AAV-UPF1

We have designed our product candidate, AAV-UPF1, with the aim of increasing UPF1 expression in the motor neurons of amyotrophic lateral sclerosis (ALS) patients. UPF1 plays an important role in nonsense mediated decay (NMD), a quality control system that regulates RNA processing in neurons. AAV-UPF1 is currently being investigated in preclinical studies.