CELL AND GENE THERAPY, INDUSTRIAL IMPACT
Rocket Pharmaceuticals, Inc | February 09, 2023
On February 7, 2023, Rocket Pharmaceuticals Inc., a renowned late-stage biotech firm advancing an integrated and sustainable pipeline of genetic therapies for rare disorders with high unmet needs, announced that FDA has granted Regenerative Medicine Advanced Therapy (RMAT) designation to RP-A501, the company's investigational adeno-associated virus (AAV)-based gene therapy for Danon Disease, a life-threatening genetic cardiac disease with no disease-altering therapies available. RMAT status was granted based on favorable safety and effectiveness findings from the Phase 1 RP-A501 clinical trial and will provide further added benefits of intensive FDA guidance and accelerated evaluation throughout the program's development.
RMAT designation, the same as Breakthrough Therapy designation, offers the advantages of intensive FDA guidance on efficient drug development, such as the ability for early FDA interactions to discuss intermediate or surrogate endpoints, potential priority review of the Biologics License Application (BLA), possible ways to support accelerated approval and satisfy post-approval requirements, and other possibilities to expedite development and assessment. Rocket's RP-A501 program also has Orphan Drug (US) and Rare Pediatric designations.
The Phase 2 pivotal trial is scheduled to begin in the second quarter of 2023. As disclosed previously, the company plans to conduct a single-arm, open-label trial with a biomarker-based composite endpoint and a natural history comparator.
The Phase 1 program's findings provide one of the most extensive investigational gene therapy datasets for any cardiac disease. In patients with Danon Disease treated in the Phase 1 study, RP-A501 was generally well tolerated, with indications of restored expression of the deficient LAMP2 protein and durable improvement or stability of clinical parameters.
Data show consistent and robust improvements in a variety of parameters, including protein expression, decreased autophagic vacuoles, troponin, brain natriuretic peptide (BNP), left ventricular (LV) mass and thickness, and enhanced NYHA class and Kansas City Cardiomyopathy Questionnaire (KCCQ/Quality of Life) measurements. Notably, these BNP and NYHA class improvements and stability contrast sharply with BNP rise and NYHA class worsening found in a representative sample of pediatric and adolescent natural history patients. Overall, the results indicate improvements and normalization in a variety of quantifiable parameters used by cardiologists in clinical practice to foster risk assessment and treatment decisions.
About Rocket Pharmaceuticals, Inc
Founded in 2015, Rocket Pharmaceuticals is a fully integrated clinical-stage firm developing an integrated and sustainable pipeline of investigational genetic therapeutics that have the potential to address the underlying causes of complex rare childhood diseases. Through its platform-agnostic approach, it can build the optimal therapy for each indication, possibly generating revolutionary options for patients suffering from rare genetic disorders. The company has a team of highly productive experts and is increasingly being recognized as a leading biotechnology innovator.
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CELL AND GENE THERAPY, INDUSTRIAL IMPACT
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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INDUSTRIAL IMPACT, MEDICAL
Celularity | January 17, 2023
Celularity Inc., a biotechnology firm based in the United States that develops placental-derived allogenic cell treatments and biomaterials, and CH Trading Group LLC, an international import, export, and trading corporation, have agreed to an exclusive territorial distribution agreement.
CH Trading Group will serve as the exclusive territorial distributor for halal-certified products previously announced by Celularity in over 100 countries. The product distribution agreement for halal-certified products by Celularity provides a five-year minimum accumulated purchase commitment of US $225 million.
CEO of CH Trading Group, Sayed Zayan, said, “Based on market demand for Celularity’s commercial biomaterial products, we believe there is a billion-dollar commercial opportunity across the Middle East and North Africa Islamic markets.” He also added, “We are excited to be in a position to expand access to these important medicines and wellness products to more people across the region through our exclusive territories distributor agreement with Celularity.”
(Source – Business Wire)
“According to Emergent Research's most recent analysis, the global regenerative medicine market was valued at $9.80 billion in 2021 and is predicted to expand at a CAGR of 15.9%, reaching $37.10 billion in 2030.”
Under the agreement, CH Trading Group will distribute products to more than 100 countries that are members of or affiliated with the following intergovernmental organizations (‘Islamic Markets’)
The Gulf Cooperation Council: Consists of Bahrain, Oman, Kuwait, Qatar, Saudi Arabia, and the UAE.
The Organization of Islamic Cooperation: It spans four continents and includes 57 member nations
The African Union: Includes 55 member countries
Robert J. Hariri, M.D., Ph.D., Celularity’s CEO, Chairman and Founder, said, “Our partnership with the CH Group represents another critical milestone towards bringing our innovative halal-certified—under globally recognized Circle H International Inc. standards – regenerative biomaterial products to treat degenerative disease across Islamic Markets.”
(Source – Business Wire)
About Celularity
Headquartered in Florham Park, N.J., Celularity is a clinical-stage biotechnology firm paving the way for the future of cellular medicine by offering off-the-shelf allogeneic placenta-derived cellular therapeutics with unmatched scale, quality, and affordability. With its novel approach to cell therapy, the company can unlock the unique therapeutic potential of the postpartum placenta. In addition, through the placenta, nature’s immunotherapy engine, the firm is driving the next generation of cellular medicine using placenta-derived T cells, NK cells, and pluripotent stem cells to address unmet and underserved clinical needs in cancer, infectious, and degenerative diseases.
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