BioCentury
ARTICLE | Regulation

Jan. 16 Quick Takes: Casgevy gains quick label expansion in β-thalassemia

Plus: Deerfield adds  Washington University in St. Louis to arsenal of academic center collaborations and updates from Allakos, GSK  

January 16, 2024 11:47 PM UTC

Two months after the landmark approval of Casgevy exagamglogene autotemcel in the U.K. made it the world’s first authorized CRISPR/Cas9 gene editing therapy, FDA has expanded the treatment’s label to include patients with transfusion-dependent β-thalassemia. The agency first approved Casgevy from Vertex Pharmaceuticals Inc. (NASDAQ:VRTX) and CRISPR Therapeutics AG (NASDAQ:CRSP) in November to treat sickle cell disease. Tuesday’s decision came more than two months before Casgevy’s March 30 PDUFA date in its new indication.

Deerfield Management announced a 10-year commitment of up to $130 million and functional support for therapeutic and diagnostic candidates developed at Washington University in St. Louis via VeritaScience, the partners’ private R&D collaboration. Washington University investigators will submit project proposals to VeritaScience’s review committee, which is led by Deerfield and guided by the university’s Office of Technology Management and Washington University School of Medicine’s business development team; accepted proposals will receive development plans to take assets through IND, and may be eligible for additional funding and company creation support. Deerfield has formed similar collaborations with nearly 30 research institutions and medical centers...