RE: FDA - ‘ Black Box ‘ request on CAR -T products - Hemo on list25 Jan 2024 17:55
JFTH, Chris & all invested
Just for reading - here is the rest of the article with the other companies mentioned -
3. Experts Weigh In on Exa-cel's FDA Approval for Transfusion-Dependent Thalassemia
On January 16, 2024, Vertex Pharmaceuticals' and CRISPR Therapeutics' exagamglogene autotemcel (exa-cel; marketed as Casgevy), an autologous CRISPR-based gene-edited cell therapy, was approved by the FDA for the treatment of patients 12 years and older with transfusion-dependent beta thalassemia (TDT). In light of this major decision by the FDA, CGTLive™ decided to reach out to several experts to get their thoughts on how exa-cel could impact the landscape of care for TDT.
4. Autolus Therapeutics' Obe-cel Will Be Reviewed by the FDA for the Treatment of R/R B-ALL
Autolus Therapeutics' biologics license application (BLA) for obecabatagene autoleucel (obe-cel) in relapsed/refractory (r/r) adult B-Cell acute lymphoblastic leukemia (ALL) has been accepted for filing by the FDA, with a Prescription Drug User Fee Act date set for November 16, 2024.
5. BlueRock Licenses FUJIFILM Cellular Dynamics' and Opsis Therapeutics' Investigational iPSC Therapy
OpCT-001 is an induced pluripotent stem cell (iPSC) derived cell therapy currently in development for the treatment of primary photoreceptor diseases. BlueRock, which plans to file an IND for the therapy this year,optioned to exclusively license it under an existing research and development collaboration agreement established between the 3 companies in 2021.
6. Orchard Therapeutics Officially Becomes Part of Kyowa Kirin
Kyowa Kirin has stated that its previously-announced agreement to acquire Orchard Therapeutics has been completed, with Orchard now being a wholly-owned subsidiary of the Japanese pharmaceutical company. Orchard Therapeutics' atidarsageneautotemcel (arsa-cel, Libmeldy) is a cell therapy for the treatment of metachromatic leukodystrophy that is approved in the European Union, UK, Iceland, Liechtenstein, and Norway, but remains an investigational therapy in the United States. Recently, the FDA accepted a biologics license application for arsa-cel with priority review.
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