Beam Therapeutics and Verve Therapeutics have each built their lead candidates on a technique billed as a safer alternative ...

A guide RNA will target the specific sequence within ... Vertex Pharmaceutical presented impressive long-term data for its Casgevy treatment. 39 out of 42 patients with SCD were free from VODs ...

CRISPR Therapeutics benefits from Casgevy’s approval and a deep pipeline, despite early rollout hurdles and cost challenges.

When the guide RNA recognizes and binds to the DNA segment ... The company reports that this patented technology is used in the gene therapy 'CASGEVY' developed by Vertex. CASGEVY is a treatment ...

Its flagship gene therapy product for treating sickle cell disease, Casgevy, faces market hurdles. However, recent political developments, including both at the White House and the Food and Drug ...