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 DNA either ex vivo (in harvested cells) or in vivo (directly in the patient). The first regulator-approved CRISPR medicine, Casgevy (exagamglogene autotemcel, Vertex/CRISPR Therapeutics), was approved by the FDA on 8 December 2023 and by the European Commission in February 2024 for sickle cell disease and transfusion-dependent β-thalassaemia in patients aged 12+, based on the Frangoul et al. NEJM 2021 trial. Longevity-oriented applications — telomerase reactivation, knockout of senescence or progeroid genes, in-vivo PCSK9 base editing (Verve Therapeutics VERVE-102) and in-vivo ANGPTL3 editing (CRISPR Therapeutics CTX310; NEJM 2025 phase-1 readout showed, at the highest dose, mean LDL −49 % and triglyceride −55 % reductions) — have produced phase-1 cardiovascular readouts but remain preclinical for any aging endpoint; no CRISPR therapy is approved for an aging indication as of 2026.
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