Genes · neurodegeneration (lead)
Nature Aging microprotein atlas of the human frontal cortex in Alzheimer’s disease
Frontal-cortex atlas (transcriptomics + MS + DL-predicted spectra; 600+ postmortem
samples) catalogs 1,067 high-confidence microproteins absent from
reviewed UniProtKB; a 63-aa MP at the MKKS locus is downregulated in AD and
its loss impairs microglial mitochondrial respiration — discovery resource for
neurodegeneration/aging biology, not a therapy.
Caveat: Research atlas / mechanistic follow-up; not a diagnostic or disease-modifying claim.
Nature Aging →
Research briefing →
Salk →
Disease-modifying · SMA
FDA approves ISEMBYLD (apitegromab-mstn) — first muscle-targeted SMA therapy
Cleared for SMA in patients ≥2 years already on SMN2-targeted treatment; Phase 3
SAPPHIRE: +2.2 HFMSE vs placebo at 1 year; broad label (not limited by SMA type /
ambulatory status). Ships “in coming days.”
Caveat: Add-on to SMN2 therapies, not a gene cure; fracture rate higher at 10 mg/kg vs placebo in trial reporting.
BioSpace →
CheckOrphan →
AI drug path · IPF
Rentosertib (AI target + AI molecule) enters Phase 3 IPF (GENESIS-IPF-3)
Insilico: first patient dosed in China Phase 3 (Peking Union / Shanghai Pulmonary,
~9 Sep 2026); company frames as first drug with both AI-identified target (TNIK)
and AI-designed molecule to reach Phase 3. Distinct from the 9/13 proteomic-aging-clocks angle already archived.
Caveat: Phase 3 underway — not approved; prior Phase 2a FVC signal needs confirmatory evidence.
ChemDiv / Insilico note →
Longevity · biomarkers
Caloric restriction improves composite biomarkers-of-aging index (pooled RCTs)
GeroScience (12 Sep 2026): 7 randomized CR trials, n=829; CR improved a
composite index (CRP, IL-6, cystatin C, insulin, GDF-15, TNF-R1); ~48.5% of effect
mediated by weight loss — residual CR-assignment effect after weight adjustment.
Caveat: Surrogate biomarkers ≠ proven lifespan extension in humans.
GeroScience →
Disease-modifying · rare genetics
Joenja (leniolisib) FDA-approved for children 4–11 with APDS
sNDA for 40/50 mg BID in kids ≥27 kg with activated PI3Kδ syndrome (rare genetic
immunodeficiency); first U.S. approved option in this age band; Phase 3 pediatric
open-label: lymphadenopathy ↓ and naïve B cells ↑ over 12 weeks.
Caveat: Rare-disease pediatric label expansion; open-label single-arm pediatric data.
CheckOrphan →
Caveat
Research AI ≠ clinical cure claims
Microprotein atlases, AI-designed Phase 3 candidates, and new FDA labels are
different evidence classes. This site keeps them labeled so a headline never
collapses into “AI cured aging.”
Browse prior editions →
