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The Drug Discovery World Podcast
DDW Highlights: 20 November 2023
Nov 20, 2023
This podcast highlights the first regulatory approval for Casgevy, a gene therapy using CRISPR gene editing for sickle cell disease. Other breakthroughs in cell and gene therapy are discussed, including Verve 101 for cardiovascular disease, Axicab2Gin Silo-Lucial for lymphoma, Cascavii for sickle cell disease and beta thalassemia, AB 1002 for advanced heart failure, and global collaboration for CAR-T DDBCMA.
17:01
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Quick takeaways
- Verve Therapeutics's gene editing medicine, Verve 101, shows promise in lowering LDL-C cholesterol levels in patients with familial hypercholesterolemia.
- Kite's CAR-T cell therapy, Axicabtagene Ciloleucel, significantly improves overall survival in patients with relapsed or refractory large B cell lymphoma.
Deep dives
Gene editing treatment shows promise in reducing cholesterol levels
A Phase 1B trial of Verve Therapeutics's Verve 101 showed significant reductions in LDL-C cholesterol levels and blood PCSK9 protein levels in patients with heterozygous familial hypercholesterolemia (HEFH). Verve 101 is an investigational gene editing medicine designed to inactivate the PCSK9 gene in the liver and durably lower LDL-C cholesterol levels. The trial data suggested successful editing in the intended genomic target, indicating the potential for single-course gene editing as a new approach for treating patients with HEFH.
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