RegenXBio Inc. ((RGNX)) announced an update on their ongoing clinical study.
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RegenXBio Inc. is conducting a clinical study titled ‘AFFINITY DUCHENNE: RGX-202 Gene Therapy in Participants With Duchenne Muscular Dystrophy (DMD).’ The study aims to evaluate the safety, tolerability, efficacy, pharmacodynamics, and pharmacokinetics of RGX-202 gene therapy in males with DMD. This study is significant as it explores a potential new treatment for DMD, a severe genetic disorder.
The intervention being tested is RGX-202, a genetic therapy delivered via a single intravenous infusion. It is designed to introduce a transgene encoding a novel microdystrophin, which includes functional elements of naturally occurring dystrophin, potentially offering therapeutic benefits for DMD patients.
The study follows a non-randomized, parallel intervention model with no masking, focusing on treatment as its primary purpose. The study is open-label, meaning both researchers and participants know which treatment is being administered. It involves dose evaluation across different cohorts to assess the therapy’s effects.
The study began on January 4, 2023, with its latest update submitted on October 2, 2025. These dates are crucial as they mark the study’s progression and ongoing recruitment status, indicating active research efforts to find a viable treatment for DMD.
This update could positively influence RegenXBio’s stock performance and investor sentiment, as successful outcomes may position the company as a leader in genetic therapies for muscular dystrophies. Competitors in the gene therapy space will be closely monitoring these developments.
The study is ongoing, with further details available on the ClinicalTrials portal.