AAV8-tMCK-AUF1

AAV8-tMCK-AUF1 (also known as REG-A40) is an investigational adeno-associated virus serotype 8 (AAV8) gene therapy designed to deliver the AU-rich mRNA binding factor 1 (AUF1) gene under the control of a truncated muscle creatine kinase (tMCK) promoter. Developed by Regerna Therapeutics in collaboration with the NYU Grossman School of Medicine, this therapy targets muscle-wasting diseases, including Duchenne muscular dystrophy (DMD) and traumatic skeletal muscle injury. AUF1 is an RNA-binding protein that acts as a master regulator of muscle repair and regeneration by binding to AU-rich elements (AREs) in the 3' untranslated regions of target mRNAs. It stabilizes transcripts that promote myogenesis, mitochondrial biogenesis, and neuromuscular junction restoration, while accelerating the degradation of transcripts that inhibit muscle regeneration. In preclinical models, AAV8-tMCK-AUF1 has demonstrated the ability to enhance muscle strength, reduce muscle atrophy, and promote muscle regeneration.

Other names

AAV8-delivered AUF1AAV-8-delivered AUF1AAV 8-delivered AUF1AAV8-tMCK-AUF1-IRES-eGFPAAV-8-tMCK-AUF1-IRES-eGFPAAV 8-tMCK-AUF1-IRES-eGFPAAV8.tMCK.AUF1AAV-8.tMCK.AUF1AAV 8.tMCK.AUF1AUF1 gene therapyAUF-1 gene therapyAUF 1 gene therapy