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.