A cadre of big and small biotechs and pharmas, plus nonprofit organizations, have signed on to a new U.S. initiative to go after gene therapies for rare diseases.
On the list of companies are Biogen, Johnson & Johnson’s Janssen pharmaceutical unit, Pfizer, the Novartis Institutes for BioMedical Research, Regenxbio, Spark Therapeutics, Takeda, Taysha Gene Therapies, Thermo Fisher Scientific and Ultragenyx. The Alliance for Regenerative Medicine, the American Society of Gene and Cell Therapy and other groups are also involved.