New Genetic-based Epilepsy Risk Scores

Technology Networks | October 15, 2019

An international team of researchers led by Cleveland Clinic has developed new genetic-based epilepsy risk scores which may lay the foundation for a more personalized method of epilepsy diagnosis and treatment. This analysis is the largest study of epilepsy genetics to date, as well as the largest study of epilepsy using human samples. The research team, led by Dennis Lal, Ph.D., calculated for the first time quantitative scores that reflect overall genetic risk for epilepsy, called polygenic risk scores. They showed that these scores can accurately distinguish on a cohort level between healthy patients and those with epilepsy, as well as between patients with generalized and focal epilepsies. After the first seizure, it can be difficult for clinicians to predict which patients will go on to develop epilepsy. Development of these novel genetic risk scores could help clinicians to intervene and treat patients earlier.

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The reason for the rapid expansion and development of the therapeutic antibody sector is the capacity of monoclonal antibodies to precisely bind a target antigen and neutralise or promote its activity. The efficacy and safety.

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The reason for the rapid expansion and development of the therapeutic antibody sector is the capacity of monoclonal antibodies to precisely bind a target antigen and neutralise or promote its activity. The efficacy and safety.

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INDUSTRIAL IMPACT, MEDICAL

Moderna and Generation Bio Announce Strategic Collaboration to Develop Non-Viral Genetic Medicines

Accesswire | March 24, 2023

Moderna, Inc. a biotechnology company pioneering messenger RNA therapeutics and vaccines, and Generation Bio Co. a biotechnology company innovating genetic medicines for people living with rare and prevalent diseases, today announced that the two companies have entered into a strategic collaboration to combine Moderna's biological and technical expertise with core technologies of Generation Bio's non-viral genetic medicine platform. The collaboration aims to expand the application of each company's platform by developing novel nucleic acid therapeutics, including those capable of reaching immune cells, to accelerate their respective pipelines of non-viral genetic medicines. "Moderna continues to invest in innovative technology to enable us to develop a breadth of transformative medicines for patients," said Rose Loughlin, Ph.D., Moderna's Senior Vice President for Research and Early Development. "Through this collaboration, which builds on Generation Bio's non-viral genetic medicines platform, we have the potential to target immune cells with diverse nucleic acid cargos and the liver for gene replacement. We are excited to have Generation Bio as our partner as we continue to broaden our therapeutic pipeline and extend the potential benefit of nucleic acid therapeutics to more patients." "Non-viral DNA therapeutics may offer durable, redosable, titratable genetic medicines to patients suffering from rare and prevalent diseases on a global scale," said Phillip Samayoa, Ph.D., Chief Strategy Officer of Generation Bio. "This collaboration represents a foundational investment in our platform science, both deepening our pipeline of rare and prevalent liver disease programs beyond hemophilia A and accelerating our work to reach outside of the liver with nucleic acid therapies. We are thrilled to collaborate with Moderna to extend genetic medicines to new tissues and cell types through the joint development of novel targeting for our stealth ctLNPs to reach immune cells." About the Collaboration Under the terms of the agreement, Moderna may advance two immune cell programs, each of which may use a jointly developed ctLNP to deliver ceDNA. In addition, Moderna may advance two liver programs, each of which may use a liver-targeted ctLNP developed by Generation Bio to deliver ceDNA. Moderna retains an option to license a third program for either immune cells or the liver. Generation Bio will receive a $40 million upfront cash payment and a $36 million equity investment issued at a premium over recent share prices. Moderna will fund all collaboration work, including a research pre-payment. Generation Bio is also eligible for future development, regulatory and commercial milestone payments, as well as royalties on global net sales of liver-targeted and immune cell-targeted products commercialized under the agreement. The agreement additionally provides Moderna with the right, subject to certain terms and conditions, to purchase additional shares of common stock in connection with a future equity financing by Generation Bio. Further, Moderna and Generation Bio will both leverage collaboration research to continue to advance in vivo immune cell targeting as a new class of genetic medicines, with downstream economics on products utilizing such technology. Generation Bio is eligible to receive certain exclusivity fees as well as potential development and regulatory milestones and royalties on products that Moderna advances using ctLNP technology developed under the collaboration. About Moderna In over 10 years since its inception, Moderna has transformed from a research-stage company advancing programs in the field of messenger RNA (mRNA), to an enterprise with a diverse clinical portfolio of vaccines and therapeutics across seven modalities, a broad intellectual property portfolio in areas including mRNA and lipid nanoparticle formulation, and an integrated manufacturing plant that allows for rapid clinical and commercial production at scale. Moderna maintains alliances with a broad range of domestic and overseas government and commercial collaborators, which has allowed for the pursuit of both groundbreaking science and rapid scaling of manufacturing. Most recently, Moderna's capabilities have come together to allow the authorized use and approval of one of the earliest and most effective vaccines against the COVID-19 pandemic. Moderna's mRNA platform builds on continuous advances in basic and applied mRNA science, delivery technology and manufacturing, and has allowed the development of therapeutics and vaccines for infectious diseases, immuno-oncology, rare diseases, cardiovascular diseases and auto-immune diseases. Moderna has been named a top biopharmaceutical employer by Science for the past eight years.

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CELL AND GENE THERAPY, INDUSTRIAL IMPACT

CBM Announces Partnership with Virion Therapeutics

Center for Breakthrough Medicines | February 27, 2023

Center for Breakthrough Medicines (CBM), a leading contract development and manufacturing organization dedicated to addressing the challenges of commercializing cell and gene therapies, and Virion Therapeutics, a clinical-stage biotech firm focused on developing accessible and adaptable CD8+ T cell-based technologies for cancer and infectious diseases, have recently announced a strategic collaboration agreement. CBM will partner and manufacture with Virion Therapeutics on their checkpoint modifier clinical development programs, including the First-in-Human VRON-0200 immunotherapy, designed to treat chronic Hepatitis B Virus (HBV) infection. The collaboration aims to develop VRON-0200, a pan-genotypic, global therapeutic immunotherapy that targets chronic HBV infection, which affects over 295 million people worldwide. HBV impairs CD8+ T cells, which results in the loss of viral control. VRON-0200 induces a broad CD8+ T cell response, which includes responses to the core and pol regions not generally induced by the infection. Therefore, VRON-0200 is designed to stimulate a highly functional immune response to help clear the virus. CBM's Co-Founder and Chief Business Officer Audrey Greenberg commented, "Virion is working to revolutionize the immunotherapy treatment landscape, and we are honored to partner with them on this bold mission as we share a common goal – to create a future free of cancer and chronic infectious diseases." She further emphasized, "We have assembled best-in-field technical teams, supported by industry-leading product and process development, manufacturing, and testing capabilities, all located within a single, world-class manufacturing facility. We look forward to working with Virion to bring their life-saving therapies to the patients who need them most, as quickly as possible." (Source – PR Newswire) About Center for Breakthrough Medicines The Center for Breakthrough Medicines is a pioneering contract development and manufacturing organization (CDMO) situated in the center of "Cellicon Valley," with a focus on cell and gene therapies. It offers an all-in-one solution to expedite the speed of delivering advanced treatments to the market. Its customer-oriented approach, combined with a culture that prioritizes the needs of patients, guarantees dependable and high-quality process and analytical development, GMP testing, viral vector manufacturing, plasmid production, cell therapy bioprocessing and cell banking services throughout the entire life cycle of a product.

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CELL AND GENE THERAPY, INDUSTRIAL IMPACT

Scientist.com Announces the Launch of Compliance Mobile App for iOS

Scientist.com | February 02, 2023

On February 1, 2023, Scientist.com, the biopharma industry's largest R&D marketplace, announced the release of a new iOS mobile app. The app provides a new, easy-to-use, on-the-go experience for purchase request approvals and compliance. In addition, iPhone users receive untethered notifications for accepting, delegating, and clarifying requests without needing to use their computers. The result is speedier approvals and fewer delays in scientific discovery. Scientist.com has spent the past fourteen years developing a digital marketplace that allows scientists to devote more time to science and less time to paperwork. The platform enables researchers to test more ideas in less time and at a lesser cost. It digitizes a company's complete research activities, automatically tracking purchases, contract management, payment processing, and generating business intelligence reports. Scientist.com Founder and CTO Chris Petersen commented, "One of the major barriers to drug discovery is the time it takes to ensure all compliance measures are met for a given research project, and understandably so." He added, "Our goal in building the Compliance Mobile App is to put compliance in the pocket of approvers, therefore minimizing the time it takes for researchers to move through the R&D process." (Source – Business Wire) Registered users of the Scientist.com platform can now get the Compliance Mobile App free from the Apple App Store About Scientist.com Founded in 2007, Scientist.com is the world's largest medical research marketplace. Its digital research platform combines a custom-built, cloud-native technology stack with a white-glove customer and scientific support, helping pharmaceutical scientists discover life-saving drugs in less time and at a lower cost. As a result, Scientist.com is accelerating and lowering the cost of medical research by connecting scientists and allowing them to exchange ideas and transact online effortlessly. The company now has a presence in enterprise marketplaces for 20 of the world's top 30 pharmaceutical firms, 80 biotechnology enterprises, and the National Institutes of Health in the United States (NIH).

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