Epigenetic reprogramming · Phase 1 · Presentation
Life Biosciences ER-100: interim Phase 1 OSK epigenetic reprogramming data to be presented at Eyecelerator @ AAO (1 Oct 2026).
First clinical candidate from Life Biosciences’ Epigenetic Restoration platform (controlled OCT4, SOX2, and KLF4
expression) in optic neuropathies (open-angle glaucoma and NAION). The company will present new interim Phase 1
data in glaucoma on 8 Oct 2026 at 2:10 pm CT. The trial is safety and tolerability first, with exploratory
visual-function endpoints (NCT07290244).
Caveat: Presentation announcement only. Interim results are not yet public. Investigational; not an approved rejuvenation or glaucoma therapy. Distinct from the 2026-09-29 archive note that an ER-100 Phase 1 trial exists.
GlobeNewswire →
ClinicalTrials.gov →
Aging biology · Epigenetics · Review
Gladyshev, Yücel, and Molière: aging as progressive decanalization of cell identity (Nature review, 30 Sep 2026).
A Nature review frames aging as erosion of a developmental regulatory grammar (PRC2 versus H3K4/36 methylation and
RNA polymerase II antagonism). Most somatic age-associated DNA methylation gain maps to PRC2-bound low-methylated
regions. The model aims to explain epigenetic clocks and to suggest architecture-stabilizing interventions.
Caveat: Theoretical review framework with testable predictions. Not a clinical protocol or an approved intervention.
Nature →
Inherited retinal disease · AAV gene therapy · Phase 1/2 Part A
Atsena ATSN-201: 18-month Part A LIGHTHOUSE results in X-linked retinoschisis (30 Sep 2026).
Subretinal AAV.SPR RS1 gene therapy. Part A (n=9 adults): no drug-related serious adverse events or dose-limiting
toxicities. Foveal schisis closure was maintained in 7 of 9 treated eyes at 18 months. Microperimetry improved by
at least 7 dB in 6 of 9 treated eyes versus 0 of 9 untreated eyes. BCVA or low-luminance visual acuity improved by
at least 10 letters in 7 of 9 treated eyes versus 1 of 9 untreated eyes. Pivotal Part C is enrolling, with a BLA
targeted for the second half of 2028.
Caveat: Early Phase 1/2 Part A only; not approved. Company-reported endpoint framing.
Atsena via BioSpace →
Duchenne · Approved gene therapy · Older ambulatory cohort
Sarepta ELEVIDYS: 2-year functional benefit in older ambulatory Duchenne (ages 8–12) at WMS 2026 (30 Sep 2026).
Delandistrogene moxeparvovec in ambulatory patients treated at ages 8–12 (n=25) versus a matched external control
(n=99): the company reported a durable NSAA difference of 3.32 points and timed-function benefit over 2 years.
Safety was described as consistent with prior experience. The same meeting included micro-dystrophin expression
data in children treated at ages 2–3; use under age 4 remains investigational.
Caveat: External-control design for the older ambulatory cohort. ELEVIDYS is already approved in other age bands. This is label and expansion evidence, not a first approval. Verify against the primary World Muscle Society materials and the label.
Sarepta via BioSpace →
Company release (PDF) →
Hearing · Approved gene therapy · Spotlight
Otarmeni (Regeneron): Nature spotlight on the first FDA-approved OTOF gene therapy for DFNB9 deafness (30 Sep 2026).
A Nature spotlight on Otarmeni, Regeneron’s OTOF gene therapy for DFNB9. In the CHORD trial (n=24, ages about
10 months to 16 years), about 80% had improved hearing by 24 weeks and 42% later regained hearing, as the piece
reports it. The FDA approved it in April 2026 as the first gene therapy for OTOF-related hearing loss, under
accelerated approval. The mechanism delivers a functional OTOF gene to inner-ear hair cells, where synaptic
transmission is otherwise the missing piece.
Caveat: Spotlight on an already approved product and ongoing follow-up, not a new pivotal readout today. Nature notes that many Deaf people do not consider this condition a disability that requires a cure, and that people often identify as part of Deaf culture. Not a claim that all hearing loss is a disease to cure.
Nature spotlight →
