CRISPR Therapeutics

NEWS
Vertex still has an ongoing diabetes partnership with CRISPR, which gives it a non-exclusive license to the latter’s platform to develop a potential cure for type 1 diabetes.
Iovance’s lifileucel for advanced melanoma and Madrigal’s resmetirom for nonalcoholic steatohepatitis are just a couple of the key decisions on the FDA’s docket this quarter.
A week after securing FDA approval, a European Medicines Agency committee has endorsed Vertex and CRISPR Therapeutics’ Casgevy for sickle cell disease and transfusion‑dependent beta thalassemia.
FDA
Friday’s FDA approval of Vertex-CRISPR’s Casgevy and bluebird bio’s Lyfgenia has immediately revealed startling differences between these two gene therapies: price and a black-box warning.
As Vertex and CRISPR Therapeutics’ exa-cel and Verve Therapeutics’ VERVE-101 move forward, questions remain about possible drawbacks of such therapies.
The gene editing company is dropping two programs and favoring its next-generation assets CTX112 and CTX131, which it will continue to develop in oncology but will also test in autoimmune diseases.
This week, the FDA could approve the first CRISPR-edited therapy in the U.S., while two other companies await decisions on topical drugs.
The U.K.’s conditional marketing authorization for Vertex Pharmaceuticals and CRISPR Therapeutics’ gene-edited therapy exa-cel raises some potential safety concerns with the risk of off-target effects.
The U.K.’s Medicines and Healthcare products Regulatory Agency on Thursday provided conditional marketing authorization for Vertex Pharmaceuticals and CRISPR Therapeutics’ gene-edited therapy exa-cel.
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