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Regenxbio Gene Therapy Candidate Gets Fast Track Status for Duchenne Muscular Dystrophy

Regenxbio Gene Therapy Candidate Gets Fast Track Status for Duchenne Muscular Dystrophy
The FDA has granted Fast Track designation to Regenxbio’s RGX-202 candidate as a potential one-time gene therapy treatment for Duchenne muscular dystrophy.

The disorder, which is characterized by progressive muscular weakness, is caused by a genetic mutation that affects the production of dystrophin, resulting in cell damage during muscle contraction. RGX-202 introduces a novel gene to make muscles resistant to the contraction caused by the lack of dystrophin.

The gene therapy candidate is being evaluated in a phase 1/2 clinical trial, in which six patients ages four to 11 years are expected to enroll for a one-time intravenous dose of the medication.

The FDA grants the Fast Track designation to facilitate development and expedite review of new therapeutics to treat serious or life-threatening conditions and to address unmet medical needs.

The agency previously granted RGX-202 Orphan Drug and Rare Pediatric Disease designations.

April 14, 2023

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