The P-I/II/III (CAMPSIITE) trial evaluates RGX-121 in patients (ages 4mos.-5yrs.) with MPSII (Hunter Syndrome). The 1EP is the measurement of CSF GAGs
Patients in the trial achieved decreased CSF levels of D2S6 at 16wks. (86% median reduction) with RGX-121 whereas the long-term follow-up data showed that 80% of patients were ERT-free at the last time point. REGENXBIO expects to submit a BLA by H2’24 & receive an RPD Priority Review Voucher in 2025
RGX-1212 is a one-time gene therapy designed to deliver the IDS gene IDS encoding the I2S enzyme using the NAV AAV9 vector that earlier received ODD, RPDD, FTD & RMAT designations from the US FDA & ATMP from the EMA. The results from the trial were presented at the WORLDSymposium 2024
Ref: REGENXBIO | Image: REGENXBIO
Related News:- REGENXBIO’s RGX-121 Receives the US FDA’s RMAT Designation for the Treatment of Hunter Syndrome
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