PBGENE-ALS

PBGENE-ALS (also known as PBGENE-CNS) is an investigational in vivo gene editing therapy being developed by Precision BioSciences for the treatment of C9orf72-associated amyotrophic lateral sclerosis (ALS). Utilizing the company's proprietary ARCUS genome editing platform, PBGENE-ALS employs a dual-ARCUS nuclease approach delivered via a single adeno-associated virus (AAV) vector. This strategy is designed to specifically target and excise the pathogenic hexanucleotide repeat expansion (HRE) within the C9orf72 gene, thereby reducing neurotoxic RNA foci and dipeptide repeat proteins while preserving the expression of the wild-type C9orf72 protein. The program is currently in the IND-enabling stage of development.