Expanding the Gene Therapy Toolbox: Dr. Bobby Gaspar, Co-Founder & CEO of Orchard Therapeutics
Join us for a super informative look at an emerging approach to gene therapy from Dr. Boby Gaspar, Co-Founder and CEO of Orchard Therapeutics, a UK-based company that’s received one of the rare FDA approvals for gene therapies. Orchard’s method uses gene-modified stem cells in your blood to correct the disease and self-renew, so a single administration can give patients a potentially lifelong effect. Host Lindsey Smith also draws out insights from Dr. Gaspar on newborn screening, conducting clinical trials for rare disorders, and how learnings from Orchard’s approved therapy could be useful in treating much more common disorders including frontotemporal dementia and Crohn’s disease.
Raise the Line · Dr. Bobby Gaspar, Lindsey Smith
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Show Notes
It seems there are news stories every week about the accelerating pace of innovation in gene therapy, but only about 50 therapies have been approved so far by the US Food and Drug Administration. Our guest today, Dr. Bobby Gaspar, leads a UK-based biotech company, Orchard Therapeutics, that developed one of those treatments using gene-modified stem cells in your blood that self-renew, so a single administration can give you potentially a lifelong effect. “Our approach is about correcting those hematopoietic stem cells and allowing them to give rise to cells that can then correct the disease,” explains Dr. Gaspar. The therapy in focus is lenmeldy, the first approved treatment for metachromatic leukodystrophy, also known as MLD, a devastating inherited disorder that affects roughly 600 children worldwide. But Dr. Gaspar is optimistic that learnings from Orchard’s work on MLD could be useful in treating much more common disorders including frontotemporal dementia, Crohn’s disease and others. This highly informative conversation with host Lindsey Smith also explores the importance of newborn screening, community collaboration in advancing clinical trials for rare diseases, and a future in which each gene therapy will be used as a tool for specific applications. “There will be many gene therapies available, some of which will become the standard of care for certain diseases, but it won't be for every disease.”
Mentioned in this episode:
Orchard Therapeutics
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