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REGENXBIO Announces Dosing of First Patient in Phase I/II Trial of RGX-111 for the Treatment of Mucopolysaccharidosis Type I
- Trial will evaluate one-time, direct-to-CNS treatment for MPS I - Second novel gene therapy for neurodegenerative disease program to be actively enrolling
About this update from Regenxbio Inc.
[{"type":"text","content":"- Trial will evaluate one-time, direct-to-CNS treatment for MPS I\n - Second novel gene therapy for neurodegenerative disease program to be actively enrolling patients\n\n\n ROCKVILLE, Md., Dec. 1, 2020 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX), a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy based on its proprietary NAV® Technology Platform, today announced that the first patient has been dosed in the Phase I/II trial of RGX-111 for the treatment of Mucopolysaccharidosis Type I (MPS I). RGX-111 is an investigational one-time gene therapy designed to deliver the α-l-iduronidase (IDUA) gene directly to the central nervous system (CNS) using the NAV AAV9 vector.\nMPS 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 (GAGs), accumulate in tissues of MPS I patients, resulting in characteristic storage lesions and diverse clinical signs and symptoms including in the central nervous system (CNS), which can include excessive accumulation of fluid in the brain, spinal cord compression, and cognitive impairment.\n\"We are leaders in the development of one-time treatments using our NAV Technology Platform and proprietary delivery procedure which is designed to administer our gene therapy candidates directly to the central nervous system (CNS). RGX-111 is our second product candidate for the treatment of a rare, neurodegenerative disease to be dosed in patients, following RGX-121, which is in clinical development for MPS II,\" said Steve Pakola, M.D., Chief Medical Officer of REGENXBIO. \"We believe one-time treatment with RGX-111 can provide sustainable IDUA enzyme for patients, potentially preventing the progression of disease. The dosing of the first patient in the clinical trial for RGX-111 marks an important milestone in our neurodegenerative disease program and our commitment to the MPS community.\"\nThe Phase I/II trial is a multi-center, open-label, dose escalation trial that will evaluate the safety, tolerability and pharmacodynamics of RGX-111 delivered to patients with MPS I via injection directly into the cerebrospinal fluid (CSF). Up to five patients will be enrolled at two dose levels: 1.0x1010 GC/g of ...