To date, what number of U.S. FDA-approved RMATs have been issued and to whom have they been awarded?
The answer is that 83 RMAT (Regenerative Medicine Advanced Therapy) designations have been publicly announced by biotech and pharma companies. However, the FDA states it has received 238 requests and issued 92, which means that a handful are not yet public knowledge. Therefore, a few companies are operating in stealth mode with regard to their RMAT designations and approximately 39% of RMAT applications get approved (92 approvals / 238 applications = 38.7%).
Thus far, Rocket Pharmaceuticals is the only company to receive four RMATs, while two other companies (AlloVir, CRISPR Therapeutics) have received three RMATs to date.
Additionally, nine companies (Abeona Therapeutics, Adaptimmune, Allogene Therapeutics, Athersys, CARsgen Therapeutics, Cook MyoSite, Humacyte, Mesoblast, and Orchard Therapeutics) have managed to secure two RMAT designations from the U.S. FDA.
The most recently awarded RMATs were given to Editas-Medicine on October 16th for EDIT-301 and Immatics N.V. on October 24th for IMA203. [Read more…]
Today, induced pluripotent stem cells (iPSCs) are being exploited for uses in drug discovery and development, toxicology screening, disease modelling, personalized medicine, cell-based therapeutics, and beyond. A fascinatingly diverse range of business models have been developed by market competitors to provide iPSC specific products, services, technologies, and therapies across the life science sector. [Read more…]
Founded in 2014 and headquartered in San Diego, CA, iXCells Biotechnologies is a cell biology and cell technology company specializing in pre-clinical drug discovery solutions. iXCells provides primary cells and iPSC derived cells, custom iPSC services, and functional bioassay development and execution, among other products and services.
In this interview with Nianwei Lin, Co-Founder and President of iXCells Biotechnologies, we discuss the company’s product pipeline, innovative drug discovery solutions, CRO services, and its future goals. [Read more…]
TORONTO and HAIFA, Israel — NurExone Biologic Inc. (TSXV: NRX) (FSE: J90) (NRX.V) (the “Company” or “NurExone”), a pioneering biotechnology company, is thrilled to announce that the U.S. Food and Drug Administration (FDA) has granted Orphan-Drug Designation (ODD) for its ExoPTEN therapy, recognizing the potential of this groundbreaking regenerative therapy for acute spinal cord injury, a condition where effective treatments are limited. [Read more…]