Short stories about how US FDA’s conference center changed since COVID-19 forced the end of in-person public events, what the agency did to officially close the books on Makena, and more.

Given the challenges of validating patient-reported outcome instruments specific to very rare diseases, agency is probably going to have to accept the use of unvalidated measures in some cases, Peter Marks tells American Society of Gene and Cell Therapy’s annual meeting.

The oncology start-up signed a co-development and commercialization agreement with the French pharmaceutical company for two next-generation EGFR inhibitors.

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.