Nonprofit models support strategic boldness to develop gene therapies for rare diseases
Généthon CEO Frédéric Revah says alternative patient access approaches are needed for ultra rare diseases.
Cytiva Navigating cell therapy WP 4
Gene editing takes centre stage in FDA’s new rare disease approval pathway
J&J to build $1bn US CGT manufacturing site in Pennsylvania
REGENXBIO receives FDA response for RGX-121 application
Eli Lilly bets on in vivo cell therapy in $2.4bn Orna Therapeutics buyout
Sensorion announces $71.9m reserved offering to expand gene therapy pipeline
Eli Lilly outlays $1.12bn for hearing loss gene therapy partnership
iRegene receives FDA RMAT designation for NouvNeu001 to treat Parkinson’s
