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Regenxbio's gene therapy for rare disease wins US FDA fast track status

Regenxbio Inc. said the U.S. Food and Drug Administration granted fast track designation to its gene therapy RGX-111 to treat a certain rare genetic disease.

The Rockville, Md.-based biotechnology company is evaluating RGX-111 as a treatment for mucopolysaccharidosis type I.

MPS I is caused by deficiency of an enzyme needed to break down polysaccharides, a type of a carbohydrate. The polysaccharides accumulate in tissues, resulting in symptoms which can include excessive accumulation of fluid in the brain, spinal cord compression and cognitive impairment.

In a phase 1 trial, Regenxbio's therapy will be administered directly in patients' cerebrospinal fluid.

The FDA fast track program is designed to assist in the development, and advance the review of new drugs that can treat serious conditions and address unmet medical needs.