VERVE-101 and VERVE-102 are an experimental gene therapy developed by Verve Therapeutics that targets the PCSK9 gene and is intended to reduce blood cholesterol levels. It works on 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.[1][2][3][4][5] Both treatments use the same RNA gene editing technology, but they use a different lipid nanoparticle delivery vehicle.[6]
References
edit- ^ "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. 10 (2): 87–88. doi:10.1093/ehjcvp/pvad095. PMID 38031331.
- ^ 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.
- ^ "Verve pauses gene therapy trial, switches delivery agent". Chemical & Engineering News.