Marisa Wexler, MS,  senior science writer—

The U.S. Food and Drug Administration (FDA) has granted fast track designation to AOC 1001, an investigational treatment for myotonic dystrophy type 1, known as DM1, a late-developing muscle disorder. The designation will enable AOC 1001’s developer, Avidity Biosciences, to have more frequent interactions with the FDA throughout…

Note: This story was updated Oct. 8, 2021, to clarify that Sarepta did not confirm previously announced plans to enroll 75 boys between the ages of 4 and 7 in the EMBARK trial in its latest announcement. Sarepta Therapeutics and Roche have announced the launch of EMBARK,…

Treatment with the investigational cell therapy CAP-1002 significantly improved arm and heart function in boys and young men at advanced stages of Duchenne muscular dystrophy (DMD), according to final data from the HOPE-2 clinical trial. The Phase 2 trial also “met various skeletal and cardiac endpoints [goals] suggesting clinically…

BBP-418, an investigational disease-modifying medication that aims to improve muscle strength and function in people with limb-girdle muscular dystrophy type 2i (LGMD2i), has been given fast track designation by the U.S. Food and Drug Administration (FDA). This designation aims to facilitate and speed the development and regulatory review of…

A new viral vector that is better than conventional vectors — tools designed to deliver genetic material into cells — at targeting muscle cells may prove useful in developing a more effective gene therapy for muscular dystrophy (MD), according to researchers. In fact, this new vector “is more than…

Benitec Biopharma is taking steps to get the needed regulatory clearances to start clinical testing in Europe and the U.S. of BB-301, its experimental gene therapy for oculopharyngeal muscular dystrophy or OPMD, a type of the genetic disease that typically first affects the eyelids and the throat. The…

The U.S. Food and Drug Administration (FDA) has granted fast track status to EDG-5506, Edgewise Therapeutics’ investigational oral therapy for Becker muscular dystrophy (BMD). This designation is given to experimental treatments that are intended to treat serious conditions and fill an unmet medical need. It confers certain benefits, like…

Sarepta Therapeutics has executed an exclusive license agreement for a gene therapy candidate that aims to treat limb-girdle muscular dystrophy type 2A (LGMD2A), the company announced. The investigational therapy, called calpain 3 or CAPN-3, was developed at the Abigail Wexner Research Institute at Nationwide Children’s Hospital, in Columbus,…

The U.S. Food and Drug Administration (FDA) has cleared Avidity Biosciences to start a Phase 1/2 clinical trial testing the company’s investigational therapy AOC 1001 in adults with myotonic dystrophy type 1 (DM1). “The FDA clearance to proceed with our Phase 1/2 clinical trial for AOC 1001…

The U.S. Food and Drug Administration (FDA) has granted fast track designation to LION-101, an investigational gene therapy that Asklepios BioPharmaceutical (AskBio) is developing as a treatment for limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9). This designation is given to therapies designed to fill an unmet need in medical care for…