PBLA05

PBLA05 is a **preclinical in vivo gene therapy** program from **Passage Bio** being developed for **amyotrophic lateral sclerosis**. Publicly available company and industry pipeline materials identify it as an active Passage Bio program and describe it as a gene therapy associated with **C9orf72**, suggesting a mutation-targeted strategy for ALS biology, but detailed construct-level information such as vector design, transgene or silencing cassette, exact mechanism, administration route, and clinical protocol have not been clearly disclosed in primary public sources. Based on the available evidence, PBLA05 appears to be an investigational CNS-focused genetic medicine intended to address ALS linked to C9orf72-related pathobiology, and it remained in preclinical development in the company's earlier pipeline disclosures.