CRISPR-based therapies (longevity context)
DECRISPR-basierte Therapien (Longevity-Kontext)
CRISPR-based therapies use CRISPR-Cas9, base editors, or prime editors to make targeted, somatic edits to your DNA. The edits can be done ex vivo (in cells taken out of you) or in vivo (directly inside you). The first regulator-approved CRISPR medicine is Casgevy (exagamglogene autotemcel, from Vertex/CRISPR Therapeutics). The FDA approved it on 8 December 2023, and the European Commission in February 2024. It treats sickle cell disease and transfusion-dependent β-thalassemia in patients aged 12 and up, based on the Frangoul et al. NEJM 2021 trial. Longevity-focused uses are further behind. These include reactivating telomerase, knocking out senescence or premature-aging genes, in-vivo PCSK9 base editing (Verve Therapeutics' VERVE-102), and in-vivo ANGPTL3 editing (CRISPR Therapeutics' CTX310). A NEJM 2025 phase-1 readout of CTX310 showed, at the top dose, mean LDL down 49% and triglycerides down 55%. These are early cardiovascular readouts. But for any aging endpoint, they stay preclinical. No CRISPR therapy is approved for an aging indication as of 2026.
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Sources
- Frangoul H, Altshuler D, Cappellini MD, et al.. (2021). CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia. *New England Journal of Medicine*doi:10.1056/NEJMoa2031054
- U.S. Food and Drug Administration. (2023). FDA Approves First Gene Therapies to Treat Patients with Sickle Cell Disease (Casgevy / Lyfgenia)
- CRISPR Therapeutics / European Commission. (2024). European Commission Approves First CRISPR/Cas9 Gene-Edited Therapy Casgevy for SCD and TDT