CELL AND GENE THERAPY, INDUSTRIAL IMPACT
Sana Biotechnology | January 30, 2023
Sana Biotechnology, Inc., a firm focused on producing and delivering engineered cells as medicines, recently announced that the FDA has approved the company's Investigational New Drug (IND) application to begin a first-in-human trial of SC291 in patients with different types of B-cell malignancies.
SC291 is an allogeneic CAR T cell therapy that targets CD19. It is developed by Sana using its hypoimmune platform. The hypoimmune platform's objective is to overcome the allogeneic cells' immunologic rejection, which, if valid for SC291, could lead to prolonged CAR T cell persistence and increased rates of durable complete responses in patients with B-cell lymphomas or leukemias.
The hypoimmune platform includes disruption of the major histocompatibility (MHC) class I and class II expression to conceal cells from the adaptive immune system, which consists of antibody and T cell responses and overexpression of CD47 to inhibit activation of the innate immune cell system, especially macrophages and natural killer (NK) cells. The company has provided evidence from numerous preclinical models demonstrating the potential of this platform to cloak cells from immune detection and the therapeutic potential of SC291 for patients with B-cell malignancies.
About Sana Biotechnology
Founded in 2018, Sana Biotechnology is a firm focused on utilizing engineered cells as medicines for patients. It is constructing differentiated capabilities across the spectrum of cell and gene therapy. The company is motivated by three goals as it seeks solutions for people with poor outcomes or diseases that are currently incurable. The first is the ability to repair and control the genes in any cell in the body. It is developing innovative delivery technologies in order to deliver any payload to any cell in a precise, predictable, and repeatable manner, paving the way for next-generation in vivo gene therapy. Next comes the ability to develop pluripotent stem cells ex vivo into immune-cloaked functional cells, to replace any missing or damaged cells in the body. Finally, it believes that it can also enable broader access to its therapies by focusing on scalable manufacturing solutions, the manufacturing costs, and aligning with key stakeholders.
CELL AND GENE THERAPY, MEDICAL
Be The Match BioTherapies® | January 18, 2023
On January 17, 2023, Be The Match BioTherapies, a company that provides solutions for organizations that develop and commercialize cell and gene therapies, introduced its offering of CIBMTR® clinical research organization services (CRO services), which significantly reduces the time needed to launch and execute high-impact clinical trials.
These CRO services are the outcome of a collaboration between Be The Match BioTherapies and CIBMTR, resulting in expertise and access to unmatched research, sites, donors, partnerships, scientific and operational knowledge, and other resources.
CIBMTR CRO services offer unique capabilities such as
Established relationships with over 225 clinical locations domestically
Nearly 20 years of expertise in transplant and cellular therapy trials and study management, as well as clinically-trained staff
On-staff transplant physicians provide direct contact with patients and donors for clinical and research insights.
A link to the CIBMTR outcomes database for over 575,000 patients
The new CRO services offer turn-key solutions, including protocol development and approval oversight, site selection, project management, site start-up, research sample/laboratory coordination, data management, study monitoring, patient-reported outcomes, statistical analysis, and financial administration. For certain services, such as data analysis, surveys, site selection and administration, and sample management, businesses may instead rely on the expertise of CIBMTR CRO services.
About Be The Match BioTherapies®
Be The Match BioTherapies is the sole supplier of cell and gene therapy solutions with fully configurable services to assist the cell therapy supply chain. It is committed to accelerating patient access to life-saving cell and gene treatments by delivering the best-quality cellular source material from Be The Match Registry®, the most-varied registry of prospective blood stem cell donors in the world, with over 7 million potential blood stem cell donors. Through global collaborations with apheresis, bone marrow collection, and transplant centers, the firm creates, onboards, trains, and maintains extensive collection networks to advance cell treatments.
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.