LGMD gene therapy gets FDA orphan drug designation
Treatment from nonprofit Cure Rare Disease targets LGMDR9
The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to CRD-003, an investigational gene therapy for limb-girdle muscular dystrophy type 2i/R9 (LGMDR9).
The FDA gives this designation to experimental therapies designed to treat rare diseases, defined as conditions affecting fewer than 200,000 people in the U.S. The designation confers incentives to companies investing in developing treatments for rare diseases, such as seven years of market exclusivity if they are ultimately approved.
CRD-003 is being developed by Cure Rare Disease, a nonprofit biotechnology company focused on developing genetic treatments for ultra-rare neuromuscular diseases.
“Receiving orphan drug designation is a critical milestone for Cure Rare Disease as we advance our gene therapy program for LGMDR9,” Richard Horgan, founder and CEO of Cure Rare Disease, said in a company press release.
Horgan said the designation “not only supports our approach, but also underscores the urgent need for innovative genetic medicines that can meaningfully change the lives of patients and their loved ones.”
Gene therapy allows cells to do their work
Limb-girdle muscular dystrophy encompasses more than 20 distinct diseases characterized by muscle wasting at the shoulders and the hips. LGMDR9, also known as LGMD type 2i, is caused by mutations in the FKRP gene. This gene provides instructions to make an enzyme called fukutin-related protein (FKRP), which is needed to make a modification, called glycosylation, to a structural protein known as alpha-dystroglycan in muscle cells.
CRD-003 is designed to deliver a healthy copy of the FKRP gene to muscle cells, allowing cells to produce a functional version of the FKRP protein and restore glycosylation of alpha-dystroglycan to maintain muscle integrity and function.
“Patients and families affected by FKRP-related muscular dystrophies currently face a devastating lack of treatment options,” Horgan said.
The gene therapy works using an adeno-associated viral (AAV) vector, a version of a virus that’s been engineered to deliver a therapeutic gene instead of causing infection. Gene therapies using AAV vectors can cause liver inflammation when infused into the bloodstream, which can result in life-threatening side effects. But according to Horgan, the vector used in CRD-003 is designed to avoid the liver, which is expected to improve safety.
“Using a liver de-targeting, second-generation AAV vector, we believe this may be a safer approach compared to first-generation [viral vectors],” Horgan said.
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