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Regenxbio Presents Positive Initial Data From Phase I/II Trial Of Rgx-111 for the Treatment of Severe MPS I at 18th Annual World Symposium™ 2022
REGENXBIO Inc. (Nasdaq: RGNX) today announced positive interim data are being presented at the 18th Annual WORLDSymposium™ from five patients in the ongoing Phase I/II trial and one patient from a single-patient Investigational New Drug (IND) application of RGX-111 for the treatment of severe Mucopolysaccharidosis Type I (MPS I).
RGX-111 is an investigational one-time gene therapy designed to deliver the gene that encodes the IDUA enzyme using the 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 α-l-iduronidase (IDUA) enzyme activity in the cerebrospinal fluid (CSF), serum and urine, neurodevelopmental assessments, and caregiver reported outcomes.
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