Longevity · Transcriptomic reprogramming · Nature Aging
Transcriptomic screen picks SRSF1 as geroprotective splicing factor (Nature Aging / Wyss / Church)
An interpretable machine-learning transcriptomic age predictor was used to screen cDNA overexpression in aged human fibroblasts. SRSF1 restored youthful transcriptional signatures without loss of cell identity and cut senescence, proteostatic and inflammatory signals in vitro. In aged mice given liver-tropic AAV, it slowed frailty progression by about 44% with about 14% longer median lifespan (n=9–10 per group).
Caveat: Preclinical mouse and fibroblast work; inventors hold related IP and some cofounded RegulaBio; not a human therapy or approved geroprotector; delivery and dosing unresolved.
Nature Aging →
Wyss Institute →
Gene therapy · Inherited retinal disease · Retina in Rio 2026
Opus OPGx-BEST1 and OPGx-LCA5 gene therapy updates (Retina in Rio 2026)
In the low-dose OPGx-BEST1 Phase 1/2 BIRD-1 study, microperimetry retinal sensitivity improved in 3 of 4 evaluable participants, with OCT and structure–function analyses guiding next assessments. OPGx-LCA5 showed sustained visual-acuity and light-sensitivity gains through 24 months in adults and 6 months in pediatric patients; Phase 3 natural-history and treatment cohorts are enrolled, with dosing expected in Q4 2026.
Caveat: Company-presented interim open-label IRD data; investigational AAV gene therapies; not approved; durability and final endpoints still pending.
Opus Genetics →
Gene therapy · Kidney disease · Fierce Biotech
Estuary exits stealth with $103M for ADPKD gene therapies (Fierce Biotech)
Estuary has raised $103M in total, led by its series A, to advance EST-201 (a full-length PKD2 AAV, aiming for the clinic around 2027) and EST-101 (a PC1-derived functional region for PKD1, around 2028) as locally delivered kidney AAV gene therapies for autosomal dominant polycystic kidney disease, aiming beyond slowing progression.
Caveat: Preclinical and financing news only; no human efficacy data; investigational; not yet a disease-modifying therapy.
Fierce Biotech →
AI drug discovery · Aging biology · Deep Origin
Deep Origin × BioAge: AI virtual screen yields wet-lab-validated hits on aging-biology target
Deep Origin’s DODock/DOScore hybrid AI–physics virtual screen delivered multiple structurally distinct hits against a novel target from BioAge’s human longevity data in under three months. BioAge validated the hits in its wet lab and is moving into hit-to-lead optimization for cardiometabolic aging programs.
Caveat: Company partnership milestone; early discovery chemistry only; no clinical candidate or human data from this collaboration yet.
GlobeNewswire →
AI drug discovery · MASH · Cardiomyopathy
AstraZeneca × insitro: Virtual Human AI for MASH and cardiomyopathy targets (pharmaphorum)
AstraZeneca pays insitro $40M upfront, with up to about $1.5B in milestones, to use the Virtual Human platform (machine learning plus genetics, lab models and functional genomics) to find novel targets and leads for MASH and cardiomyopathies. AstraZeneca runs future trials.
Caveat: Discovery alliance announcement; no clinical readouts; target and lead identification only.
pharmaphorum →
