AAV-UPF1

AAV-UPF1 is an investigational gene therapy developed by MeiraGTx for the treatment of amyotrophic lateral sclerosis (ALS). It utilizes an adeno-associated virus (AAV) vector to deliver the *UPF1* gene, which encodes the master regulator of the nonsense-mediated decay (NMD) pathway. UPF1 is critical for RNA metabolism and surveillance; its upregulation is intended to restore NMD function and protect motor neurons from toxicity associated with TDP-43 proteinopathy, a hallmark of both sporadic and familial ALS. The therapy aims to target the underlying cell biology driving motor neuron death rather than a specific genetic mutation, potentially allowing for the treatment of a broad ALS patient population. The program is currently in preclinical development, with IND-enabling studies planned to support clinical entry.

Other names

MeiraGTx UPF1 gene therapyAAV-UPF1-ALSAAV-UPF-1-ALSAAV-UPF 1-ALS