AXV-201 / Axovia Therap - LARVOL DELTA

A codon-optimised human sequence of human MC4R regulates cAMP levels and normalise weight in Mc4r mutant obese males and females when delivered with as a self-complementary AAV9 (scAAV9) with an intracerebroventricular delivery. (ASGCT 2025) - "In summary, we describe here a new human sequence and AAV9 vector (we called AXV-201) that can rescue the weight gain phenotype and could prove to be a powerful treatment for MC4R patients with severe obesity and very high BMIs. Disease Focus of Abstract:Obesity"

Genetic Disorders • Obesity • MC4R

Axovia Therapeutics Unveils New Preclinical Data for AXV-201, for Treatment of Genetic Obesity Caused by MC4R Mutations, at ASGCT (GlobeNewswire) - "Preclinical proof-of-concept data highlights include: New codon-optimized self-complementary human cMC4R sequence was able to express the MC4R transgene activity greater than five times more efficiently than the wild-type cDNA. In vivo administration of AXV-201 in Mc4r-null mice prevented the development of obesity in males and females, restoring a normal weight trajectory comparable to wild-type controls and showed normalization of neurometabolic markers. No safety concerns were observed when wild-types cohorts were dosed with AXV-201."

Preclinical • Obesity