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December 1, 2023

REGENXBIO has announced that the first patient has received RGX-202 at dose level 2 in the Phase I/II AFFINITY DUCHENNE® trial.

What is RGX-202?

RGX-202 is an investigational one-time AAV Therapeutic for Duchenne muscular dystrophy. It is designed to support the delivery and targeted expression of genes throughout skeletal and heart muscle using the NAV® AAV8 vector, a vector used in numerous clinical trials, and a well-characterized muscle-specific promoter (Spc5-12). One-time administration of RGX-202 aims to address the underlying cause of Duchenne by potentially enabling the production of microdystrophin in muscle cells to protect them from damage and ultimately preserve muscle function.

“Progressing to dose level 2 is an important milestone in our updated strategic plans and for accelerating the development of RGX-202. There is a large unmet need for new therapies for boys with Duchenne, and the market is capable of supporting multiple gene therapies. We believe RGX-202 has unique, differentiating features that support its potential to be a best-in-class product.”

Kenneth T. Mills, President and Chief Executive Officer of REGENXBIO

Initial biomarker data in two patients who received RGX-202 at dose level 1 and who completed a three-month assessment demonstrated robust RGX-202 microdystrophin expression with localization to the muscle cell membrane. In recently completed preclinical efficacy studies, RGX-202 at dose level 2 showed improvement in functional performance, compared to dose level 1, as determined by forelimb muscle strength and treadmill exhaustion in mdx mice.

REGENXBIO expects to share initial strength and functional assessment data for both dose levels and anticipates pivotal dose determination and the initiation of a pivotal program in 2024.

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