Shots:

• The US FDA has granted RMAT designation to RGX-121, an investigational one-time AAV therapeutic for MPS II also known as Hunter syndrome
• RGX-121 is currently being evaluated in the P-I/II/III (CAMPSIITE) trial for patients aged 4mos.- 5yrs. & trial is enrolling MPS II patients as part of a pivotal program. Patients who continued to receive pivotal program dose levels showed the greatest decreases in CSF GAGs
• The company highlighted interim data from the P-I/II part of the trial which showed that RGX-121 was well-tolerated across 15 patients. The company plans to file the BLA of RGX-121 in 2024 using the accelerated approval pathway while the P-I/II trial of RGX-121 is ongoing for pediatric patients aged ≥5yrs. with MPS

Ref: PR Newswire | Image: REGENXBIO

Related News:- REGENXBIO Presents Interim Results of RGX-121 in P-I/II/III (CAMPSIITE) Trial for MPS II (Hunter Syndrome) at SSIEM 2022

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