Verve Therapeutics
| Industry | |
|---|---|
| Founders | |
| Headquarters | Boston, Massachusetts, U.S. |
| Products | VERVE-101 |
Verve Therapeutics is a Boston-based biopharmaceutical company which is developing cholesterol lowering gene therapies for individuals with rare genetic disorders.[1][2][3][4][5][6][7]
VERVE-101
Verve's investigational drug, VERVE-101, is currently undergoing clinical trial for treatment of heterozygous familial hypercholesterolemia (HeFH). The drug targets and edits the PCSK9 gene which plays a role in HeFH. PCSK9 is the same protein as the cholesterol-lowering drugs known as PCSK9 inhibitors but, unlike them, is permanent. It works via base editing, a form of CRISPR gene editing. It is one of the first gene therapies that could be beneficial to a wider segment of the population, in contrast to earlier gene therapies that were developed to treat a rare genetic disorder.[8][9][10][11][12]
References
- ^ Arnold, Carrie (7 December 2023). "11 clinical trials that will shape medicine in 2024". Nature (journal). doi:10.1038/s41591-023-02781-y. Retrieved 29 Jan 2024.
- ^ Naddaf, Miryam. "First trial of 'base editing' in humans lowers cholesterol — but raises safety concerns". Nature (journal). doi:10.1038/d41586-023-03543-z. Retrieved 29 January 2024.
- ^ Powell, Alvin (12 December 2023). "Gene-editing treatment could replace cholesterol meds". Harvard Gazette. Retrieved 29 January 2024.
- ^ Kaiser, Jocelyn (12 November 2023). "Base editing, a new form of gene therapy, sharply lowers bad cholesterol in clinical trial". Science (journal). Retrieved 29 January 2024.
- ^ Stein, Rob (12 November 2023). "For the first time, gene-editing provides hints for lowering cholesterol". NPR. Retrieved 29 January 2024.
- ^ Kolata, Gina (12 November 2023). "New Gene Editing Treatment Cuts Dangerous Cholesterol in Small Study". New York Times. Retrieved 29 January 2024.
- ^ Jarvis, Lisa (13 November 2023). "Crispr Offers Hope for a One-Time Cholesterol Drug". Bloomberg. Retrieved 29 January 2024.
- ^ "Next up for CRISPR: Gene editing for the masses?". MIT Technology Review. Retrieved 5 December 2023.
- ^ Philippidis, Alex (1 January 2023). "StockWatch: Verve Investors Accentuate the Negative as Shares Tumble, Then Recover: Safety concerns propel 40% drop before stock bounces back as analysts, researchers focus on VERVE-101's positive human proof-of-concept data". GEN Edge. 5 (1): 808–813. doi:10.1089/genedge.5.1.153. S2CID 265960415.
- ^ Lewis, Basil S (29 November 2023). "First-in-human trial of PCSK9 gene editing therapy for lowering cholesterol: a new frontier in cardiovascular pharmacotherapy? News from AHA". European Heart Journal - Cardiovascular Pharmacotherapy. doi:10.1093/ehjcvp/pvad095.
- ^ Knutsen, Ashleen (1 March 2022). "Opening New Lines of Attack against Cardiovascular Disease: Developers of cardiovascular drugs hope to disrupt newly identified molecular mechanisms by deploying highly specific small-molecule drugs and gene therapies". Genetic Engineering & Biotechnology News. 42 (3): 62–65. doi:10.1089/gen.42.03.19. S2CID 247339058.
- ^ Lee, Richard G.; Mazzola, Anne Marie; Braun, Maurine C.; Platt, Colin; Vafai, Scott B.; Kathiresan, Sekar; Rohde, Ellen; Bellinger, Andrew M.; Khera, Amit V. (17 January 2023). "Efficacy and Safety of an Investigational Single-Course CRISPR Base-Editing Therapy Targeting PCSK9 in Nonhuman Primate and Mouse Models". Circulation. 147 (3): 242–253. doi:10.1161/CIRCULATIONAHA.122.062132. PMID 36314243.
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