REGENXBIO reports positive interim Phase I/II data for RGX-121 (MPS II) and RGX-111 (MPS I)

REGENXBIO Inc.

Both RGX-121 (13 pts) and RGX-111 (5 pts + single-patient IND) well-tolerated with no drug-related SAEs.
RGX-121: Dose-dependent CSF biomarker reductions; Cohort 3 D2S6 near normal levels at last follow-up.
RGX-111: CSF heparan sulfate decreased in all 4 patients; IDUA enzyme detected in 3 of 4 post-dose.
Neurodevelopmental improvement: RGX-121 patients (Cohorts 1-2) show skill acquisition up to 2 years; RGX-111 shows gains in younger and one older patient.
Next steps: RGX-121 Cohort 3 expansion with cGMP material planned 1H2022; RGX-111 Cohort 2 expanded to enroll up to 6 more patients.

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