Passage Bio (PASG) Chardan's 8th Annual Genetic Medicines Conference summary

Company evolution and strategic focus

• Shifted from broad neurodegenerative gene therapy to focus on PBFT02 for adult FTD with GRN mutation.

• Ended basic research collaboration with University of Pennsylvania; maintains preclinical ties with Gemma Bio.

• Prioritizing clinical execution and data generation in a single indication.

• Out-licensed pediatric gene therapy programs to Gemma Bio to concentrate resources.

Clinical program and study design

• PBFT02 is an AAV gene therapy injected into the cisterna magna, targeting progranulin deficiency in FTD-GRN.

• Phase I/II dose escalation study with two main cohorts; strong early data led to no dose escalation.

• High, durable, and consistent CSF progranulin levels observed in all five initial patients.

• Safety improved with enhanced immunosuppression; no SAEs in last four patients.

Data and clinical impact

• Progranulin levels in CSF are markedly higher than other clinical programs, with no fading over 12 months.

• No adverse effects from high progranulin levels; plasma levels remain unchanged.

• Results exceed preclinical non-human primate models due to lack of antibody response in humans.

• Confidence in clinical benefit is supported by external data linking progranulin increases to improved disease markers.