Avidity Biosciences brings to Novartis three RNA therapies in pivotal testing for rare neuromuscular diseases along with the technology that produced them. This platform develops drugs in a new class of targeted medicines offering potential to expand the delivery of RNA treatments beyond the liver.
Sarepta Therapeutics knew about the death of a patient treated with its experimental gene therapy for a type of limb-girdle muscular dystrophy, but said nothing publicly for a month. Financial analysts lambasted Sarepta executives for failing to disclose the fatality when the company announced a restructuring that includes stopping work on this limb-girdle program.
How to turn analytics into actual policy outcomes.
Sanofi is collaborating with startup miRecule to develop an RNA therapy for facioscapulohumeral muscular dystrophy. The pharmaceutical giant gets global rights to miRecule’s therapy for this type of muscular dystrophy, which has no FDA-approved treatments.
British lawmakers voted on Tuesday to allow in vitro creation of babies using the DNA of three people, making it the first country to do so. Despite backlash based on ethical and religious perspectives, this would not be an attempt at genetic modification or creating “designer babies.” The purpose of this particular procedure is purely […]