REG-A40

REG-A40 is an investigational AAV8-mediated gene therapy developed by Regerna Therapeutics for the treatment of Duchenne muscular dystrophy (DMD). The therapy delivers AU-rich element RNA-binding protein 1 (AUF1), an mRNA-binding protein that acts as a master regulator of muscle repair, regeneration, and mitochondrial biogenesis. In preclinical models of DMD, REG-A40-mediated supplementation of AUF1 has been shown to stabilize and increase the translation of key muscle-regeneration mRNAs, notably boosting the expression of utrophin (a functional homolog of dystrophin). This mechanism helps restore myofiber integrity, muscle strength, and endurance, offering a potential disease-modifying approach that addresses muscle wasting and regenerative exhaustion.