Vertex/CRISPR Partnered Cell Therapy Shows Encouraging Response In Sickle Cell Disease, Thalassemia Patients
Portfolio Pulse from Vandana Singh
Vertex Pharmaceuticals and CRISPR Therapeutics announced that their partnered cell therapy, exagamglogene autotemcel (exa-cel), met primary and key secondary endpoints in pivotal trials for transfusion-dependent beta-thalassemia (TDT) and severe sickle cell disease (SCD). The FDA has accepted the application for exa-cel and granted priority review for SCD and standard review for TDT.
June 09, 2023 | 3:23 pm
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POSITIVE IMPACT
CRISPR Therapeutics' partnered cell therapy with Vertex Pharmaceuticals, exa-cel, met primary and key secondary endpoints in TDT and SCD trials. FDA has accepted the application and granted priority review for SCD and standard review for TDT.
The positive trial results for exa-cel indicate a potential new treatment option for TDT and SCD patients. The FDA's acceptance of the application and granting of priority review for SCD and standard review for TDT show regulatory support for the therapy, which could lead to eventual approval and commercialization. This is likely to have a positive short-term impact on CRISPR Therapeutics' stock price.
CONFIDENCE 90
IMPORTANCE 75
RELEVANCE 50
POSITIVE IMPACT
Vertex Pharmaceuticals' partnered cell therapy with CRISPR Therapeutics, exa-cel, met primary and key secondary endpoints in TDT and SCD trials. FDA has accepted the application and granted priority review for SCD and standard review for TDT.
The positive trial results for exa-cel indicate a potential new treatment option for TDT and SCD patients. The FDA's acceptance of the application and granting of priority review for SCD and standard review for TDT show regulatory support for the therapy, which could lead to eventual approval and commercialization. This is likely to have a positive short-term impact on Vertex's stock price.
CONFIDENCE 90
IMPORTANCE 75
RELEVANCE 50