REGENXBIO's MPSII Gene Therapy RGX-121 Continues to Show Efficacy in Long-Term Trial Data
REGENXBIO's RGX-121 shows sustained efficacy in reducing heparan sulfate levels in CSF, potentially allowing patients to remain off ERT, and is well-tolerated. The company plans to submit a BLA for accelerated approval using CSF D2S6 as a surrogate end point.
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RGX-121, an investigational gene therapy, showed an 85% median reduction in cerebrospinal fluid (CSF) levels of heparan sulfate (HS) D2S6 in patients with mucopolysaccharidosis type II (MPS II), supporting its potential as the first gene therapy for this condition. The therapy demonstrated sustained reductions for up to 2 years and was well-tolerated, with REGENXBIO planning to initiate a rolling biologics license application (BLA) in Q3 2024. The BLA will use CSF D2S6 as a surrogate end point to predict clinical benefit.
REGENXBIO's RGX-121 shows sustained efficacy in reducing heparan sulfate levels in CSF, potentially allowing patients to remain off ERT, and is well-tolerated. The company plans to submit a BLA for accelerated approval using CSF D2S6 as a surrogate end point.