Aug 6, 2024
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 disease."
"But what is interesting about progranulin is that a growing amount of research shows that progranulin and raising progranulin in general can be neuroprotective across other neurodegenerative diseases as well. We will be pursuing this progranulin-raising one-time gene therapy, not just in patients who have that specific genetic deficiency in the GRN gene. Also, in other patients, such as patients with ALS and patients with other gene deficiencies with FTD where progranulin and raising progranulin could help their neurodegeneration. So in that way, this is a unique approach to gene therapy. Usually, you have one product and can only help a limited group of patients. Here we’re looking to use the single product and try and help multiple populations of patients with neurodegenerative disease."
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