Empowered Patient Podcast

Dr. Will Chou, President and CEO of Passage Bio, is developing gene therapies for neurodegenerative diseases, with their lead product being PBFTO2, an AAV gene therapy for a specific genetic variant of frontotemporal dementia. They are also exploring the potential of raising progranulin levels to help patients with other neurodegenerative diseases. Passage Bio's gene therapy, utilizing AAV as the vector, is delivered through an ICM injection into the cisterna magna, bypassing the blood-brain barrier. Will explains, "What we’re doing is a little unique. We are starting in the normal way gene therapies approach this, which is we have a patient population with a specific genetic deficiency. They have frontotemporal dementia caused by a mutation in the granulin gene, in the GRN gene, which means they are deficient in creating a protein called progranulin. Our gene therapy product replaces that progranulin, and by replacing what they don’t have enough of, we hope to stop the neurodegenerative decline from this dis