Longevity, genes,
and disease-modifying AI.

UAB doses world’s first patient in investigational BAG3 gene therapy for dilated cardiomyopathy (2026-09-24)

First patient dosed at UAB in a first-in-human Phase 1/2 trial of a one-time IV gene therapy from Alexion, AstraZeneca Rare Disease (NCT07218887) for BAG3 mutation–associated dilated cardiomyopathy. The aim is to treat the genetic root cause rather than rely only on standard heart-failure medication.

Caveat: Investigational, early-phase; safety and efficacy not established; no outcome data yet.

Wiskott-Aldrich syndrome: NEJM long-term results for etuvetidigene autotemcel (etu-cel / Waskyra)

Autologous lentiviral HSPC gene therapy; 27 patients treated at a median age of ~2.6 years, followed ≥5.7 years (some >13). Survival 96% at 1 and 5 years; marked reduction in severe infections and bleeding; all patients stopped immunoglobulin replacement. The release reports no product-attributable adverse events and no insertional oncogenesis. EU and US authorization Dec 2025–Jan 2026 (Fondazione Telethon / SR-Tiget).

Caveat: Ultra-rare disease; conditioning risks apply; check the full NEJM paper and product label. Distinct from the earlier CRISPR-Cas9-AAV6 WAS item in our archive (2026-09-16).

Ocugen OCU400: Bahamas LARTA provisional approval, priority designation, and expanded access program for retinitis pigmentosa (2026-09-25)

OCU400, an NR2E3 modifier gene therapy designed to work across RP regardless of causal gene (>100 genes), received provisional approval and priority designation from The Bahamas’ LARTA Board. Ocugen plans an expanded access program, aiming to treat the first patient within ~90 days of full LARTA approval. Phase 3 ongoing; topline expected 1Q 2027; BLA planned 2Q 2027. Holds FDA RMAT/ODD and EMA OMPD.

Caveat: LARTA provisional approval is not FDA approval and not US commercial availability; Phase 3 efficacy data pending.

Rocket RP-A501: FDA alignment to complete pivotal Phase 2 in Danon disease (2026-09-15)

Enrollment continues under a modified protocol: 12 males at the recalibrated dose of 3.8×10¹³ GC/kg. Co-primary endpoints at 12 months are myocardial LAMP2 protein expression and a ≥10% reduction in LV mass index, intended to support a potential accelerated approval pathway. Dosing expected to finish by mid-2027.

Caveat: FDA alignment is not an approval; the program’s prior safety history matters when reading this.

Caloric restriction improves a composite aging-biomarker index in older adults (GeroScience, pooled RCTs)

Pooled analysis of 7 randomized trials (n=829; published 2026-09-12) using a TAME-style composite index — CRP, IL-6, cystatin C, insulin, GDF-15, and TNF-R1. About 48.5% of the effect was explained by weight loss, suggesting some signal independent of weight.

Caveat: The index is not a qualified surrogate endpoint; trials were short; not a lifespan claim.

Research AI ≠ clinical cure claims

A first-in-human dosing, long-term follow-up of an approved gene therapy, a non-US provisional approval, FDA protocol alignment, and a biomarker-index analysis are different evidence classes. This site keeps them labeled so a headline never collapses into “AI cured aging.”