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REGENXBIO Shares New Positive Initial Efficacy Data From AFFINITY DUCHENNE Trial

REGENXBIO Inc. has shared an update on the Phase I/II AFFINITY DUCHENNE® trial of RGX-202 in patients with Duchenne ages 4 to 11 years old, including RGX-202 microdystrophin expression from dose level 2. RGX-202 is an an investigational gene therapy product for the potential treatment of Duchenne, utilizing a novel adeno-associated virus (AAV8) to transport a shortened version of the dystrophin gene (micro-dystrophin) to muscle that may provide benefit in place of the missing full-length dystrophin protein.

REGENXBIO reported that RGX-202 at dose level 2 (2×1014 genome copies (GC)/kg body weight) has demonstrated significantly increased microdystrophin expression in a 12-year-old patient compared to control at three months and a reduction from baseline in serum creatinine kinase (CK) levels at ten weeks. This data supports evidence of clinical improvement.

REGENXBIO expects to make a pivotal dose determination in mid-2024. The company also expects to share strength and functional assessment data for both dose levels and the initiation of a pivotal trial in the second half of 2024.

Read REGENXBIO’s press release here.

The post REGENXBIO Shares New Positive Initial Efficacy Data From AFFINITY DUCHENNE Trial appeared first on Parent Project Muscular Dystrophy.

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