Regenxbio initiates gene therapy trial for DMD as part of its AAV therapeutics strategy for 2025

By Jane Byrne contact

- Last updated on GMT

© GettyImages/Yuuji
© GettyImages/Yuuji

Related tags: Duchenne muscular dystrophy, DMD, Regenxbio, Gene therapy

Regenxbio has begun recruiting for its Phase I/2 gene therapy trial in Duchenne muscular dystrophy (DMD).

The US company is evaluating RGX-202 for the treatment of DMD, a potential one-time gene therapy treatment for the disease that is designed to deliver a transgene for a novel microdystrophin. Dystrophin is a protein missing or found in very small amounts in people with Duchenne. 

RGX-202 uses Regenxbio’s proprietary NAV AAV8 vector.

The clinical trial, Affinity Duchenne, is a multicenter, open-label dose evaluation and dose expansion study to evaluate the safety, tolerability, and clinical efficacy of a one-time intravenous (IV) dose of RGX-202 in patients with Duchenne.

Additionally, Regenxbio is recruiting patients in the Affinity Beyond trial, an observational screening study. The primary objective of that program is to evaluate the prevalence of AAV8 antibodies in patients with Duchenne up to 12 years of age. Information collected in this study may be used to identify potential participants for the Affinity Duchenne trial and potential future trials of RGX-202, said the developer.

Kenneth Mills, CEO of Regenxbio, said the RGX-202 program is a key piece of the company’s '5x'25' strategy to have five adeno-associated virus (AAV) therapeutics either on the market or in late-stage development by 2025.

In-house manufacturing

Regenxbio has manufactured additional clinical supply of RGX-202 in its in-house manufacturing innovation center.

Located in the developer’s 132,000 square foot headquarters in Rockville, Maryland, that facility is designed to meet global clinical and commercial regulatory standards, and includes two independent bulk drug substance production suites, a final drug product suite and integrated quality control labs. Regenxbio says it is one of only a few gene therapy companies worldwide with a cGMP facility capable of production at scales up to 2,000 liters.

In-house manufacturing is a key differentiator for Regenxbio as a leader in gene therapy,”​ said Curran Simpson, chief of operations and technology officer at Regenxbio in June last year. “Quality manufacturing is crucial to all stages of AAV gene therapy development… bringing our manufacturing in-house allows us to control the process from beginning to end and provides flexibility to support a wide range of clinical and commercial needs.”

Indeed, that control aspect came to the fore in May 2022 when Regenxbio announced​ it had to delay dosing of patients in its DMD gene therapy clinical trial following a quality issue at an unnamed third-party contract manufacturer.

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