Longevity, genes,
and disease-modifying AI.

FDA approves AQNEURSA (levacetylleucine) as first treatment for ataxia in ataxia-telangiectasia

First U.S. (and worldwide) approved therapy for ataxia in A-T (adults and pediatric patients ≥15 kg); ATM-related rare genetic neurodegenerative disorder. RCT NCT06673056 (n=73) showed better fSARA vs placebo; second indication after NPC (2024).

Caveat: Treats ataxia symptoms — not a cure for A-T; embryo-fetal risk; common AEs include fall, skin laceration, UTI.

FDA approves Inluriyo (imlunestrant) + Verzenio (abemaciclib) for ESR1-mutated ER+/HER2− advanced breast cancer

All-oral SERD + CDK4/6 after endocrine progression; EMBER-3 ESR1-mut subgroup (n=159) mPFS 11.1 vs 5.5 mo vs imlunestrant alone; Guardant360 CDx companion for ctDNA ESR1.

Caveat: Subgroup / precision-oncology label context; OS immature; Verzenio warnings (diarrhea, neutropenia, ILD, VTE, etc.).

FDA updates WINREVAIR (sotatercept) label with Phase 3 HYPERION data in newly diagnosed PAH

Activin signaling inhibitor; HYPERION (N=320) — added to background Rx within ~1 year of PAH diagnosis cut clinical-worsening risk ~76% (HR 0.24) vs placebo in FC II/III intermediate–high risk.

Caveat: Label expansion / evidence update for earlier use — not a gene therapy; interpret within PAH risk stratification and background therapy.

BEAM-302 in vivo base editing for severe AATD — updated Phase 1/2 (ERS 2026): durable AAT restoration; pivotal 60 mg cohort advancing

First-in-human in vivo base editing for AATD; 60 mg single dose: total AAT above ~11 µM protective threshold (≈14.4 / 13.5 µM Parts A/B); ~84% Z-AAT drop; corrected M-AAT dominant; generally LNP-consistent safety.

Caveat: Early-phase / company-presented data — not approved; pivotal cohort ongoing; not a general “cure aging” claim.

GRAIL Galleri MCED — FDA Molecular & Clinical Genetics Panel AdComm set for 2026-09-23 to review PMA

Methylation + AI multi-cancer early detection blood test; Breakthrough Device; PMA supported by PATHFINDER 2 (~25k) + NHS-Galleri intervention-arm data; intended adjunct to (not replacement for) guideline screening.

Caveat: Advisory committee / pre-approval — no PMA decision yet; company claims; verify against FDA briefing materials when posted.

Research AI ≠ clinical cure claims

FDA approvals for rare neurologic and oncology indications, PAH label evidence updates, early-phase in vivo base editing, and pre-approval MCED AdComm reviews are different evidence classes. This site keeps them labeled so a headline never collapses into “AI cured aging.”