Author Archives: Marisa Wexler MS

FDA Grants Fast Track Status to Edgewise’s EDG-5506 for BMD

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 Acquires Investigational Gene Therapy for LGMD2A

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,…

FDA OKs Trial of Investigational DM1 Therapy

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…

LION-101 Gene Therapy for Rare MD Type Now on FDA Fast Track

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…

Vita Raises $32M to Advance Cell Therapies for LGMD, Other Dystrophies

Vita Therapeutics announced that it has raised $32 million in financing to advance its work into cell-based therapies for muscular dystrophy. “At Vita Therapeutics our mission is to deliver long-term disease-modifying cell engineered treatments for patients living with muscular dystrophies and other high unmet medical needs,” Douglas Falk, the…

Gene Therapy Trial for Rare Form of MD Planned for Next Year

Asklepios BioPharmaceutical (AskBio) is planning to launch a Phase 1/2 clinical study to evaluate LION-101, its investigational gene therapy for limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9). Dosing in the trial is expected to start in the first half of next year. “We are excited to begin clinical trials…