Vertex and CRISPR look beyond beta thalassemia and sickle cell disease. Also, Bicycle gets $50m up front in Novartis deal, Regeneron commits $75m initially under a Treg collaboration with Sonoma, and Jounce gets a better deal from Concentra to wave off Redx merger.

The Swiss major’s CDK4/6 inhibitor has met the primary endpoint in a pivotal breast cancer trial earlier than expected, with success in the intermediate risk group representing a $4bn opportunity untapped by rival Eli Lilly.

Amarin is digging deep to take Hikma back before a US district court in its induced infringement case against the generics manufacturer.

A November 2022 refuse-to-file letter cited clinical and statistical issues for the stem cell therapy, which failed its Phase III clinical efficacy endpoint. However, BrainStorm has consistently asserted that NurOwn demonstrated benefit in patients with less advanced disease at baseline, and it is encouraged by regulatory flexibility FDA recently has shown for the neurodegenerative disease.