with the goal of exploiting TCR technology in the form of T-cell therapy. The company engineers the TCRs naturally present on the patients own immune T cells to improve their ability to identify cancerous cells.

Adaptimmune is now getting ready to start late-stage clinical trials in multiple cancer types. Thanks to a deal with Astellas Pharma, the company is also gearing up to start clinical testing of a version of its T-cell therapy that doesnt require engineering each dose individually for each patient, using donor cells instead. Adaptimmune also has several partnerships with companies including GSK, Noile-Immune Biotech, and Alpine Immune Sciences.

Oxford Biodynamics was spun out of Oxford University in 2007 with the goal of developing liquid biopsy tests that can perform a diagnosis from just a drop of blood. The company specializes in epigenetics, that is changes to the structure of our DNA that determines which genes are switched on or off.

Oxford Biodynamics works in a wide range of indications, including cancer, diabetes, Alzheimers, multiple sclerosis, and rheumatoid arthritis among many others. The tests are not only designed to diagnose a disease; they can also be made to determine which patients are going to benefit the most from a specific drug, and how likely the disease is to progress faster or relapse.

With these tests, the company is supporting the development of personalized medicine approaches and helping drug developers increase their chances of succeeding in clinical trials. Partners include big pharma such as Pfizer and EMD, as well as universities and research institutes.

Chronos Therapeutics started out in 2009 as a spinout of the University of Oxford with the goal of developing drugs for age-related conditions. The companys lead program targets amyotrophic lateral sclerosis (ALS), the most common motor neuron disease, for which there are no treatments able to stop its progression.

Over time, the company has expanded its portfolio into other indications, particularly those that affect the brain, by acquiring assets from third parties. They include programs targeting fatigue caused by multiple sclerosis, addictive behaviors such as binge eating and alcohol use disorder, and post-traumatic stress disorder.

Evox Therapeutics is developing a drug delivery technology based on exosomes nanoparticles that our bodies naturally use to transport molecules. The company engineers exosomes to carry different types of drugs, such as proteins, RNA, or small drugs. The key advantage is that these natural carriers are able to reach targets that conventional drug delivery systems cant, such as the brain.

Founded in 2016, the company has signed big deals with Takeda and Eli Lilly. Its programs are all in preclinical testing and mostly target rare diseases. One of them targets the rare liver disorder argininosuccinic aciduria and is scheduled to enter clinical trials in 2021.

Exscientia is a pioneer in the application of artificial intelligence to drug discovery. The company uses AI to identify potential drug candidates and optimize their structure to maximize their chances of success in clinical trials. The goal is to speed up and reduce the costs of the drug discovery process.

Earlier this year, the companys drug candidate for obsessive-compulsive disorder became one of the first AI-designed drugs to enter clinical trials. While a drug typically takes five years from identification until clinical trials, this one did so in just a year.

Exscientia is partnered with Bayer, BMS, Sanofi, and GSk among others. In the wake of the Covid-19 pandemic, the company set out to go through a database of 15,000 approved and investigational drugs that had already passed safety testing to find candidates that can then be fast-tracked to clinical testing in Covid-19 patients.

Oxford Biomedica was set up in 1995 as a developer and provider of lentiviral vectors for gene and cell therapy. These vectors allow the permanent introduction of a desired DNA sequence into a target cell, be it in the test tube or directly in the patients body.

The technology of Oxford Biomedica is regularly used by companies such as Novartis, Sanofi, Boehringer Ingelheim, Imperial Innovations, and Orchard Therapeutics. Notably, the vectors developed by the company are used in Novartis Kymriah, the first CAR T-cell therapy approved in Europe and the US as a cancer treatment.

Oxford Biomedica also has a preclinical pipeline of proprietary programs in a wide range of applications, including cancer, eye disease, ALS, and liver disease. Last year, the company struck a deal with Microsoft to reduce the complexity and costs of gene and cell therapy manufacturing using artificial intelligence.

Founded in 2016, Arctoris aims to bring the benefits of automation to cancer research. Through the companys services, a researcher could just order an experiment online and spend their valuable time designing experiments and analyzing results rather than performing the repetitive tasks needed to complete them.

Arctoris aims to contribute to reducing the costs of drug discovery, which are increasing every year as treatments become personalized and results more difficult to replicate. In the context of the Covid-19 pandemic, Arctoris has established assays that allow scientists the possibility of running Covid-19 experiments remotely.

Founded in 2015, Orbit Discovery is a drug discovery company focusing on the identification of peptide drugs. The company has developed a technology that significantly improves on conventional methods of drug screening such as phage display or mRNA display.

The technology consists of fusing peptides to the DNA sequence encoding them and presenting them to live cells. This method allows the screening of peptide targets that were previously missed by other technologies, and to study their effect on live cells to better predict their function.

The company has already identified several candidates in the areas of cardiology, immunology, and cancer, and is working with partners such as Zealand Pharma in their preclinical development.

OxStem is a drug discovery company with an unusual approach to stem cell treatments. Instead of using stem cells as a therapy, the firm focuses on developing small molecule drugs that can reprogram the patients own stem cells to treat a wide range of diseases related to aging, including cancer and diabetes as well as neurological, cardiovascular, and ocular conditions.

Founded in 2013, the strategy of OxStem is to spin out companies that specialize in each disease area to focus on the development of the drugs found by the parent company, with five subsidiaries set up so far.

See more here:
The Top 10 Biotech Companies Brewing at... - Labiotech.eu

One of the major challenges facing gene therapy a way to treat disease by replacing a patients defective genes with healthy ones is that it is difficult to safely deliver therapeutic genes to patients without the immune system destroying the gene, and the vehicle carrying it, which can trigger life-threatening widespread inflammation.

Three decades ago researchers thought that gene therapy would be the ultimate treatment for genetically inherited diseases like hemophilia, sickle cell anemia, and genetic diseases of metabolism. But the technology couldnt dodge the immune response.

Since then, researchers have been looking for ways to perfect the technology and control immune responses to the gene or the vehicle. However, many of the strategies tested so far have not been completely successful in overcoming this hurdle.

Drugs that suppress the whole immune system, such as steroids, have been used to dampen the immune response when administering gene therapy. But its difficult to control when and where steroids work in the body, and they create unwanted side effects. My colleague Mo Ebrahimkhani and I wanted to tackle gene therapy with immune-suppressing tools that were easier to control.

I am a medical doctor and synthetic biologist interested in gene therapy because six years ago my father was diagnosed with pancreatic cancer. Pancreatic cancer is one of the deadliest forms of cancer, and the currently available therapeutics usually fail to save patients. As a result, novel treatments such as gene therapy might be the only hope.

[Read: These tech trends defined 2020 so far, according to 5 founders]

Yet, many gene therapies fail because patients either already have pre-existing immunity to the vehicle used to introduce the gene or develop one in the course of therapy. This problem has plagued the field for decades, preventing the widespread application of the technology.

Traditionally scientists use viruses from which dangerous disease-causing genes have been removed as vehicles to transport new genes to specific organs. These genes then produce a product that can compensate for the faulty genes that are inherited genetically. This is how gene therapy works.

Though there have been examples showing that gene therapy was helpful in some genetic diseases, they are still not perfect. Sometimes, a faulty gene is so big that you cant simply fit the healthy replacement in the viruses commonly used in gene therapy.

Another problem is that when the immune system sees a virus, it assumes that it is a disease-causing pathogen and launches an attack to fight it off by producing antibodies and immune response just as happens when people catch any other infectious viruses, like SARS-CoV-2 or the common cold.

Recently, though, with the rise of a gene-editing technology called CRISPR, scientists can do gene therapy differently.

CRISPR can be used in many ways. In its primary role, it acts as a genetic surgeon with a sharp scalpel, enabling scientists to find a genetic defect and correct it within the native genome in desired cells of the organism. It can also repair more than one gene at a time.

Scientists can also use CRISPR to turn off a gene for a short period of time and then turn it back on, or vice versa, without permanently changing the letters of DNA that makes up our genome. This means that researchers like me can leverage CRISPR technology to revolutionize gene therapies in the coming decades.

But to use CRISPR for either of these functions, it still needs to be packaged into a virus to get it into the body. So some challenges, such as preventing the immune response to the gene therapy viruses, still need to be solved for CRISPR-based gene therapies.

Being trained as a synthetic biologist, I teamed up with Ebrahimkhani to use CRISPR to test whether we could shut down a gene that is responsible for the immune response that destroys the gene therapy viruses. Then we investigated whether lowering the activity of the gene, and dulling the immune response, would allow the gene therapy viruses to be more effective.

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CRISPR can precisely remove even single units of DNA. KEITH CHAMBERS/SCIENCE PHOTO LIBRARY/Getty Images

A gene called Myd88 is a key gene in the immune system and controls the response to bacteria and viruses, including the common gene therapy viruses. We decided to temporarily turn off this gene in the whole body of lab animals.

We injected animals with a collection of the CRISPR molecules that targeted the Myd88 gene and looked to see whether this reduced the number of antibodies that were produced to specifically fight our gene therapy viruses. We were excited to see that the animals that received our treatment using CRISPR produced less antibodies against the virus.

This prompted us to ask what happens if we give the animal a second dose of the gene therapy virus. Usually, the immune response against a gene therapy virus prevents the therapy from being administered multiple times. Thats because after the first dose, the immune system has seen the virus, and on the second dose, antibodies swiftly attack and destroy the virus before it can deliver its cargo.

We saw that animals receiving more than one dose did not show an increase in antibodies against the virus. And, in some cases, the effect of gene therapy improved compared with the animals in which we had not paused the Myd88 gene.

We also did a number of other experiments that proved that tweaking the Myd88 gene can be useful in fighting off other sources of inflammation. That could be useful in diseases like sepsis and even COVID-19.

While we are now beginning to improve this strategy in terms of controlling the activity of the Myd88 gene. Our results, now published in Nature Cell Biology, provide a path forward to program our immune system during gene therapies and other inflammatory responses using the CRISPR technology.

This article is republished from The Conversation by Samira Kiani, Associate Professor of Pathology, University of Pittsburghunder a Creative Commons license. Read the original article.

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How CRISPR is tackling the troubling immune response thats plagued gene therapy until now - TNW

BOSTON, Sept. 15, 2020 /PRNewswire/ --GreenLight Bioscienceshas received a $3.3 million grant from the Bill & Melinda Gates Foundation to develop new mRNA-based gene therapies for Sickle Cell Disease and other global health challenges.

The funding will support GreenLight's research and testing of affordable therapies using the company's novel messenger RNA (mRNA) approach to gene editing. mRNA technology is already being used to develop vaccine candidates for infectious diseases, including the COVID-19 pandemic.

While initial research will focus on a cure for Sickle Cell Disease, GreenLight plans to develop a versatile gene editing platform to address a variety of diseases affecting underserved patient populations, such as treating HIV in developing countries.

Sickle Cell Disease is a group of inherited blood disorders in which red blood cells develop abnormally, causing pain and anemia. More than 4 million people currently suffer from the disease, with another 40+ million having the sickle cell trait, which can be passed on to future generations. The disease primarily targets people of African, Hispanic, or Middle Eastern descent. Current treatment regimens including blood transfusions and bone marrow transplants are costly, invasive, and impractical for treating large segments of affected patient populations.

"Funders are recognizing the potential of our innovative approach to gene editing that, in combination with our proprietary RNA manufacturing capability, has the potential to deliver accessible gene therapies and improve human health globally," said Marta Ortega-Valle, senior vice president of Human Health and Corporate Development at GreenLight Biosciences. "Finding a safe and effective therapy is critical, but equally important is the ability to produce it affordably for broader access. We are grateful for the Gates Foundation's support to advance novel gene editing approaches for populations in which those therapies are currently out of reach."

Gene editing therapies hold significant promise in the treatment of Sickle Cell Disease since it is a disorder caused by gene mutation. Using RNA as its core, GreenLight Biosciences is working to develop an in vivo gene therapy that could ultimately offer a cure to the disease.

Once the therapy candidate is validated and moves into clinical use, GreenLight Biosciences' biomanufacturing platform will accelerate production of affordable treatments at scale. "Manufacturing sufficient quantities of high-quality RNA at an accessible cost is critical for achieving the full potential of new therapies that aim to reach a global patient population. That capability does not yet exist in the market, but GreenLight's end-to-end, self-contained manufacturing platform aims to make that possible for all mRNA-based therapies and vaccines," Ortega-Valle added.

About GreenLight Biosciences, Inc.GreenLight is a bio-performance company with a unique, cell-free production platform that delivers high-performing RNA solutions to human, plant and animal challenges. GreenLight develops RNA products for plant and life science applications, and collaborates with industry leaders to advance vaccine development, pandemic preparation, crop management, and plant protection. The cutting-edge, natural platform delivers higher-quality RNA at a lower cost and higher speed than was ever before possible. The GreenLight team values diversity, inclusion, and equality and promises to use collaboration to remain scientifically imaginative and passionately focused on making a difference in the world. For more information, visithttps://www.greenlightbiosciences.com/.

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GreenLight Biosciences Receives $3.3 Million Grant to Develop Sickle Cell Disease Cure Using mRNA Gene Therapy - PRNewswire

JOHNSON CITY, Tenn., Sept. 10, 2020 /PRNewswire/ --LabConnect, the preeminent provider of clinical trial central lab services, today announced the expansion of its Johnson City facility to support its significant growth in cell and gene therapy and immuno-oncology studies. The company,which recentlyrelocated its headquarters to Tennessee, is doubling the capacity of its biorepository for sample storage and its clinical trial kit building capacity. LabConnect welcomed Tennessee Governor Bill Lee, Congressman Phil Roe, Economic Development Commissioner Bob Rolfe, elected officials, and other dignitaries to celebrate this milestone in the company's growth.

"While we have all had to adjust and adapt during these unprecedented circumstances, Tennessee's business climate has remained strong and welcoming to companies around the globe," said Gov. Lee. "I applaud LabConnect for continuing to invest and create jobs in our state and for choosing to bring its headquarters to Tennessee. I look forward to the many great things that will come from this facility in Johnson City."

"We are pleased that another company has chosen to expand its presence in Tennessee," Commissioner Rolfe said. "LabConnect is committed to leading the way in central laboratory services, which will have a global impact from its Tennessee=based facility. We appreciate LabConnect for its continued innovation and for creating high quality jobs."

"We are excited that the incentives with the State, Tennessee Valley Authority, and Northeast Tennessee Regional Economic Partnership have enabled LabConnect to expand its operations," said Tom Sellig, LabConnect CEO. "Our location offers several advantages which has allowed us to provide unique services to pharmaceutical and biotech clients. We are currently serving 200 leading biopharmaceutical clients and proud of the more than 20 products we have supported that are now FDA approved and used to treat patients around the world. The expanded capacity will allow us to scale our organization to meet LabConnect's growing demand for our clients' cell & gene therapy, rare and orphan diseases, and immuno-oncology projects."

For more information, visit http://www.labconnect.com.

About LabConnectConnect with LabConnectthe preeminent provider of central laboratory support services for analytically and logistically complex studies such as immuno-oncology, cell and gene therapies, and rare & orphan diseases. The company offers unique and innovative services that have been specifically designed to meet the exacting demands of today's clinical trials. The worldwide scope of services includes routine and specialized testing, real-time sample tracking, data integration, biorepository, sample processing and specialized functional outsourcing. Leading the evolution in central laboratory services since 2002, LabConnect's services are customized to fit the unique needs of biopharmaceutical clients. Get connected by requesting a proposal at http://www.labconnect.com or via email at [emailprotected].

SOURCE LabConnect

http://www.labconnect.com

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LabConnect Announces Expansion to Support Cell & Gene Therapy Growth - PRNewswire

New Jersey contract manufacturer Catalent has been right in the mix in the COVID-19 response effort, signing pacts to help produce frontrunners in the vaccine hunt. Now, the company is fleshing out a Maryland facility to aid in that effortand position Catalent's cell and gene therapy offerings well into the future.

Catalent will infuse $130 million into its cell and gene therapy manufacturing facility in Harmans, Maryland, to broaden the CDMO's late-stage production capacity, the company said Wednesday.

The newest investment will add five late-stage clinical and commercial manufacturing suites to the Harmans site, expected to go online in the first half of 2022, Catalent said.

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That expansion will bring the total number of manufacturing suites to 15 at the planned 350,000-square-foot complexnear the Baltimore/Washington International airport. The Harmans facility recently received FDA approval for commercial production, and its initial 10 manufacturing suites are set to be fully operational by the first quarter of 2021.

The five new suites will be located in a second building at the site that will also house cold-storage warehousing and added office space, Catalent said. The Harmans complex is one of five Maryland sites for Catalent's cell and gene therapy manufacturing portfolio.

Adding capacity at its Harmans site is a future play for Catalent in the bustling cell and gene therapy space, but the facility could also benefit the CDMO's immediate COVID-19 response efforts.

RELATED:AstraZeneca ropes in Catalent gene therapy site to produce viral vectors for COVID-19 vaccine

Last month, British drugmaker AstraZeneca tappedCatalent to help produce bulk drug substance and viral vectors at the Harmans facility for the University of Oxford's adenovirus-based COVID-19 vaccine.

Catalent will start production there this quarter,buildingon its previous pact with AstraZeneca for fill-finish and packaging duties at its Anagni, Italy, site.

Catalent's work will include production of viral vectors for a genetically modified form of the adenovirus used in Oxford's shot, dubbed AZD1222. The modified virus, known as ChAdOx1 nCoV-19, aims to induce a lasting immune response to spike proteins added to the virus's surface.

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Catalent injects $130M into Maryland cell and gene therapy site drafted into COVID-19 vaccine hunt - FiercePharma

Sarepta has gone all-in on gene therapy over the last few years, racing with Pfizer and Solid Biosciences to be the first to develop a genetic fix for Duchenne muscular dystrophy, one of the most common rare diseases.

Sarepta has been comfortably in the lead, collecting the first positiveresults and snaring a $1.15 billion cash commercialization deal with Roche, but this week the company hit a snag. Late yesterday, Sarepta provided a program update for its gene therapy, revealing that in a scheduled meeting the FDA had raised concerns about the kinds of tests they would use to measure potency in the pivotal study and commercial supply for the gene therapy. The company has assays that might fit the criteria, they said, but needed additional dialogue with the agency to confirm.

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FDA knocks back Sarepta in Duchenne gene therapy race with Pfizer, but analysts urge caution - Endpoints News

CAMBRIDGE, Mass., Sept. 09, 2020 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that it has completed a Type C written response only meeting with the Office of Tissues and Advanced Therapies (OTAT), part of the Center for Biologics Evaluation and Research (CBER) at the U.S. Food and Drug Administration (FDA), to obtain OTATs concurrence on the commencement of its next clinical trial for SRP-9001 using commercial process material. SRP-9001 (AAVrh74.MHCK7.micro-dystrophin) is Sareptas investigational gene transfer therapy for the treatment of Duchenne muscular dystrophy.

Among other items, OTAT has requested that Sarepta utilize an additional potency assay for release of SRP-9001 commercial process material prior to dosing in a clinical study. Sarepta has several existing assays and data that it believes could be employed in response to OTATs request. However, additional dialogue with the Agency is required to determine the acceptability of the potency assay approach.

We look forward to working with OTAT to potentially satisfy their requests and to obtain clarity on the timing of the commencement of our commercial supply study. We will provide further updates as we are able, said Doug Ingram, president and chief executive officer, Sarepta Therapeutics. Every day, thousands of children degenerate from the irreversible damage caused by Duchenne muscular dystrophy. It is for that reason that we will work relentlessly with the Division to satisfy any requests of OTAT and continue the advancement of a potentially transformative therapy for these patients.

About SRP-9001 (AAVrh74.MHCK7.micro-dystrophin)SRP-9001 is an investigational gene transfer therapy intended to deliver the micro-dystrophin-encoding gene to muscle tissue for the targeted production of the micro-dystrophin protein. Sarepta is responsible for global development and manufacturing for SRP-9001 and plans to commercialize SRP-9001 in the United States. In December 2019, the Company announced a licensing agreement granting Roche the exclusive right to launch and commercialize SRP-9001 outside the United States. Sarepta has exclusive rights to the micro-dystrophin gene therapy program initially developed at the Abigail Wexner Research Institute at Nationwide Childrens Hospital.

AboutSarepta TherapeuticsAt Sarepta, we are leading a revolution in precision genetic medicine and every day is an opportunity to change the lives of people living with rare disease. The Company has built an impressive position in Duchenne muscular dystrophy (DMD) and in gene therapies for limb-girdle muscular dystrophies (LGMDs), mucopolysaccharidosis type IIIA, Charcot-Marie-Tooth (CMT), and other CNS-related disorders, with more than 40 programs in various stages of development. The Companys programs and research focus span several therapeutic modalities, including RNA, gene therapy and gene editing. For more information, please visitwww.sarepta.com or follow us on Twitter, LinkedIn, Instagram and Facebook.

Sarepta Forward-Looking Statements

This press release contains "forward-looking statements." Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as "believes," "anticipates," "plans," "expects," "will," "intends," "potential," "possible" and similar expressions are intended to identify forward-looking statements. These forward-looking statements include statements regarding Sareptas belief that its existing assays and data could be employed in response to OTATs request; the acceptability of Sareptas potency assay approach by the FDA; our plan to work with OTAT to potentially satisfy their requests and to obtain clarity on the timing of the commencement of our commercial supply study; and the potential of SRP-9001 to be a transformative therapy for DMD patients.

These forward-looking statements involve risks and uncertainties, many of which are beyond Sareptas control. Known risk factors include, among others: delays in the commencement of Sareptas next clinical study for SRP-9001 could delay, prevent or limit our ability to gain regulatory approval for SRP-9001; any inability to complete successfully clinical development could result in additional costs to Sarepta or impair Sareptas ability to generate revenues from product sales, regulatory and commercialization milestones and royalties; SRP-9001 may not result in a viable treatment suitable for commercialization due to a variety of reasons, including the results of future research may not be consistent with past positive results or may fail to meet regulatory approval requirements for the safety and efficacy of product candidates; Sarepta may not be able to execute on its business plans and goals, including meeting its expected or planned regulatory milestones and timelines, clinical development plans, and bringing its product candidates to market, due to a variety of reasons, many of which may be outside of Sareptas control, including possible limitations of company financial and other resources, manufacturing limitations that may not be anticipated or resolved for in a timely manner, regulatory, court or agency decisions, such as decisions by the United States Patent and Trademark Office with respect to patents that cover Sareptas product candidates and the COVID-19 pandemic; and those risks identified under the heading Risk Factors in Sareptas most recent Annual Report on Form 10-K for the year ended December 31, 2019, and most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) as well as other SEC filings made by Sarepta which you are encouraged to review.

Any of the foregoing risks could materially and adversely affect Sareptas business, results of operations and the trading price of Sareptas common stock. For a detailed description of risks and uncertainties Sarepta faces, you are encouraged to review the SEC filings made by Sarepta. We caution investors not to place considerable reliance on the forward-looking statements contained in this press release. Sarepta does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof.

Internet Posting of Information

We routinely post information that may be important to investors in the 'For Investors' section of our website atwww.sarepta.com. We encourage investors and potential investors to consult our website regularly for important information about us.

Source: Sarepta Therapeutics, Inc.

Sarepta Therapeutics, Inc.

Investors:Ian Estepan, 617-274-4052iestepan@sarepta.com

Media:Tracy Sorrentino, 617-301-8566tsorrentino@sarepta.com

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Sarepta Therapeutics Provides Program Update for SRP-9001, its Investigational Gene Therapy for the Treatment of Duchenne Muscular Dystrophy -...

Collaboration partners,Neurocrine Biosciences (NASDAQ:NBIX) and Voyager Therapeutics (NASDAQ:VYGR) announced data from an early stage clinical trial with Parkinson's disease patients and NBIb-1817, an experimental gene therapy. Three years after a single administration, 14 out of 15 patients are still reporting motor function improvements.

At the moment, NBIb-1817 is in the middle of a phase 2 study that was put on hold in April due to the COVID-19 pandemic. If allowed to restart, the phase 2 RESTORE-1 trial will randomize patients to receive NBIb-1817 or a placebo, then measure for a change in "On" time without troublesome dyskinesia.

Image source: Getty Images.

This potential new treatment option uses a viral vector to deliver a gene encoding an enzyme that helps Parkinson's disease patients convert levodopa into the dopamine they need.

Administering NBIb-1817 requires magnetic resonance imaging (MRI) to guide a pair of infusions that deliver the therapy directly into the striatum, a structure deep in the center of the brain. Needles through the skull aren't anybody's idea of a good time, but the side effects that come with daily doses of levodopa aren't any fun either.

For patients with severe Parkinson's disease that has a diminishing response to levodopa, the temporary discomfort that comes with NBIb-1817 treatment seems like a trade-off most will be willing to accept. Three years after a single treatment with NBIb-1817, patients were able to reduce their daily levodopa doses from a baseline of 1500.9 milligrams per day to 1061.4 milligrams per day. Despite reducing their levadopa intake, patients receiving three different dosage strengths of NBIb-1817 improved average "On" time by up to 2.23 hours.

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Parkinson's Disease Patients Get Long-Term Benefits From Experimental Gene Therapy - The Motley Fool

CAMBRIDGE, Mass., Sept. 15, 2020 /PRNewswire/ --Obsidian Therapeutics, Inc., a biotechnology company pioneering controllable cell and gene therapies, today announced that Bristol Myers Squibb (NYSE:BMY) has exercised its option to an exclusive worldwide license to a cell therapy candidate based on Obsidian's cytoDRiVE technology for the controlled expression of the immunomodulatory factor CD40L. This announcement marks the first opt-in decision by Bristol Myers Squibbsince the companies announced their collaboration to develop novel cell therapies in January 2019. Under the terms of the agreement, Obsidian is eligible to receive potential future milestone and royalty payments.

"We are very interested in exploring innovative approaches to developing engineered cell therapies, including the cytoDRiVE platform," said Rupert Vessey, D. Phil., Executive Vice President, Research and Early Development, Bristol Myers Squibb. "By controllingthe expression of armed payloads like CD40L, Obsidian's cell therapy candidates may have the potential to overcome tumor microenvironment resistance and unlock the power of cell therapy in solid tumors and other malignancies."

"This announcement marks an important milestone validating Obsidian's cytoDRiVE platform, and we look forward to continuing to work with Bristol Myers Squibbto bring powerful new immunotherapies to patients," said Paul K. Wotton, Ph.D., Chief Executive Officer of Obsidian Therapeutics. "We are also pleased with the pace with which our own pipeline programs are progressing as we continue to advance our lead controllable tumor infiltrating lymphocyte (TIL) therapy to the clinic."

About Obsidian TherapeuticsObsidian Therapeutics is a biotechnology company pioneering controllable cell and gene therapies to deliver transformative outcomes for patients with intractable diseases. Obsidian's proprietary cytoDRiVE technology provides a way to control protein degradation using FDA-approved small molecules, permitting precise control of the timing and level of protein expression. The cytoDRiVE platform can be applied to design controllable intracellular, membrane and secreted proteins for cell and gene therapies as well as other applications. The Company's initial applications focus on developing novel cell therapies for the treatment of cancer. Obsidian is headquartered in Cambridge, Mass. For more information, please visit http://www.obsidiantx.com.

Media Contact:

Maggie BellerRusso Partners, LLC[emailprotected]646-942-5631

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Obsidian Therapeutics Announces Bristol Myers Squibb Opt-In of cytoDRiVE Cell Therapy Candidate - PRNewswire

Pfizer has tipped its pipeline to deliver new products that add more than $15 billion to revenues by 2025. The anticipated pipeline contributions led Pfizer to predict it will weather the next wave of patent expirations that is set to start in 2026.

At its virtual investor day Tuesday, Pfizer predicted (PDF) that JAK1 inhibitor abrocitinib, antisense therapy vupanorsen and a 20-valent pneumococcal conjugate vaccine can generate peak annual revenues of more than $3 billion each. Pfizer also tipped its Duchenne muscular dystrophy gene therapy to rake in an excess of $2 billion and identified six other potential blockbusters.

With Pfizer tipping another five pipeline prospects to generate revenues of $500 million to $1 billion, the Big Pharma said sales of as-yet-unapproved experimental products could come to $15 billion by 2025.

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The peak years of the experimental medicines likely lie beyond 2025. The timing of the anticipated peaks of those products coincides with the years in which Pfizer expects to face another patent cliff.

In 2026 and 2027, the U.S. basic product patents on Prevnar 13, Eliquis and Xtandi are set to expire, potentially exposing products that generated sales of $6.3 billion in Pfizers home market last year to off-patent competition. All told, analysts expect Pfizer to lose up to $20 billion in sales due to patent expirations starting in 2026. Pfizers internal forecast is roughly in line with analyst estimates.

After Lyrica went off patent last year, Pfizer entered a window in which its portfolio is virtually free from the loss of exclusivity through to 2026. The lull gives Pfizer a period to get some of its pipeline prospects to market and start growing their sales, leading management to predict the current slate of experimental assets will at least replace the revenues lost to off-patent rivals starting in 2026.

That prediction rests on Pfizers ability to get key pipeline products to market. Pfizer used the virtual event to claim a growing effectiveness in that regard. As of 2015, 5% of the drugs Pfizer took into the clinic went on to win approval. By 2019, that figure had increased to 9%, causing Pfizers success rate to go from well below the industry average to slightly above the performance of its peers.

Pfizer also noted an improved phase 2 success rate. In 2017, 17% of Pfizers phase 2 trials succeeded. Since then, Pfizer has averaged 46%. The success rate so far this year stands at 53%. Pfizer claims to be moving faster, too, stating it is on course to reduce development timelines by 2.5 years between 2017 and 2021. Automation of processes accounts for the biggest anticipated time saving.

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Pfizer tips pipeline to add $15B to sales in coming years - FierceBiotech

TEL AVIV, Israel, Sept. 15, 2020 (GLOBE NEWSWIRE) -- VBL Therapeutics (Nasdaq: VBLT) will present today a corporate overview, including an update on the OVAL pivotal study, at the H. C. Wainwright 22nd Annual Global Investment Conference being held virtually on September 14-16, 2020.

"Our OVAL trial of VB-111 in ovarian cancer continues to progress well, with over a third of the study participants already enrolled," said Dror Harats, MD, Chief Executive Officer of VBL Therapeutics. "We are pleased that the high response rate seen in our interim analysis in March, continues to be high in the total patient population to date. With blinded data becoming more mature, we currently see a good correlation between the CA-125 and RECIST responses, as well as with preliminary PFS and OS data. So far, OVAL blinded data recapitulate what we have seen in our positive Phase 2 study, which is very encouraging."

Presentation Details:

About VBLVascular Biogenics Ltd., operating asVBL Therapeutics, is a clinical stage biopharmaceutical company focused on the discovery, development and commercialization of first-in-class treatments for areas of unmet need in cancer and immune/inflammatory indications. VBL has developed three platform technologies: a gene-therapy based technology for targeting newly formed blood vessels with focus on cancer, an antibody-based technology targeting MOSPD2 for anti-inflammatory and immuno-oncology applications, and the Lecinoxoids, a family of small-molecules for immune-related indications. VBLs lead oncology product candidate, ofranergene obadenovec (VB-111), is a first-in-class, targeted anti-cancer gene-therapy agent that is being developed to treat a wide range of solid tumors. It is conveniently administered as an IV infusion once every 6-8 weeks. It has been observed to be well-tolerated in >300 cancer patients and demonstrated activity signals in a VBL-sponsored all comers Phase 1 trial as well as in three VBL-sponsored tumor-specific Phase 2 studies. Ofranergene obadenovec is currently being studied in a VBL-sponsored Phase 3 potential registration trial for platinum-resistant ovarian cancer.

Forward Looking StatementsThis press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as anticipate, believe, could, estimate, expect, goal, intend, look forward to, may, plan, potential, predict, project, should, will, would and similar expressions. These forward-looking statements may include, but are not limited to, statements regarding our programs, including VB-111, including their clinical development, therapeutic potential and clinical results. These forward-looking statements are not promises or guarantees and involve substantial risks and uncertainties. Among the factors that could cause actual results to differ materially from those described or projected herein include uncertainties associated generally with research and development, clinical trials and related regulatory reviews and approvals, the risk that historical clinical trial results may not be predictive of future trial results, the impact of the COVID-19 pandemic on our business, operations, clinical trials, supply chain, strategy, goals and anticipated timelines and clinical results, that our financial resources do not last for as long as anticipated, and that we may not realize the expected benefits of our intellectual property protection. A further list and description of these risks, uncertainties and other risks can be found in our regulatory filings with theU.S. Securities and Exchange Commission, including in our annual report on Form 20-F for the year endedDecember 31, 2019, and subsequent filings with theSEC. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof.VBL Therapeuticsundertakes no obligation to update or revise the information contained in this press release, whether as a result of new information, future events or circumstances or otherwise.

INVESTOR CONTACT:Michael RiceLifeSci Advisors, LLC(646) 597-6979

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VBL Therapeutics to Provide an Update on the OVAL Study Today at the H. C. Wainwright 22nd Annual Global Investment Conference - GlobeNewswire

As the global COVID-19 situation is rapidly changing, staying abreast with the latest news can be challenging. In this article, Sheraz Gul provides an overview of the broad range of potential treatments in development and discusses how the regulatory landscape can shift at any time.

IN EARLY AUGUST 2020, the World Health Organization (WHO) reported the number of confirmed cases of COVID-19 has exceeded 20 million including over 700,000 deaths. These staggering figures have come about despite global lockdown measures being in place for the past few months. The resulting flattening of confirmed cases and deaths is now being jeopardised because, as the lockdown measures are being relaxed, we are seeing a resurgence of cases in some locations. In order to prevent a possible second wave of COVID-19 cases and deaths, the wearing of masks in public places is now mandatory in many countries. At the start of August 2020, New Zealand, which had gone 102 days without recording a locally transmitted case of COVID-19, saw new cases emerging. This highlights that we are still vulnerable to the spread of the virus.

Thus far, no US Food and Drug Administration (FDA)-approved therapy for use to treat COVID-19 is available. In order to provide some respite, on 1 May 2020 the FDA, as part of its Coronavirus Treatment Acceleration Program (CTAP), issued an Emergency Use Authorization (EUA) for the investigational antiviral drug remdesivir to treat suspected or laboratory-confirmed COVID-19 in adults and children hospitalised with severe disease. Although there is limited information known about the safety and effectiveness of using remdesivir to treat people in the hospital with COVID-19, this investigational drug has been shown in a clinical trial to shorten the time to recovery in some patients. It is noteworthy that prior to this, on 28 March 2020, the FDA also issued an EUA for chloroquine and hydroxychloroquine. This was subsequently revoked on 15 June 2020, as the FDA considered that chloroquine and hydroxychloroquine no longer met the statutory criteria for the EUA. In conclusion, the use and revocation of COVID-19 drugs can change at any time, so referring to official guidance is essential.

A recent update of the US ClinicalTrials. gov website listed around 2,000 COVID-19 interventional studies that are underway, around 90 of which are now completed. These cover a broad range of potential treatments including: 1) antiviral drugs to prevent viruses from multiplying; 2) immunomodulators aimed at reducing the bodys own immune reaction to the virus; 3) neutralising antibody therapies to fight the virus (manufactured, animalsourced and blood-derived from people who have previously had COVID-19); 4) cell therapy products (cellular immunotherapies and other types of both autologous and allogeneic cells, such as stem cells, and related products); and 5) gene therapy products that modify or manipulate the expression of a gene or alter the biological properties of living cells for therapeutic use. This diversity of therapeutic approaches is important as it allows opportunities for all types of treatments to be discovered, whilst expanding our understanding of the disease. Bearing in mind the timescale to discover a therapy for any given disease, the scientific community has made major advances towards developing a COVID-19 therapy. We eagerly await the output of various clinical trials over the next few months with the expectation that some of these yield sufficiently efficacious and safe therapies that gain approval by the regulators.

As the global COVID-19 situation is rapidly changing, it is advisable to stay abreast with the latest news only from reputable sources (see suggested reading below).

Sheraz Gul is an expert in drug discovery with experience gained in academia (University of London), industry (GlaxoSmithKline Pharmaceuticals) and the largest applied research organisation in Europe (Fraunhofer Institute). He is also an adjunct lecturer at NUI-Galway, Ireland and scientific co-founder of Transcriptogen Ltd. He has co-ordinated work packages in drug discovery projects, which have attracted more than 7 million funding and has organised 42 drug discovery workshops since 2011 across the globe and trained 780 scientists

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COVID-19 therapies in the pipeline - Drug Target Review

-Parkinsons Disease R&D Day to be hosted by Gavin Corcoran, MD, Chief R&D Officer, on October 30th, 2020, with leading movement disorder specialists

NEW YORK and BASEL, Switzerland, Sept. 08, 2020 (GLOBE NEWSWIRE) -- Axovant Gene Therapies Ltd (Nasdaq: AXGT), a clinical-stage company developing innovative gene therapies, today announced that the company will provide a corporate overview and participate in 1:1 meetings at upcoming virtual investor and patient conferences in September. Additionally, the company announced plans for their 1st Parkinsons Disease R&D Day with key opinion leaders.

Investor and Patient Conference Presentation details:

A live webcast of the presentations will be available in the Events section of Axovant's website at http://www.axovant.com. A replay will be available for approximately 30 days following the conference.

About Axovant Gene Therapies

Axovant Gene Therapies is a clinical-stage gene therapy company focused on developing a pipeline of innovative product candidates for debilitating neurodegenerative diseases. Our current pipeline of gene therapy candidates target GM1 gangliosidosis, GM2 gangliosidosis (also known as Tay-Sachs disease and Sandhoff disease), and Parkinsons disease. Axovant is focused on accelerating product candidates into and through clinical trials with a team of experts in gene therapy development and through external partnerships with leading gene therapy organizations. For more information, visit http://www.axovant.com.

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Parag MeswaniAxovant Gene Therapies Ltd.(212) 547-2523media@axovant.cominvestors@axovant.com

Source:Axovant Gene Therapies

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Axovant Announces First Parkinson's Disease Gene Therapy R&D Day and Participation in Upcoming September Conferences" - GlobeNewswire

One of the major challenges facing gene therapy a way to treat disease by replacing a patients defective genes with healthy ones is that it is difficult to safely deliver therapeutic genes to patients without the immune system destroying the gene, and the vehicle carrying it, which can trigger life-threatening widespread inflammation.

Three decades ago researchers thought that gene therapy would be the ultimate treatment for genetically inherited diseases like hemophilia, sickle cell anemia and genetic diseases of metabolism. But the technology couldnt dodge the immune response.

Since then, researchers have been looking for ways to perfect the technology and control immune responses to the gene or the vehicle. However, many of the strategies tested so far have not been completely successful in overcoming this hurdle.

Drugs that suppress the whole immune system, such as steroids, have been used to dampen the immune response when administering gene therapy. But its difficult to control when and where steroids work in the body, and they create unwanted side effects. My colleague Mo Ebrahimkhani and I wanted to tackle gene therapy with immune-suppressing tools that were easier to control.

I am a medical doctor and synthetic biologist interested in gene therapy because six years ago my father was diagnosed with pancreatic cancer. Pancreatic cancer is one of the deadliest forms of cancer, and the current available therapeutics usually fail to save patients. As a result, novel treatments such as gene therapy might be the only hope.

Yet, many gene therapies fail because patients either already have pre-existing immunity to the vehicle used to introduce the gene or develop one in the course of therapy. This problem has plagued the field for decades, preventing the widespread application of the technology.

Traditionally scientists use viruses from which dangerous disease-causing genes have been removed as vehicles to transport new genes to specific organs. These genes then produce a product that can compensate for the faulty genes that are inherited genetically. This is how gene therapy works.

Though there have been examples showing that gene therapy was helpful in some genetic diseases, they are still not perfect. Sometimes, a faulty gene is so big that you cant simply fit the healthy replacement in the viruses commonly used in gene therapy.

Another problem is that when the immune system sees a virus, it assumes that it is a disease-causing pathogen and launches an attack to fight it off by producing antibodies and immune response just as happens when people catch any other infectious viruses, like SARS-CoV-2 or the common cold.

Recently, though, with the rise of a gene editing technology called CRISPR, scientists can do gene therapy differently.

CRISPR can be used in many ways. In its primary role, it acts like a genetic surgeon with a sharp scalpel, enabling scientists to find a genetic defect and correct it within the native genome in desired cells of the organism. It can also repair more than one gene at a time.

Scientists can also use CRISPR to turn off a gene for a short period of time and then turn it back on, or vice versa, without permanently changing the letters of DNA that makes up or genome. This means that researchers like me can leverage CRISPR technology to revolutionise gene therapies in the coming decades.

But to use CRISPR for either of these functions, it still needs to be packaged into a virus to get it into the body. So some challenges, such as preventing the immune response to the gene therapy viruses, still need to be solved for CRISPR-based gene therapies.

Being trained as a synthetic biologist, I teamed up with Ebrahimkhani to use CRISPR to test whether we could shut down a gene that is responsible for immune response that destroys the gene therapy viruses. Then we investigated whether lowering the activity of the gene, and dulling the immune response, would allow the gene therapy viruses to be more effective.

CRISPR can precisely remove even single units of DNA. KEITH CHAMBERS/SCIENCE PHOTO LIBRARY/Getty Images

A gene called Myd88 is a key gene in the immune system and controls the response to bacteria and viruses, including the common gene therapy viruses. We decided to temporarily turn off this gene in the whole body of lab animals.

We injected animals with a collection of the CRISPR molecules that targeted the Myd88 gene and looked to see whether this reduced the quantity of antibodies that were produced to specifically fight our gene therapy viruses. We were excited to see that the animals that received our treatment using CRISPR produced less antibody against the virus.

This prompted us to ask what happens if we give the animal a second dose of the gene therapy virus. Usually the immune response against a gene therapy virus prevents the therapy from being administered multiple times. Thats because after the first dose, the immune system has seen the virus, and on the second dose, antibodies swiftly attack and destroy the virus before it can deliver its cargo.

We saw that animals receiving more than one dose did not show an increase in antibodies against the virus. And, in some cases, the effect of gene therapy improved compared with the animals in which we had not paused the Myd88 gene.

We also did a number of other experiments that proved that tweaking the Myd88 gene can be useful in fighting off other sources of inflammation. That could be useful in diseases like sepsis and even COVID-19.

While we are now beginning to improve this strategy in terms of controlling the activity of the Myd88 gene. Our results, now published in Nature Cell Biology,provide a path forward to program our immune system during gene therapies and other inflammatory responses using the CRISPR technology.

Samira Kiani, Associate Professor of Pathology, University of Pittsburgh

This article is republished from The Conversation under a Creative Commons license. Read the original article.

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CRISPR Can Help Combat the Troubling Immune Response Against Gene Therapy - Gizmodo Australia

New Jersey, United States, The Gene Therapy Market report 2020 provides a detailed impression, describe the product industry scope and the market expanded insights and forecasts up to 2027. It shows market data according to industry drivers, restraints and opportunities, analyzes the market status, the industry share, size, future Trends and growth rate of the market. The Gene Therapy Market report is categorized by application, end user, technology, product / service types, and other, as well as by region. In addition, the report includes the calculated expected CAGR of chitosan acetate-market derivative from the earlier records of the Gene Therapy Market, and current market trends, which are organized with future developments.

Gene Therapy Market was valued at USD 3.69 Billion in 2019 and is projected to reach USD 24.78 Billion by 2027, growing at a CAGR of 26.9% from 2020 to 2027.

Gene Therapy Market, By Product

Viral Vectorso Adeno-associated virus vectorso Retroviral vectors Gammaretroviral vectors Lentiviral vectorso Other viral vectors (herpes simplex and adenoviral vectors) Non-viral Vectorso Oligonucleotideso Other non-viral vectors (plasmids and RNAi)

Gene Therapy Market, By Indication

Neurological Diseases Cancer Hepatological Diseases Duchenne Muscular Dystrophy Other Indications

Gene Therapy Market, By Delivery Method

Ex vivo In vivo

The report provides detailed coverage of the Gene Therapy Market, including structure, definitions, applications, and Industry Chain classifications. The Gene Therapy Market analysis is provided for the international markets including development trends, competitive landscape analysis, investment plan, business strategy, opportunities and development status of key regions. Development policies and plans are discussed and manufacturing processes and cost structures analyzed. This report also includes information on import / export consumption, supply and demand, costs, industry share, policy, Price, Sales and gross margins.

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Gene Therapy Market forecast up to 2027, with information such as company profiles, product picture and specification, capacity production, price, cost, revenue, and contact information. Upstream raw materials and equipment as well as downstream demand analyses are also carried out. The Gene Therapy Market size, development trends and marketing channels are analyzed. Finally, the feasibility of new investment projects is assessed and general research results are offered.

The Gene Therapy Market was created on the basis of an in-depth market analysis with contributions from industry experts. The report covers the growth prospects in the coming years and the discussion of the main providers.

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Gene Therapy Market is Thriving Worldwide 2020 | Trends, Growth and Profit Analysis, Forecast by 2027 - The Daily Chronicle

The report covers the forecast and analysis of the gene therapy market on a global and regional level. The study provides historical data from 2015 to 2018 along with a forecast from 2019 to 2027 based on revenue (USD Million). The study includes drivers and restraints of the gene therapy market along with the impact they have on the demand over the forecast period. Additionally, the report includes the study of opportunities available in the gene therapy market on a global level.

In order to give the users of this report a comprehensive view of the gene therapy market, we have included a competitive landscape and an analysis of Porters Five Forces model for the market. The study encompasses a market attractiveness analysis, wherein all the segments are bench marked based on their market size, growth rate, and general attractiveness.

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The report provides company market share analysis to give a broader overview of the key players in the market. In addition, the report also covers key strategic developments of the market including acquisitions & mergers, new service launches, agreements, partnerships, collaborations & joint ventures, research & development, and regional expansion of major participants involved in the market on a global and regional basis.

The study provides a decisive view of the gene therapy market by segmenting the market based on the type, vector type, therapy area, and regions. All the segments have been analyzed based on present and future trends and the market is estimated from 2019 to 2027. The regional segmentation includes the current and forecast demand for North America, Europe, Asia Pacific, Latin America, and the Middle East and Africa.

Gene therapy is utilized for treating neurodegenerative disorders like Alzheimer, amyotrophic lateral sclerosis, and spinal muscular atrophy. Gene therapy is one of the key treatment kinds that will propel the market growth over the forecast period. Moreover, gene therapy also finds lucrative applications in precision medicine. In addition to this, a rise in the occurrence of cancer is prompting the demand to treat the disease through gene therapy.

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Based on the type, the market can be segregated into Germ Line Gene Therapy and Somatic Gene Therapy. In terms of vector type, the gene therapy industry can be divided into Viral Vectors, Non-Viral Vectors, and Human Artificial Chromosome. On the basis of therapy area, the market for gene therapy can be classified into Cancer, Neurological Diseases, Infectious Diseases, Genetic Disorders, Rheumatoid Arthritis, and Others.

The key players included in this market are Advanced Cell & Gene Therapy, Audentes Therapeutics, Benitec Biopharma, Biogen, Blubird Bio, Inc., Bristol-Myers Squibb Company, CHIESI Farmaceutici SPA, Eurofins Scientific, Geneta Science, Genzyme Corporation, Gilead, GlaxoSmithKline PLC, Human Stem Cells institute, Novartis AG, Orchard Therapeutics, Pfizer Inc., Sangamo therapeutics, Spark therapeutics, and Voyager Therapeutics.

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Gene Therapy Market By Industry Type, By Brand And Major Players 2020-2027 - The Daily Chronicle