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REGENXBIO Announces Completion of Dosing in the Phase I/II Trial of RGX-111 for the Treatment of Severe MPS I
RGX-111 is an investigational AAV Therapeutic for the treatment of severe MPS I that is part of REGENXBIO's clinical-stage pipeline of neurodegenerative
About this update from Regenxbio Inc.
[{"type":"text","content":"RGX-111 is an investigational AAV Therapeutic for the treatment of severe MPS I that is part of REGENXBIO's clinical-stage pipeline of neurodegenerative disease programs.Expanded Cohort 2 enrollment is complete; eight patients have received RGX-111 in the trial Company intends to manufacture commercial-scale cGMP material to support the continued development of RGX-111 Company expects additional interim updates from the RGX-111 trial in the first half of 2023ROCKVILLE, Md., Dec. 8, 2022 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced it has completed dosing in the expanded Cohort 2 of the Phase I/II trial of RGX-111 for the treatment of severe Mucopolysaccharidosis Type I (MPS I). The trial has now completed dosing, with eight patients enrolled across two dose cohorts. As previously reported, RGX-111 has also been administered to a patient with MPS I through a single-patient IND.\nRGX-111 is an investigational one-time gene therapy designed to deliver the gene that encodes the α-l-iduronidase (IDUA) enzyme using the NAV AAV9 vector. RGX-111 is administered directly to the CNS. The primary endpoint of the trial is to evaluate the safety of RGX-111. Secondary and exploratory endpoints include biomarkers of IDUA enzyme activity in the cerebrospinal fluid (CSF), serum and urine, neurodevelopmental assessments, and caregiver reported outcomes. Patients were treated across two dose cohorts: 1.0x1010 genome copies per gram (GC/g) of brain mass (n=2) and 5.0x1010 GC/g of brain mass (n=6). In the single-patient IND for RGX-111, a severe MPS I patient was dosed with 1x1010 GC/g of brain mass.\n\"We have made great progress this year advancing our pipeline of neurodegenerative disease programs, and completing the dosing in this MPS I trial is another important clinical milestone as we continue to develop potential one-time gene therapies for patients,\" said Steve Pakola, M.D., Chief Medical Officer of REGENXBIO. \"Earlier, at this year's WORLDSymposium meeting, we presented data on biomarker and neurodevelopmental assessments that indicated an encouraging CNS profile in patients dosed with RGX-111. We intend to advance the program for RGX-111 with the aim of providing a much-needed new treatment option for the MPS I community as quickly as possible.\"\nThe Phase I/II trial of RGX-111 is a global trial, which includes sites i...