Passage Bio (PASG) H.C. Wainwright 26th Annual Global Investment Conference 2024 summary

Conference overview

• Over 650 companies and 24 senior analysts participated, covering all sectors.

• The event featured presentations and meetings accessible via an online portal.

Lead program highlights

• PBFT02 is a one-time AAV gene therapy targeting neurodegenerative diseases, initially for FTD-GRN.

• Strong clinical responses have prompted expansion into FTD-C9, ALS, and Alzheimer's disease.

• In-house process and analytical development help mitigate late-stage risks.

Clinical and scientific insights

• FTD-GRN is an orphan disease with about 18,000 patients in the US and Europe.

• PBFT02 leverages cross-correction, allowing effective therapy without transducing every cell.

• Ependymal cells, targeted by AAV1, act as long-lived factories for progranulin in the CNS.