- In August 2023, Orchard Therapeutics announced positive long-term results from an updated integrated analysis of 39 patients with metachromatic leukodystrophy (MLD) treated with the investigational gene therapy, OTL-200. This promising data supports the potential of gene therapy as an effective treatment option, instilling greater confidence in gene therapies. The success will accelerate further investments and innovation within the sector, advancing the development of novel therapies for rare diseases
- In April 2022, The FDA expanded its guidance for the development, manufacture, and control of advanced biological products, including chimeric antigen receptor (CAR) T cell therapies. This update provides clearer recommendations to developers and manufacturers, outlining key steps in CAR-T product development. The move aims to streamline the regulatory process and foster the growth of cutting-edge therapies, including personalized treatments for cancers and other complex conditions
- In February 2021, Bristol Myers Squibb’s Breyanzi, a CAR-T cell therapy, received FDA approval for treating adults with relapsed or refractory large B-cell lymphoma. The approval marks a significant milestone for CAR-T therapies, offering patients with difficult-to-treat cancers a new, potentially life-saving treatment option. The therapy uses genetically modified T cells to target and destroy cancer cells, representing a breakthrough in cancer immunotherapy
- In January 2021, Organogenesis Holding Inc. received FDA Regenerative Medicine Advanced Therapy (RMAT) designation for ReNu, a cryopreserved amniotic suspension allograft designed to treat knee osteoarthritis symptoms. The RMAT designation expedites the development of regenerative medicine treatments, facilitating faster clinical trials and regulatory approval. ReNu’s potential to alleviate knee pain and improve joint function positions it as a promising treatment for patients suffering from osteoarthritis
- In January 2021, FUJIFILM Diosynth Biotechnologies announced a $40 million investment in a new process research and manufacturing facility for innovative medicines and viral vectors. This investment underscores the growing demand for advanced manufacturing capabilities in the biotechnology industry, particularly for the production of viral vectors used in gene and cell therapies. The new facility will help address the increasing need for scalable production solutions for cutting-edge therapeutics
