CRISPR Therapeutics
NEWS
Vertex and CRISPR Therapeutics are setting up treatment centers for patients with beta thalassemia and sickle cell disease to compete with bluebird’s established infrastructure.
Vertex Pharmaceuticals and CRISPR Therapeutics have secured another FDA approval for their Casgevy Cas9 gene-edited cell therapy, this time in treating transfusion-dependent beta thalassemia.
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.
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.
IN THE PRESS