Prime editing · RNA chemistry (lead)
Dense RNA-modified epegRNAs push in vivo prime editing toward clinical doses (Nature Biomedical Engineering)
Sem⁺-epegRNA (dense 2′-O-methyl / 2′-fluoro / phosphorothioate across spacer, scaffold, RT template, PBS, evopreQ1)
reached ~67% bulk mouse-liver Pcsk9 editing after one LNP dose; at ~1 mg/kg ~51% vs ~0.6% for end-modified pegRNA,
with ~96% serum Pcsk9 and ~60% cholesterol drops — also lifted other CRISPR modalities without clear off-target
inflation across 78 predicted sites.
Caveat: Mouse/preclinical delivery paper — not a human therapy; durability, immunogenicity, and multi-organ safety unproven.
Nature BME →
CRISPR Medicine News →
Aging clocks · IPF (Phase 2a)
AI-designed rentosertib shifts six proteomic aging clocks in a phase 2a IPF subset (Nature Biotechnology)
Retrospective Olink proteomes from a 12-week rentosertib (TNIK) IPF trial (n≈42 with complete samples) showed
all six independent proteomic clocks (ProtAge, OrganAge variants, PAC, ipfP3GPT, PAOPAC) predicting lower
biological age on treatment vs placebo — peak ~week 4, roughly ~3–4 years on strongest doses — supporting
dual-purpose disease + geroprotective trial readouts.
Caveat: Biomarker signal only — small IPF cohort; cannot separate anti-fibrotic vs true anti-aging effects; not validated surrogate for lifespan/healthspan; not an approved longevity indication.
Nature Biotechnology →
Artificial Science →
CAR-T · generative AI (preclinical)
MSK generative-AI de novo CAR binders outperform clinical BCMA scFv in mice (Nature Biomedical Engineering)
Lareau lab pipeline (million candidates → ranked binders → wet-lab + CARPNN) built AI-designed CAR binders
that controlled BCMA+ tumors in mice better than FDA-approved BCMA CAR binders; work also explains CD19 design
failures and premature activation via charge/alanine rules.
Caveat: Preclinical mouse/proof-of-concept — not cleared to change CAR-T practice; human trials pending.
MSK news →
Nature BME DOI →
CRISPR phage · BSI prevention (Phase 2)
SNIPR001 CRISPR-armed phage enters Phase 2 for E. coli BSI prevention in HSCT (NCT06938867)
First patient enrolled in Part 2 of Phase 1b/2a; +42 patients → total n=66 (25 already dosed in Part 1);
sites now include MSK and Dana-Farber; top-line Phase 2 expected H2 2027 — enrollment/efficacy-path milestone,
not an efficacy readout.
Caveat: Investigational; Part 2 is for efficacy look; no approval; results not yet available.
BioSpace →
Neoantigen mRNA · melanoma (Phase III)
ML-designed individualized neoantigen mRNA (intismeran / V940) + pembrolizumab meets Phase III RFS primary in resected melanoma
INTerpath-001 (n=1,137, stage IIB–IV post-resection) interim: intismeran autogene + pembro improved
recurrence-free survival (primary) and distant metastasis–free survival vs pembro alone — first Phase III win
for an individualized neoantigen / mRNA cancer therapy; neoantigen selection uses deterministic ML/AI on tumor NGS.
Caveat: Company interim; full data pending medical meeting + regulatory review; not yet labeled standard of care; OS and other secondaries still maturing.
ASCO AI →
Caveat
Research AI ≠ clinical cure claims
Preclinical prime-editing chemistry, proteomic aging-clock shifts, AI CAR binders, investigational phage,
and interim Phase III oncology readouts are different evidence classes. This site keeps them labeled so a
headline never collapses into “AI cured aging.”
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