Longevity · AMPK · Preclinical models
Direct AMPK activator 991 extends lifespan in yeast, worms, and flies (Aging Cell; UKRI / MRC LMS, 29–30 Sep 2026).
First demonstration that a direct small-molecule AMPK activator (compound 991) prolongs lifespan across three distant
model organisms, in some cases by more than 25%, by switching cells into an energy-saver metabolic state.
Caveat: Invertebrate and yeast models only. Authors say the field is still a long way from anti-aging clinical trials in humans; the next step is mouse work. Not a clinical longevity product.
UKRI / MRC LMS →
EurekAlert →
Huntington’s disease · AAV gene therapy · Phase I/II update
uniQure AMT-130 (ifezuntirgene inilparvovec), updated Phase I/II Huntington’s data at 36 and 48 months (29 Sep 2026).
One-time striatal AAV gene-silencing therapy. An expanded high-dose 36-month analysis (n=15) reported about 80% slowing
on cUHDRS versus an updated ENROLL-HD external control (nominal p=0.005) and about 67% on TFC (nominal p=0.011). At
48 months (n=12), cUHDRS slowing of 44% did not reach significance (p=0.144); TFC showed 61% slowing (nominal p=0.008).
A BLA was already submitted under the accelerated pathway on earlier 36-month data; these new results were not in that BLA.
The 36-month analysis, the 48-month analysis, and the submitted BLA are different evidence classes.
Caveat: External-control design with rising missingness at 48 months; mixed durability; not an approved disease-modifying HD therapy.
uniQure via BioSpace →
Muscle aging · Mitochondria · Preclinical
Cardiolipin loss drives aging muscle fiber-type shift via ERRγ; restoring Crls1 rescues premature mortality in mice (Nature Aging, 29 Sep 2026).
An age-associated decline in the mitochondrial membrane lipid cardiolipin (and the synthase Crls1/CRLS1) in mouse and
human skeletal muscle links mitochondrial dysfunction to the glycolytic-to-oxidative fiber shift via mitonuclear ERRγ
signaling. Restoring Crls1 in inducible knockouts reestablishes cardiolipin, begins reversing atrophy, and fully rescues
premature mortality in the model.
Caveat: Mechanistic and preclinical. Not a human therapy.
Nature Aging →
Delivery · Lipid nanoparticles · Preclinical
LC-1 ionizable lipid: a cargo-sized LNP screen extends CRISPR/ABE delivery beyond the liver (Nature Biotechnology, 28 Sep 2026; covered 30 Sep).
A Toronto team screened 384 lipids with gene-editor-sized RNA (about 5.7 kb) rather than short luciferase reporters.
The lead ionizable lipid, LC-1, delivered Cas9 and adenine base editors in mice to hepatocytes (intravenous), neurons
(intrathecal), and lung epithelium (intratracheal) at efficiencies several-fold above ALC-0315 and LP-01. It also edited
Pcsk9, CFTR R553X, and Ube3a-ATS in models.
Caveat: Preclinical mouse and humanized-liver work. A delivery advance is not an approved therapy.
Nature Biotechnology →
CRISPR Medicine News →
Lipid disorders · CRISPR trial enrollment · Access
OHSU enrolls first West Coast participant in the CTX310 CRISPR ANGPTL3 cholesterol trial (OHSU News, 29 Sep 2026).
Site-level enrollment for the multicenter open-label CRISPR-Cas9 lipid-nanoparticle trial knocking down hepatic ANGPTL3
(CTX310). OHSU is the only West Coast center among about 17 worldwide. Adults 18–75 with medication-resistant high LDL
or triglycerides.
Caveat: Enrollment and access story, not new efficacy data. Distinct from the prior NEJM/ESC one-year durability coverage already in the 2026-09-28 archive. Still investigational.
OHSU News →
