Gen1 sup-tRNA AAV

Gen1 sup-tRNA AAV is a first-generation (parental) gene therapy candidate developed by Tevard Biosciences in collaboration with Alnylam Pharmaceuticals. It utilizes an adeno-associated virus (AAV) vector to deliver engineered suppressor tRNAs (sup-tRNAs) designed to treat genetic diseases caused by nonsense mutations. The mechanism involves the sup-tRNAs enabling ribosomal readthrough of premature termination codons (PTCs)—specifically UAA, UAG, and UGA—thereby allowing the production of full-length, functional proteins from mutated genes. In the context of Duchenne muscular dystrophy (DMD), Gen1 sup-tRNA AAV was used as the foundational proof-of-concept for restoring full-length dystrophin. While Gen1 demonstrated modest rescue, it served as the baseline for subsequent engineered generations (up to Gen4) that achieved significantly higher potency and functional recovery in disease models.

Other names

parental suppressor tRNA AAVGen1 sup-tRNAGen-1 sup-tRNAGen 1 sup-tRNA