BIO, the world’s largest trade association for the biotech industry, has responded to the US Food and Drug Administration's draft guidance on developing human gene therapy products that incorporate genome editing (GE) of human somatic cells.
Charles River Laboratories International and ASC Therapeutics have announced plans to manufacture ASC618, a second-generation gene therapy for hemophilia A.
Novartis investigates the death of an infant in a gene therapy clinical trial to determine whether it was related to the treatment but does not anticipate an impact on FDA approval.
Thermo Fisher Scientific bolsters its gene therapy capabilities by acquiring Brammer Bio for $1.7bn, as the market becomes an ‘increasing focus’ for customers.
Biogen agrees Nightstar acquisition for $800m to take on its pipeline of AAV treatments for retinal disorders, diversifyingy its pipeline in the rare disease market.
Sarepta Therapeutics signed a long-term, strategic manufacturing partnership with Paragon Bioservices to produce micro-dystrophin to grow its gene therapy program.
Fibrocell Science will make its gene therapy candidate FCX-007 from a facility in Pennsylvania previously dedicated to making its autologous personalised cosmetic Laviv (azficel-T).
Abeona Therapeutics says it plans to file its single injection adeno-associated virus (AAV) gene therapy for Sanfilippo syndrome in the US in late 2018.
