
FYI - For Your Innovation A New Era Of Medicine | The Brainstorm EP 114
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Dec 17, 2025 Shea, a genomics and gene-editing expert, dives into revolutionary CRISPR advancements. The discussion highlights the shift from ex vivo therapies to more efficient in vivo treatments for common conditions like cardiovascular diseases. Shea reveals how lipid nanoparticles could simplify patient experiences with potentially single-dose treatments. They explore the vast market implications and potential savings in healthcare costs, while considering the allure of one-time genetic edits versus ongoing statin use. The future of gene editing holds exciting promise as we rethink treatment paradigms for widespread diseases.
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In Vivo Editing Shrinks Treatment Journeys
- In vivo gene editing replaces complex ex vivo workflows by editing cells inside the body using lipid nanoparticles.
- This could convert year-long, inpatient journeys into single outpatient infusions and vastly broaden treatable diseases.
Casgevi Shows Ex Vivo Tradeoffs
- Casgevi (CRISPR for sickle cell) is an ex vivo success that requires cell collection and conditioning chemotherapy.
- Shea contrasted that year-long, intensive journey with the envisioned single-infusion in vivo approach.
Mimicking Natural Protective Variants
- Editing the liver to mimic natural loss-of-function variants can lower lipids and reduce coronary artery disease risk.
- Partial editing (e.g., ~70%) could translate to meaningful population-level risk reductions by copying protective genetic profiles.
