Ultragenyx receives complete response letter for UX111 BLA
The FDA's request stems from observations made during recent inspections of Ultragenyx's manufacturing facilities.
Cell & Gene Therapy
JCR Pharma and Alexion partner on JUST-AAV gene therapy platform
DoH and Children’s National Hospital link for cell and gene therapy
Caribou cuts pipeline and workforce to focus on lead CAR-T programmes
European agencies catch up with speedier regulatory pathways for CGTs in the US
Inborn errors of metabolism elicit unique challenges for therapy development
Insmed’s gene therapy poised to challenge DMD landscape after IND clearance
Novartis acquires Kate Therapeutics to advance gene therapies
