Therapy Areas: Central Nervous System
FDA Awards Fast Track Designation to Regenxbio Gene Therapy for Treatment of MPS I
18 June 2018 - - Rockville, Maryland-based clinical-stage biotechnology company Regenxbio (NASDAQ: RGNX) has been granted fast track designation for RGX-111 by the US Food and Drug Administration, the company said.
RGX-111 is being developed as a novel, one-time, direct-to-central nervous system (CNS) treatment for mucopolysaccharidosis type I (MPS I) that includes the NAV AAV9 vector encoding a gene for human iduronidase (IDUA).
Delivery of the enzyme could provide a permanent source of secreted IDUA, which is otherwise untreated in intravenous-only therapy due to the blood-brain barrier. This strategy could also provide rapid IDUA delivery to the brain, potentially preventing the progression of cognitive deficits that otherwise occurs in MPS I patients.
MPS I is a rare autosomal recessive genetic disease caused by deficiency of IDUA, an enzyme required for the breakdown of the polysaccharides in lysosomes.
These polysaccharides, called glycosaminoglycans, accumulate in tissues of MPS I patients, resulting in characteristic storage lesions and diverse clinical signs and symptoms including in the CNS.
Regenxbio is seeking to improve lives through the curative potential of gene therapy with the NAV technology platform, a proprietary adeno-associated virus gene delivery platform, consists of exclusive rights to more than 100 novel AAV vectors.
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