Longevity, genes,
and disease-modifying AI.

CTX310: one-year durability for a CRISPR therapy targeting ANGPTL3 (NEJM; ScienceDaily 2026-09-27)

One-time CRISPR-Cas9 therapy delivered to the liver, tested in a 15-patient Phase 1 trial for lipid disorders. At the highest dose, LDL fell about 52.5% on average and triglycerides about 47.8% at 12 months. No therapy-related serious adverse events in the first year of follow-up. Presented at ESC 2026, with a durability note in NEJM.

Caveat: Small Phase 1 trial of an investigational therapy, with 15 years of safety follow-up planned. Not an approval.

eCgCas12n: engineered mini Cas12n with about a 60-fold editing boost, plus a base editor that fits in a single AAV (Molecular Systems Biology; Scienmag 2026-09-27)

Adding arginines at structure-guided positions and shortening the guide turned inactive CgCas12n into eCgCas12n, which makes strong indels in mammalian cells. A compact cytosine base editor built on it fits in one AAV9 vector; an in vivo mouse demo on a Dmd stop codon gave about 11% C-to-T edits.

Caveat: Preclinical tool paper (cells and mouse intramuscular injection). Not a human therapy or a cure.

CRISPR_SCD001: clinical-grade, patient-derived CRISPR-Cas9 gene-correction product for sickle cell (Molecular Therapy, online 2026-09-19)

A manufacturing and pharmacology paper reports about 22% correction of the HBB allele. In culture, HbS went down and HbA and HbF were restored. The cells engrafted well in NBSGW mice without notable toxicity, supporting the path to a first-in-human trial (NCT04774536).

Caveat: Preclinical product characterization. Not a clinical efficacy result or an approval.

DNG64-CAR-V: FDA CBER authorizes delivery into the spinal fluid for a second patient with leptomeningeal disease (Aveni / EIN 2026-09-23)

Emergency / Right-to-Try authorization for weekly tumor-targeted gene therapy (DNG64-CAR-V / DeltaRex-G) given into the brain’s ventricles through an Ommaya reservoir. The patient has diffuse midline glioma with leptomeningeal disease. The sponsor calls it the first CBER authorization for direct spinal-fluid delivery of this class of therapy; a Phase I/II trial for leptomeningeal disease is planned.

Caveat: Single-patient expanded access under emergency use. Not a trial efficacy result or an approval.

Genflow GF-1002 / SIRT6: SLAB dog trial met its GRIM-clock primary endpoint (RNS 2026-09-08; Animal Longevity Summit 2026-10-01 to 10-02)

A randomized, blinded trial of 24 beagles older than 10 years across naked-DNA dose groups, an AAV8 group and saline. It met its primary endpoint: a reduction in biological age on the GRIM methylation clock versus control. Full numbers and histology are due at the Animal Longevity Summit in Toronto.

Caveat: Veterinary longevity study disclosed by the company, with no published effect sizes yet. Not a claim about human lifespan.

Research AI ≠ clinical cure claims

A small Phase 1 durability report, a preclinical tool paper, preclinical product characterization, a single-patient emergency authorization, and a company-disclosed veterinary trial are different evidence classes. This site keeps them labeled so a headline never collapses into “AI cured aging.”