Alejandra Viviescas, PhD,  —

Sarepta Therapeutics and Aldevron have entered into a long-term partnership focusing on the supply of DNA molecules, called plasmids, that serve as carriers for different gene therapies, the companies announced. Under their agreement, Aldevron guarantees to provide the plasmids needed for Sarepta’s ongoing gene therapy programs and any potential programs…

Amicus Therapeutics has dosed the first patient in the Phase 3 PROPEL clinical trial that aims to compare investigational therapy AT-GAA (ATB200/AT221) to standard treatment in patients with late-onset Pompe disease. The trial (NCT03729362) is still recruiting patients. Throughout 2019, approximately 100 participants who have been previously…