Category Archives: Gene Medicine

Posted 18 June 2020 | By Michael Mezher

Officials from the US Food and Drug Administration (FDA) discussed the agencys recent efforts to support the development of products to treat rare diseases during a session at DIAs Global Annual Meeting on Wednesday.Orphan and rare pediatric disease designationsWhile the number of products approved to treat rare diseases has increased over the last decade, the vast majority of rare diseases lack approved treatment options.Janet Maynard, director of FDAs Office of Orphan Products Development, gave an update on FDAs orphan drug designation program. According to Maynard, orphan drug designation requests have stabilized at just over 500 requests per year since 2016. The number of designations granted have also remained stable, in the mid-300s since 2015, with a spike to 477 in 2017.Maynard also discussed FDAs rare pediatric disease designation and priority review voucher program, which is set to sunset later this year without legislative action.Between 2013 and 2016 we saw a steady increase in terms of the number of rare pediatric disease designations requests we have received, and that number has remained greater than 50 since 2016, she said. FDA has granted more than 50 rare pediatric disease designations each year since 2017.However, Maynard pointed out that, After 30 September 2020, FDA may only award a voucher for an approved rare pediatric disease product application if the sponsor has rare pediatric disease designation for the drug and that designation was granted by 30 September 2020, and that the agency will no longer issue vouchers regardless of designation after 30 September 2022.Companies looking to get in under the wire will need to submit requests for rare pediatric disease designation soon, Maynard said. FDA guarantees a 60-day review for rare pediatric disease designation requests issued alongside a fast track or orphan designation request but does not commit to a specific timeline for requests submitted separately.The Offices of Orphan Product Development and Pediatric Therapeutics cannot commit to providing a response to rare pediatric disease designation requests by 30 September 2020 for any requests received after 31 July 2020, Maynard said.OND reorganization and rare disease hubAfter undergoing a major reorganization earlier this year, FDAs Office of New Drugs (OND) is better equipped to address rare disease drug development, said Hylton Joffe, acting director of the new Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine.Joffes office oversees three clinical divisions, the Division of Pediatrics and Maternal Health, the Division of Rare Diseases and Medical Genetics and the Division of Urology, Obstetrics and Gynecology.You can imagine, given the significant burden of rare diseases in children, how having these three divisions together could lead to very strong collaborations in the rare disease space, Joffe said.Joffe pointed out that the newly created Division of Rare Diseases and Medical Genetics, which oversees the rare disease group and inborn errors review group, is the only division within OND that is solely focused on rare diseases. While other offices within OND also deal with rare diseases, We see this new division and this office as the new OND rare disease hub, Joffe said, adding that the goal of the hub will be to enhance communication and consistency for rare diseases.Gene therapiesFollowing the approval of the first few gene therapies, Elizabeth Hart, branch chief at the Office of Tissues and Advanced Therapies within the Center of Biologics Evaluation and Research, said expectations are high for future gene therapies to treat rare diseases.Hart said that the number of gene therapies in development has increased significantly in recent years, with investigational new drug applications (INDs) for gene therapies increasing more than 140% between 2016 and 2019. According to Hart, 70% of gene therapy INDs are targeting rare diseases.While the approval standard for gene therapies and other rare diseases is the same as for other drugs, Hart said that the clinical development plans for those products could look very different from those for more common conditions.We do have the option to exert regulatory flexibility, and this is especially important for rare diseases. Clinical development programs for the different diseases may vary substantially, and so they really need to be individualized, she said.Hart also said that, There are no specific minimum number of patients to be studied to establish effectiveness and safety of a gene therapy, the number of subjects needed is determined on a case by case basis taking into consideration the persuasiveness of the data, including comprehensiveness and quality; the nature of the benefit provided; the length of the treatment exposure, the patient population that would be treated after marketing approval; and concerns for potential harm from the treatment.

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FDA officials update on orphan drugs, gene therapies at DIA - Regulatory Focus

ST. LOUIS, June 18, 2020 /PRNewswire/ -- OpenCell Technologies, an R&D-stage biomedical venture, has announced the hiring of Kevin Gutshall as CEO. Kevin leaves his role as MilliporeSigma's director of life science business development and M&A focusing on the cell and gene therapy business unit, to join OpenCell and lead its efforts to translate and commercialize its core technology platform, POROS.

OpenCell was established based on technology developed at the Georgia Institute of Technology, by Engineering faculty and company co-founders Mark Meacham, PhD, Andrei Fedorov, PhD, and Levent Degertekin, PhD. Dr. Meacham was subsequently recruited to Washington University in St. Louis, and OpenCell relocated to the BioGenerator Labs in the Cortex Innovation Community adjacent to the Washington University School of Medicine. The company's core technology, which has broad applications ranging from fuel and energy to life sciences, is focused on the rapidly emerging cell and gene therapy market.

"I am thrilled to join OpenCell, as it is now poised to move from an R&D stage to a commercial business," said Gutshall. "I believe that the POROS platform will be a disruptive technology platform in the cell and gene therapy marketplace."

During its seed stage, the company benefitted from BioGenerator Entrepreneurs-in-Residence (EIR) that brought key expertise to the company as it pivoted from the research tools market to cell and gene therapy applications. Paul Olivo, MD, PhD, a former BioGenerator EIR and current Venture Partner at Synchrony Bio, which also participated in the current financing, serves as a key advisor to OpenCell, managing the company's research team. In her role as BioGenerator EIR, Heather Holeman, PhD, now CEO of Lifespan Biosciences, facilitated key business development connections for the company. Concurrent with the financing, Charlie Bolten, Senior Vice President of BioGenerator, joins OpenCell's board of directors.

"Together with Synchrony Bio, BioGenerator's investment in OpenCell is the culmination of extensive due diligence and hands-on support by our investment, Entrepreneur-in-Residence and Grants-2-Business teams," said Bolten. "With the successful recruitment of a CEO with deep experience in commercialization, business development and M&A, we are pleased to see OpenCell take an important step toward commercializing the POROS platform."

"I am excited to welcome Kevin as the new CEO of OpenCell," added Chad Stiening, OpenCell Executive Chairman and Managing Director at Synchrony Bio. "His professional background and personal passion in the cell and gene therapy space is a perfect fit for the company as it seeks to realize the full potential of its technology and enable the development and manufacturing of promising new therapies in this dynamic market."

In addition to investments from BioGenerator and Synchrony Bio, the company has leveraged significant Federal grant funding over $3M total to help secure equity financing and achieve key milestones that helped attract strategic partnering interest as well as its new CEO.

About OpenCellOpenCell Technologies provides efficient, high-throughput and scalable transfection tools to the Life Science Industry, enabling it to use difficult-to-transfect cells (e.g., primary and cancer stem cell cultures) in development of cell-based analysis techniques and discovery of new therapeutic cell-based therapies. Unlike currently available products, the OpenCell technology features precise control of biophysical actions on a single-cell basis without sacrificing throughput. OpenCell's vision is to realize a novel, cost-effective approach to transfection that will overcome existing research and development obstacles.Our mission is to make cellular therapies effective, affordable and scalable for the clinical and research communities. Visit opencelltech.com for more information.

About BioGenerator BioGenerator, the investment arm of BioSTL, produces a sustained pipeline of successful bioscience companies and entrepreneurs in St.Louis by creating, growing and investing in promising new enterprises. Visit biogenerator.org for additional information, and follow us on LinkedIn and Twitter.

About Synchrony BioSynchrony Bio seeks to achieve consistently superior investor returns in early-stage biomedical and life science ventures by aligning seasoned talent, staged investment capital, and process efficiencies.Careful and coordinated alignment of all three is key to overcoming unique challenges faced by medical device and diagnostics ventures, in order to realize significant upside and superior returns.Synchrony's extended network of experts and advisors includes professionals with deep, cross-functional experience and backgrounds. Visit synchronybio.com for additional information.

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http://www.biostl.org

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Cell and gene therapy-focused OpenCell Technologies hires MilliporeSigma executive Kevin Gutshall as new CEO - PRNewswire

]]> Giroctocogene fitelparvovec is being developed as part of an agreement between Sangamo and Pfizer for the global development of gene therapies for hemophilia A. Credit: Coolcaesar.

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Pfizer and Sangamo Therapeutics have reported positive follow-up data from the Phase I/II Alta study of giroctocogene fitelparvovec (PF-07055480) to treat patients with severe hemophilia A.

The investigational gene therapy Giroctocogene fitelparvovec consists of a recombinant adeno-associated virus serotype 6 vector (AAV6) encoding the complementary deoxyribonucleic acid for B domain deleted human FVIII.

The open-label, dose-ranging, multi-centre Alta clinical trial has been designed to assess the safety and tolerability of the therapy in severe hemophilia A patients.

Across four dose cohorts, the mean age of the 11 patients assessed is 30 years and all are male.

All five patients with severe hemophilia A were given the 3e13 vg/kg dose and they showed sustained factor VIII (FVIII) activity levels, with a median of 64.2% via chromogenic assay.

No patients experienced bleeding events or required FVIII infusions during the trial.

Pfizer Rare Disease Research Unit senior vice-president and chief scientific officer Seng Cheng said: We are excited that these data affirm previous findings from this Phase I/II study, and that all five patients have sustained levels of factor VIII activity with no bleeding events or use of factor replacement therapy.

The Phase III lead in study is ongoing, and we look forward to dosing patients with this investigational gene therapy in the pivotal Phase III trial later this year.

Giroctocogene fitelparvovec received orphan drug, fast track, and regenerative medicine advanced therapy (RMAT) designations from the US Food and Drug Administration and Orphan Medicinal Product designation from the European Medicines Agency.

The therapy is being developed as part of a collaboration agreement between Sangamo and Pfizer for the global development and commercialisation of gene therapies for hemophilia A.

Sangamo chief medical officer Bettina Cockroft said: These follow-up data indicate that treatment with giroctocogene fitelparvovec resulted in sustained factor levels up to 14 months following treatment and suggests the potential of this investigational gene therapy to alleviate the treatment burden of current hemophilia disease management.

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Pfizer and Sangamo report positive data from hemophilia A therapy trial - Clinical Trials Arena

Overview: Regenerative medicine is an interdisciplinary field that applies life science and engineering principles for the regeneration or repair of injured/diseased tissues or organs resulting from various causes including, disease, defects, trauma and aging. The field includes the generation and use of tissue engineering, therapeutic stem cells and the production of artificial organs. It also allows scientists to grow organs or tissues in the lab and implant them in the body safely when the body fails to heal itself. Notably, it has great potential to solve the problem of organ shortage.

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According to the estimation of World Health Organization, there is an increasing prevalence of diabetes among adults over the age of 18 years, that has increased to 8.5% in 2014 compared to 4.5% in 1980 across the globe. As per the estimation of Arthritis Foundation, the number of people expected to be diagnosed from arthritis will be more than 78 million, by 2040.

The market for regenerative medicine is driven by increasing prevalence of neuronal disease, cancer and genetic disease, emerging application of regenerative medicine, and advancement in technology. Huge number of ongoing clinical trails and strong product pipeline are providing market growth oppurtunity. High cost of the treatment, regulatory issues and ethical issuesare hampering the market growth.

Market Analysis: The Global Regenerative Medicine market is estimated to witness a CAGR of 16.6% during the forecast period 20182024. The global market is analyzed based on three segments Therapy, Application and regions.

Regional Analysis: The regions covered in the report are the North America, Europe, Asia Pacific, and Rest of the World (ROW). North America is the major shareholder in the global regenerative medicine market, followed by Europe. Asia-Pacific region is expected to have the fastest growth rate with the market growth centered at Japan, China and India. This is mainly due to increasing funding in healthcare research, rising research activities, growing patient pool, flexible regulatory environment for clinical trials, and rising healthcare expenditure.

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Therapy Analysis:Immunotherapy occupied major market share of global regenerative medicine market in 2017, and is expected to remain same during the forecasted period. Increasing product approvals, emerging technological advancements in cell and gene therapy, flexible regulatory for stem cell based research, and growing awareness regarding the benefits of stem cell therapies.

Application Analysis: The market by application is segmented into cancer, central nervous system, orthopedic and musculoskeletal, diabetes, dermatology, cardiovascular and others. Among various application, dermatology occupied the largest share in 2017 and cancer segment is expected to grow at fastest rate during the forecasted period. Growing aging population, changing lifestyle, increasing disease prevalence makes cancer, the fastest growing application segment during the forecasted period.

Key Players: Allergan plc, Integra lifesciences, Mimedx Group, Inc., Medtronic plc, Organogenesis Inc., Zimmer Biomet, Acelity L.P. Inc., Nuvasive, Inc., Stryker Corporation, Japan Tissue Engineering Co., Ltd. (Fujifilm Holdings Corporation subsidiary), Osiris Therapeutics, Inc., Vericel Corporationand other predominate and niche players.

Competitive Analysis: Currently dermatology segment dominates the global regenerative medicine segment. A lot of researches are going on cancer, CNS, cardiovascular, orthopedic & musculoskeletal applications. The increasing importance of regenerative medicine has resulted in the launch of new products and also increased acquisition, approvals, funding to develop new product.

For instance, in August 2017, Tissue Regenix Group plc completed the acquisition of acquisition of CellRight Technologies, an US based specialist in regenerative osteoinductive bone scaffolds. In April 2018, Roche acquired a program named Inception 5, focused on regenerative therapies for multiple sclerosis. In May 2018, Novartis received second FDA approval for Kymriah, CAR-T cell therapy for B-cell acute lymphoblastic leukemia (ALL)

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Benefits: The report provides complete details about the usage and adoption rate of regenerative medicine in various therapeutic verticals and regions. With that, key stakeholders can know about the major trends, drivers, investments, and vertical players initiatives. Moreover, the report provides details about the major challenges that are going to impact on the market growth. Additionally, the report gives the complete details about the key business opportunities to key stakeholders to expand their business and capture the revenue in the specific verticals to analyze before investing or expanding the business in this market.

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Regenerative Medicine Market to Record a Robust Growth Rate for the COVID-19 Period - Cole of Duty

UCLA researchers and colleagues have received a $13.65 million grant from the National Institutes of Health to investigate and further develop an immunotherapy known as CAR T, which uses genetically modified stem cells to target and destroy HIV.

The five-year grant, part of an NIH effort to develop gene-engineering technologies to cure HIV/AIDS, will fund a collaboration among UCLA; CSL-Behring, a biotechnology company in the United States and Australia; and the University of WashingtonFred Hutchinson Cancer Research Center.

Scott Kitchen, an associate professor of medicine in the division of hematology and oncology, and Irvin Chen, director of the UCLA AIDS Instituteat the David Geffen School of Medicine at UCLA, are leading the effort. The project will build on their previous research using CAR T therapy to combat the virus, which is constantly mutating and difficult to beat.

The overarching goal of our proposed studies is to identify a newgene therapy strategy to safely and effectively modify a patients own stem cells to resist HIV infection andsimultaneously enhance their ability to recognize and destroy infected cells in the body in hopes of curing HIV infection, said Kitchen, who also directs the humanized mouse core laboratory for UCLAs Center for AIDS Research and Jonsson Comprehensive Cancer Center.It is a huge boost to our efforts at UCLA and elsewhere to find a creative strategy to defeat HIV.

The only known cure of an HIV-infected person was announced in 2008. The famous Berlin patient received a stem cell transplant from a donor whose cells naturally lacked a crucial receptor that HIV binds to in order to kill cells and destroy the immune system. The main problems with this approach, the researchers say, are that the donor and recipient have to be highly matched often a rare event and that it often fails to produce a sufficient amount of HIV-protected cells that can clear the virus from the body.

Transplantation of blood-forming stem cells has been the only treatment strategy that has resulted ina functional cure for HIV infection, Kitchen said. Over 13 years after the first successfully cured HIV-infected patient, there is a substantial need to develop strategies that are capable of being used on everyone with HIV infection.

One of those strategies, CAR T, has been the subject of ongoing research at UCLA by Chen, Kitchen and others. This approach involves genetically engineering a patients own blood-forming stem cells to carry genes for chimeric antigen receptors, or CARs. Once these stem cells are modified and transplanted back into the patient, they form specialized infection-fighting white blood cells known as T cells in this case, CAR T cells that specifically seek out and kill HIV-infected cells. In a recent study, the UCLA scientists found that engineered CAR T cells not only destroyed infected cells but also lived for more than two years the length of the study.

The thinking behind the NIH-funded project, the researchers say, is that a combination of CARs and broadly neutralizing antibodies may be a long-lasting, perhaps permanent, cure for HIV.

Our work under the NIH grant will provide a great deal of insight into ways the immune response can be modified to better fight HIV infection, said Chen, who is a professor of medicine and of microbiology, immunology and molecular genetics at the Geffen School of Medicine. The development of this unique strategy that allows the body to develop multiple ways to attack HIV could have an impact on other diseases as well, including the development of similar approaches targeting other types of chronic viral infections and cancers.

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UCLA receives nearly $14 million from NIH to investigate gene therapy to combat HIV - UCLA Newsroom

Houston has played a significant role in boosting the nations biotech industry. While Houston is still a hotspot for energy and oil, the city is steadily becoming a burgeoning life sciences hub. In fact, the city boasted the third fastest-growing biotech community in the nation between 2014 and 2017, according to a CBRE report. Houstons biotech industry is gaining momentum due to an increase in funding as well. According to the Greater Houston Partnership, nearly $180 million in VC funding was allocated to the citys ecosystem of life sciences-related companies in 2019 alone.

Like many startups and tech companies across Houston, the citys life sciences leaders have been tackling some of the worlds most pressing issues. Whether theyre developing oncology drug candidates or advancing genomic medicine through the creation of sequencing technologies, the citys biotech organizations are pulling on decades of research and determination to transform the medical landscape on a global scale. Heres a look at 15 biotech companies in Houston making a major impact on medical research and discovery.

Founded: 2015

Focus: Canine Cancer Treatment

What they do:CAVU Biotherapiesprovides immune-based solutions to treat cancer and autoimmune diseases in dogs. The company offers an immune health monitoring service, which describes a dogs immune system through the use of a blood sample, as well as an autologous prescription product that retrains and expands a dogs T cells to recognize and fight cancer. CAVU Biotherapies ultimate aim is to use its immune-guided medicine to treat horses, cats, andeventually, humans.

Founded: 2006

Focus: Stem Cell Banking + Therapy

What they do: Founded by David Eller and Dr. Stanley Jones, Celltex Therapeutics focuses on developing stem cell therapies for a variety of conditions. The companys stem cell processing and banking methods are designed to ensure the genetic integrity and uniformity of an individuals cells in quantities necessary for therapeutic applications. Using proprietary technology, Celltex Therapeutics enables stem cells to be used for regenerative therapy for conditions like vascular, autoimmune and degenerative diseases.

Founded: 2006

Focus: Cell Therapy

What they do: InGeneron is a clinical stage cell therapy company that specializes in novel, evidence-based regenerative medicine therapies. The companys therapy is designed to repair injured tissue, improve the quality of life for patients and modify the progression of their disease. InGeneron focuses mainly on musculoskeletal indications such as pain management.

Founded: 2006

Focus: Cancer Treatment

What they do: Moleculin Biotech is a pharmaceutical company dedicated to the treatment of highly resistant cancers and viruses. The company develops oncology drug candidates for highly resistant tumors as well as as prodrug to exploit the potential uses of inhibitors of glycolysis. Guided by the aim to provide new hope to cancer patients, Moleculin Biotech focuses on discovering new treatments for acute myeloid leukemia, skin cancer, pancreatic cancer and brain tumors.

Founded: 2001

Focus: Nanomedicine

What they do: Nanospectra Biosciences is spearheading a patient-centric use of nanomedicine for the removal of cancerous tissues. The companys ultra-focal nanoshell technology is designed to thermally destroy solid tumors without damaging adjacent healthy tissue. Nanospectra Biosciences aims to maximize treatment efficacy while minimizing side effects associated with surgery, radiation and traditional focal therapies.

Founded: 2018

Focus: Cell Therapy

What they do: Marker Therapeutics is an immuno-oncology company that focuses on the development of next-generation T cell-based immunotherapies. With the aim of treating hematological malignancies and solid tumor indications, the company uses its own MultiTAA T cell technology, which is based on the selective expansion of non-engineered, tumor-specific T cells. Marker Therapeutics is also working on developing proprietary DNA expression technology that is intended to improve the cellular immune systems ability to recognize and destroy diseased cells.

Founded: 2008

Focus: 3D Cell Culture

What they do: Nano3D Biosciences is dedicated to the development of 3D cell culture solutions. The companys core technology allows them to levitate or bioprint cells, which results in the formation of cultures that are more easily assembled and handled. Nano3D Biosciences products and services are intended for biomedical research, drug discovery, precision medicine, toxicology and regenerative medicine.

Founded: 2017

Focus: Small Molecule Inhibitors

What they do: Tvardi Therapeutics is a clinical-stage biotech company working on a new class of medicines for cancer, chronic inflammation and fibrosis. The company is focusing on the creation of orally delivered, small molecule inhibitors of STAT3, which is a key regulatory protein positioned at the intersection of many disease pathways. Tvardi Therapeutics is dedicated to delivering safe and effective solutions for use in the treatment of numerous diseases.

Founded: 2011

Focus: Targeted Cancer Therapies

What they do: Salarius Pharmaceuticals focuses on developing targeted therapies to treat various types of cancers. The companys lead candidate, Seclidemstat, is intended to treat Ewing sarcoma, a pediatric and young adult bone cancer that currently lacks targeted therapies. Salarius Pharmaceuticals performs clinical trials for the treatment of other advanced solid tumors including prostate, breast and ovarian cancers.

Founded: 2013

Focus: Genomic Medicine

What they do: Founded by Michael Metzker, RedVault Biosciences develops technologies with the aim of advancing genomic medicine. The company is currently working on a variety of projects including the development of sequencing technologies to determine haplotypes and structural variation in complex genomes. RedVault Biosciences is dedicated to identifying technology needs, creating and testing ideas, and transferring deliverables to production and distribution.

Founded: 2010

Focus: DNA Sequencing

What they do: Avance Biosciences focuses on assay development, assay validation and sample testing using next-generation DNA sequencing and other biological methods. The company offers biologics testing, diagnostic assay validation, GMO genomic testing, gene / cell therapy testing, digital and real-time PCR, microbial testing and more. Avance Biosciences aim is to assist its clients in advancing drug development and genomic research.

Founded: 2008

Focus: Bioremediation

What they do: Bionex Technology develops cost-effective, natural solutions for cleaning oil-polluted soil. The companys Super Microbe spill solution is naturally derived from microbes that digest and convert harmful contaminants on the ground and in soil, therefore lowering flammability, suppressing harmful vapors and creating a safer environment for spill responders. Bionex Technology offers a variety of other bioremediation products such as a customizable degreaser and detergent used for cleaning industrial tools.

Founded: 2016

Focus: Stem Cell Research

What they do: Located in nearby Sugar Land, Hope Biosciences is dedicated to developing stem cell-based therapies that are safe, effective and secure. The companys proprietary technology enables patients to make virtually unlimited and identical stem cells from their own tissue. Hope Biosciences offers stem cell banking solutions for both adults and newborns.

Founded: 2013

Focus: Interventional Cardiology

What they do: Saranas has created technology that enables the early detection and monitoring of bleeding complications associated with vascular access procedures. The companys monitoring system checks changes in the blood vessels electrical resistance before monitoring if bleeding has occurred from an unintentionally injured blood vessel. Saranas aims to allow physicians to mitigate downstream consequences by addressing bleeds before they become complications.

Founded: 1984

Focus: Microbiology

What they do: Microbiology Specialists Inc. specializes in microbiology testing, playing a role in microbial investigations and studies. The company also focuses on infectious disease diagnosis, forensic bacteriology and mycology, medical device testing and infection prevention. Microbiology Specialists Inc. is committed to delivering reliable, accurate and cost-effective microbiological results.

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15 Biotech Companies In Houston To Know - Built In

NEW YORK, June 19, 2020 (GLOBE NEWSWIRE) --Ovid Therapeutics Inc.(NASDAQ: OVID), a biopharmaceutical company committed to developing medicines that transform the lives of people with rare neurological diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation to OV101 (gaboxadol) for the treatment of Angelman syndrome. OV101 is believed to be the only delta ()-selective GABAA receptor agonist in development and is currently being evaluated in the Companys pivotal Phase 3 NEPTUNE trial in Angelman syndrome, with topline results expected in the fourth quarter of 2020. The FDA has previously granted Orphan Drug and Fast Track designations for OV101 for the treatment of Angelman syndrome.

Under the Creating Hope Act passed into federal law in 2012, the FDA grants Rare Pediatric Disease Designation for serious and life-threatening diseases that primarily affect children ages 18 years or younger and fewer than 200,000 people in the U.S. If a new drug application (NDA) for OV101 in Angelman syndrome is approved, Ovid may be eligible to receive a priority review voucher from the FDA, which can be redeemed to obtain priority review for any subsequent marketing application or may be transferred and/or sold to other companies for their programs, such as has recently been done by other voucher recipients.

OV101 has the potential to become the first FDA-approved therapy for individuals living with Angelman syndrome. Receiving Rare Pediatric Disease Designation from the FDA is a significant milestone for this program and underscores the critical value of our work, said Amit Rakhit, M.D., MBA, President and Chief Medical Officer of Ovid Therapeutics. Importantly, with this designation, we may be eligible to receive a priority review voucher from the FDA, providing significant value as we work diligently towards the completion of our ongoing pivotal Phase 3 NEPTUNE trial. We are grateful to the FDA and Congress for having enacted this law which helps Ovid and all companies developing innovative drugs for rare pediatric conditions.

About Angelman SyndromeAngelman syndrome is a rare genetic condition that is characterized by a variety of signs and symptoms. Characteristic features of this condition include delayed development, intellectual disability, severe speech impairment, problems with movement and balance, seizures, sleep disorders and anxiety. The most common cause of Angelman syndrome is the loss of function of the gene that codes for ubiquitin protein ligase E3A (UBE3A), which plays a critical role in nerve cell communication, resulting in impaired tonic inhibition. Individuals with Angelman syndrome typically have normal lifespans but are unable to live independently. Therefore, they require constant support from a network of specialists and caregivers. Angelman syndrome affects approximately 1 in 12,000 to 1 in 20,000 people globally.

There are no approved therapies by the FDA, European Medicines Agency (EMA), or rest of world for Angelman syndrome, and treatment primarily consists of behavioral interventions and pharmacologic management of symptoms.

Angelman syndrome is associated with a reduction in tonic inhibition, a function of the delta ()-selective GABAA receptor that allows a human brain to decipher excitatory and inhibitory neurological signals correctly without being overloaded. If tonic inhibition is reduced, the brain becomes inundated with signals and loses the ability to separate background noise from critical information.

About OV101 (gaboxadol)OV101 is believed to be the only delta ()-selective GABAA receptor agonist in development and the first investigational drug to specifically target the disruption of tonic inhibition, a central physiological process of the brain that is thought to be the underlying cause of certain neurodevelopmental disorders. OV101 has demonstrated in laboratory studies and animal models to selectively activate the -subunit of GABAA receptors, which are found in the extrasynaptic space (outside of the synapse), and thereby impact neuronal activity through modulation of tonic inhibition.

Ovid is developing OV101 for the treatment of Angelman syndrome and Fragile X syndrome to potentially restore tonic inhibition and thereby address several core symptoms of these conditions. In both these syndromes, the underlying pathophysiology includes disruption of tonic inhibition modulated through the -subunit of GABAA receptors. In preclinical studies, it was observed that OV101 improved symptoms of Angelman syndrome and Fragile X syndrome. This compound has also previously been tested in more than 4,000 patients (more than 1,000 patient-years of exposure) and was observed to have favorable safety and bioavailability profiles. Ovid is conducting a pivotal Phase 3 clinical trial with OV101 in Angelman syndrome (NEPTUNE) and has completed a Phase 2 signal-finding clinical trial with OV101 in Fragile X syndrome (ROCKET).

OV101 has received Rare Pediatric Disease Designation from the FDA for the treatment of Angelman syndrome. The FDA has also granted Orphan Drug and Fast Track designations for OV101 for both the treatment of Angelman syndrome and Fragile X syndrome. In addition, the European Commission (EC) has granted orphan drug designation to OV101 for the treatment of Angelman syndrome. The U.S. Patent and Trademark Office has granted Ovid patents directed to methods of treating Angelman syndrome and Fragile X syndrome using OV101. The issued patents expire in 2035 without regulatory extensions.

About Ovid TherapeuticsOvid Therapeutics Inc. is a New York-based biopharmaceutical company using its BoldMedicine approach to develop medicines that transform the lives of patients with rare neurological disorders. Ovid has a broad pipeline of potential first-in-class medicines. The Companys most advanced investigational medicine, OV101 (gaboxadol), is currently in clinical development for the treatment of Angelman syndrome and Fragile X syndrome. Ovid is also developing OV935 (soticlestat) in collaboration with Takeda Pharmaceutical Company Limited for the potential treatment of rare developmental and epileptic encephalopathies (DEE). For more information on Ovid, please visit http://www.ovidrx.com/.

Forward-Looking StatementsThis press release includes certain disclosures that contain forward-looking statements, including, without limitation, statements regarding: advancing and commercializing Ovids product candidates, progress, timing, scope and the development and potential benefits of Ovids product candidates; and the anticipated reporting schedule of clinical data regarding Ovids product candidates. You can identify forward-looking statements because they contain words such as will, appears, believes and expects. Forward-looking statements are based on Ovids current expectations and assumptions. Because forward-looking statements relate to the future, they are subject to inherent uncertainties, risks and changes in circumstances that may differ materially from those contemplated by the forward-looking statements, which are neither statements of historical fact nor guarantees or assurances of future performance. Important factors that could cause actual results to differ materially from those in the forward-looking statements include uncertainties in the development and regulatory approval processes, and the fact that initial data from clinical trials may not be indicative, and are not guarantees, of the final results of the clinical trials and are subject to the risk that one or more of the clinical outcomes may materially change as patient enrollment continues and/or more patient data become available. Additional risks that could cause actual results to differ materially from those in the forward-looking statements are set forth in Ovids filings with the Securities and Exchange Commission under the caption Risk Factors. Such risks may be amplified by the COVID-19 pandemic and its potential impact on Ovids business and the global economy. Ovid assumes no obligation to update any forward-looking statements contained herein to reflect any change in expectations, even as new information becomes available.

Contacts

Investors and Media:Ovid Therapeutics Inc.Investor Relations & Public Relationsirpr@ovidrx.com

Or

Investors: Steve KlassBurns McClellan, Inc.sklass@burnsmc.com(212) 213-0006

Media:Katie Engleman1ABkatie@1abmedia.com

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Ovid Therapeutics Receives FDA Rare Pediatric Disease Designation for OV101 for the Treatment of Angelman Syndrome - BioSpace

Precision Medicine Market Size was valued around USD 50 billion in 2018 and is expected to witness lucrative growth from 2019 to 2025.

Rising demand and advancements in cancer biology will augment personalized medicine market during the forecast timeframe. Development of novel genetic technologies that discovers the functional effect of genetic information that leads in developing cancer, thus, should propel huge demand for cancer biology. However, the high price associated with usage of precision medicine may restrict the precision medicine market growth over forecast period.

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On the basis of technology the precision medicine market is segregated into big data analytics, bioinformatics, gene sequencing, drug discovery, companion diagnostics. Rising focus of competitors on producing advanced drugs leading to better treatment for several chronic diseases will drive drug discovery segmental growth. Similarly, rising incidence of chronic as well as respiratory diseases will drive the growth of the market.

Precision medicine market by application is further divided into oncology, immunology, central nervous system (CNS), respiratory diseases. Increasing prevalence of cancer cases and usage of precision medicine in development of new drugs will increase the oncology segmental market growth. Similarly, increasing demand for bioinformatics and big data analytics to set apart human genome data secured from immunological processes augment segmental growth.

On the basis of end users, the precision medicine market is further divided as pharmaceutical companies, diagnostic companies, healthcare IT companies. Rising demand for producing novel tools for rapid integration, storage, and analysis of patient information will drive the business growth.

North America is anticipated to account for the largest share of the Global Precision Medicine Market. Increasing prevalence of cancer across the U.S will augment the growth of the precision medicine market. Similarly, rising healthcare expenditure will drive the growth of precision medicine market over the forecast period. Asia-Pacific is expected to show rapid growth in coming years owing to growing number of investments in R&D activities.

By Technology

By Application

By End users

By Region

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Conclusion:

In this report, we had discussed the market situation of Precision Medicine Market. As we know the world is going to be tech savvy, the demand of trending products and technologies is also increasing. This report can help to understand the business growth in the Precision Medicine Market and new opportunities at new places.

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Global Precision Medicine Market Growth From 2019 to 2025- Market Report, Insights Analysis And Opportunities - Cole of Duty

A study has found that disabling a gene in the myeloid cells of mice prevents them from developing obesity.

New research has found that inhibiting an immune cell gene in mice prevented them from developing obesity, even when they consumed a diet high in fat.

The studys findings, published in The Journal of Clinical Investigation, may one day help scientists develop therapies that can help people with obesity burn calories more easily.

Obesity is a major health issue, and in the United States, rates of the condition have risen over the past 40 years.

The Centers for Disease Control and Prevention (CDC) report that between 2017 and 2018, 42.4% of people in the country had obesity. Between 1999 and 2000, that figure was 30.5%.

Obesity increases the risk of heart disease, strokes, diabetes, and some types of cancer.

The CDC say that lifestyle changes, including eating a more healthful diet and getting more regular exercise, are key to reducing obesity.

One issue, however, involves obesitys effects on metabolism previous research in mice lead to the suggestion that a person with obesity burns fewer calories than a person who does not have obesity.

Better understanding how and why this might happen, and what scientists and clinicians can do about it, may help with reducing obesity.

In the present study, the researchers inhibited a gene in immune cells in mice. They did this because of an association between obesity and increased inflammation, and immune cells play a key role in controlling inflammation.

The researchers had wanted to find out what part the immune cells play in the metabolic complications of obesity. To their surprise, they found that the cells have a central role in regulating obesity and weight gain.

To study the effects of inhibiting the immune cell gene, the researchers conducted two experiments. In the first, they deleted the gene Asxl2, and in the second, they injected regular mice with nanoparticles that interfered with the function of the gene.

Once the researchers had inhibited this gene in the immune cells, they found that the mice did not develop obesity when fed a high fat diet, and that this was likely due to increased energy expenditure.

Compared with a control group of mice who had obesity but none of the gene inhibition, the mice with the inhibition burned 45% more calories, despite eating high fat diets.

For the studys principal investigator, Prof. Steven L. Teitelbaum, of the Washington University School of Medicine, in St. Louis, MO, Weve developed a proof of concept, here, that you can regulate weight gain by modulating the activity of these inflammatory cells.

It might work in a number of ways, but we believe it may be possible to control obesity and the complications of obesity by better regulating inflammation.

The team is not yet sure why inhibiting the gene in the mices immune cells resulted in them not gaining weight while on a high fat diet. The researchers suspect that the answer may involve encouraging white fat cells to burn fat rather than store it, as brown fat cells do.

While this is only preliminary research, the findings may eventually help people with obesity burn calories at a higher rate, supporting them as they make broader lifestyle changes that involve the diet and exercise.

According to Prof. Teitelbaum, A large percentage of Americans now have fatty livers, and one reason is that their fat depots cannot take up the fat they eat, so it has to go someplace else.

These mice consumed high fat diets, but they didnt get fatty livers. They dont get type 2 diabetes. It seems that limiting the inflammatory effects of their macrophages allows them to burn more fat, which keeps them leaner and healthier.

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Obesity in mice prevented by disabling gene - Medical News Today

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Researchers seek answers to viruss mysteries, clues to possible treatments

Washington University School of Medicine in St. Louis is one of more than 30 genome sequencing hubs worldwide participating in a study to sequence the DNA of young, healthy adults and children who develop severe COVID-19 despite having no underlying medical problems. The researchers also will study people who never become infected despite repeated exposures to coronavirus. Knowledge gained from understanding COVID-19s extremes could lead to new therapeutic strategies for the illness.

To help unravel the mysteries of COVID-19, scientists are sequencing the DNA of young, healthy adults and children who develop severe illness despite having no underlying medical problems. The researchers are looking for genetic defects that could put certain individuals at high risk of becoming severely ill from the novel coronavirus.

The McDonnell Genome Institute at Washington University School of Medicine in St. Louis is one of more than 30 genome sequencing hubs worldwide participating in the study. Rheumatologist Megan A. Cooper, MD, PhD, an associate professor of pediatrics, is leading the research at Washington University. Called the COVID Human Genetic Effort, the international project is co-led by the National Institute of Allergy and Infectious Diseases of the National Institutes of Health (NIH), and Rockefeller University.

The researchers also plan to study people who never become infected with SARS-CoV-2, the virus that causes COVID-19, despite repeated exposures. Such individuals may have genetic variations that protect against infection. For example, certain rare genetic variants are known to thwart some types of viral infections, including HIV and norovirus. Knowledge gained from understanding COVID-19s extremes unusual susceptibility and resistance could lead to new therapeutic strategies for the illness.

The first focus of our study will be patients with severe responses to SARS-CoV-2 infection severe enough to require intensive care who appear otherwise healthy and are younger than 50, said Cooper, who also leads the clinical immunology program and the Jeffrey Modell Diagnostic and Research Center for Primary Immunodeficiencies at St. Louis Childrens Hospital.

These patients dont have uncontrolled diabetes, heart disease, chronic lung disease or any other condition that we know increases the risk of severe complications from COVID-19, she said. For example, we sometimes see stories about, say, a marathon runner or a generally fit, healthy person who nevertheless got very sick from this virus, or the few healthy children who are getting very sick with COVID-19. These are the kinds of patients were interested in for this study. A small proportion of hospitalized patients will fit this category, likely less than 10%.

Cooper studies primary immunodeficiencies in children. Primary immunodeficiencies are a group of more than 450 genetic disorders of the immune system. They often are caused by mutations in single genes that affect different aspects of immunity.

With this pandemic, we can use our skills in gene hunting to search for genes that might be associated with severe COVID-19 in children and younger adults, she said. We can foresee a future ability to do a genetic sequencing test for individual patients hospitalized with SARS-CoV-2 and get an idea of whether they are likely to need more intensive care. In the meantime, we will be able to learn a great deal about how the immune system responds to this virus and what it needs to be able to respond effectively and in an appropriate manner.

These patients genetics could reveal the important immune pathways that the body needs to fight the virus. That knowledge could lead to therapies that also could help other patients who dont have a genetic susceptibility to the virus but perhaps have high-risk conditions, such as diabetes or heart disease.

Our immune systems have never seen this virus before, Cooper said. Were seeing severe COVID-19 complications play out across the world right now. It is going to take a global effort to investigate the genetic factors and the immune system factors that really control this infection.

Research related to COVID-19, including collecting and distributing of patient samples, is managed through Washington Universitys Institute of Clinical and Translational Sciences (ICTS), led by William G. Powderly, MD, who is also the Larry J. Shapiro Director of the Institute for Public Health, the J. William Campbell Professor of Medicine and co-director of the Division of Infectious Diseases.

This research is supported by funding from the St. Louis Childrens Hospital Foundation and the Jeffrey Modell Foundation.

Washington University School of Medicines 1,500 faculty physicians also are the medical staff of Barnes-Jewish and St. Louis Childrens hospitals. The School of Medicine is a leader in medical research, teaching and patient care, ranking among the top 10 medical schools in the nation by U.S. News & World Report. Through its affiliations with Barnes-Jewish and St. Louis Childrens hospitals, the School of Medicine is linked to BJC HealthCare.

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COVID-19 study looks at genetics of healthy people who develop severe illness - Washington University School of Medicine in St. Louis