VRTX - Questions Surround Vertex/CRISPR's Sickle Cell Gene Therapy As FDA AdComm Meeting Approaches | Benzinga
The FDA's Cellular, Tissue, and Gene Therapies Advisory Committee will meet next week, Tuesday, 31 October, to discuss whether Vertex Pharmaceuticals Inc (NASDAQ: VRTX) and CRISPR Therapeutics AG's (NASDAQ: CRSP) sickle cell disease (SCD) gene therapy candidate.
While the agency did not raise any concerns regarding the efficacy of the therapy, dubbed exagamglogene autotemcel (exa-cel), the FDA questioned whether Vertex used an appropriate sample size for its off-target editing analysis, according to FDA briefing documents released.
Also Read: Mixed Signals For CRISPR Therapeutics, Analyst Weighs Barriers, Potential Sales For Sickle Cell Therapy.
The agency noted that "it is ...