Industry Insights
Latest in gene therapy and rare disease
Gilead reveals partial clinical hold on 2 CAR-T cell therapy trials
Gilead Sciences announced on its third-quarter earnings call Oct. 29 that the company has placed a partial clinical hold on two studies for the CAR-T cell therapy KITE-753 in diffuse large B-cell lymp
Cell therapy investment grows as safety events rankle Big Pharma's CAR T autoimmune push
Sail Biomedicine’s $925 million deal with Johnson & Johnson helped boost the cell and gene therapy sector over biologics in terms of venture capital fundraising in the third quarter.
UniQure snags May Day decision date for Huntington’s gene therapy after arduous year
After a year of controversial regulatory reversals, the FDA has accepted uniQure’s application for ifezuntirgene inilparvovec for priority review, with a May 1 action date. If approved, the therapy—al
Plug-and-play gene therapy: FDA executes on vision that’s been in the works for years
Former biologics boss Peter Marks had a vision. With the FDA’s latest draft guidance, it’s coming to fruition. Speaking with BioSpace, current CBER leader Karim Mikhail said the “adapted pathway” is d
StockWatch: Gene Editing Shares Jump on ESGCT Data, FDA Draft Guidance
Shares of gene editing therapy companies mostly surged this past week on two growth catalysts: Data readouts at the European Society of Gene and Cell Therapy’s 33rd ESGCT Annual Congress, and the FDA'
New biotech Setara forms with $90M to take on Jazz’s bonus rare disease programs
Setara Biosciences has formed around a handful of assets from Actio Biosciences, which was acquired by Jazz Pharmaceuticals earlier this year.
Building the Future of AAV Gene Therapy
This eBook brings together perspectives from across the AAV development and manufacturing continuum. It explores how early commercial planning can help avoid costly delays, how standardized platform a