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 companies are hoping that new data on recently-filed eplontersen will boost the drug's chances of challenging Alnylam’s Amvuttra and Onpattro.

Data from part B of the Swedish firm’s pivotal trial of Tarpeyo in the rare kidney disorder showed that the treatment effects lasted two years across the entire population, enabling the move from conditional to full approvals in several markets.

The latest drug development news and highlights from the Pink Sheet’s US FDA Performance Tracker