This week, The Lancet publishes a first-in-human interventional study of gene supplementation therapy for children with AIPL1-associated retinal dystrophy, which causes severe impairment of sight from...

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Here are the latest Gene and Cell Therapy developments: Sarepta’s confirmatory DMD study failed as Elevidys faces scrutiny, with FDA talks planned. Caribou’s allogeneic vispa-cel matched autologous CAR-T benchmarks, as FDA guides a about 250-patient Phase 3. FDA reversed course on uniQure’s AMT-130. Lilly licensed MeiraGTx’s AAV-AIPL1 for LCA4, with planned UK and US filings. Tenaya’s TN-201 trial is on hold to standardize immunosuppression. Intellia reported a death amid severe liver toxicity, pausing trials. Dyno unveiled bn8 AAV. Opus advanced OPGx-LCA5 toward adaptive pivotal testing.

Entered into broad strategic collaboration with Eli Lilly and Company (“Lilly”) in the area of ophthalmology, granting Lilly worldwide exclusive rights to the Company’s AAV-AIPL1 program for treatment of one of the most severe inherited retinopathies, Leber congenital amaurosis 4 (LCA4) Released

Eli Lilly announced a strategic licensing agreement with MeiraGTx granting it worldwide exclusive rights to develop and commercialize AAV-AIPL1, a gene

Eli Lilly is paying $75 million up front for global rights to AAV-AIPL1, a gene therapy on track for U.S. and European regulatory submissions in the rare, inherited eye disorder Leber congenital amaurosis 4, or LCA4. The deal brings to Lilly another late-stage gene therapy for the eyes following the pharma giant’s October acquisition of Adverum Biotechnologies and its lead program for wet AMD.

Eli Lilly is paying $75 million up front for global rights to AAV-AIPL1, a gene therapy on track for U.S. and European regulatory submissions in the rare, inherited eye disorder Leber congenital amaurosis 4, or LCA4. The deal brings to Lilly another late-stage gene therapy for the eyes following the pharma giant’s October acquisition of Adverum Biotechnologies and its lead program for wet AMD.

Eli Lilly partners with MeiraGTx in a $475M deal for a breakthrough gene therapy, AAV-AIPL1, designed to restore vision in children born legally blind.

MeiraGTx (Nasdaq: MGTX) announced a broad strategic collaboration with Eli Lilly on Nov 10, 2025 to develop and commercialize genetic medicines in ophthalmology.MeiraGTx granted Lilly worldwide exclusive rights to its AAV-AIPL1 program for LCA4 and exclusive access to in‑house intravitreal capsids, bespoke promoters (including AI‑generated promoters), and certain riboswitch technology for ocular gene editing. Clinical data reported that 11 children born legally blind gained vision after AAV-AIPL1, with additional developmental benefits described.Financial terms include a $75 million upfront payment, eligibility for over $400 million in milestones, and tiered royalties on licensed products.

Six-year-old Jace Broadbin, born blind due to Leber congenital amaurosis (LCA), a genetic condition caused by mutations in the AIPL1 gene

UK Compassionate Use gene therapy for retinal disease improves vision for boy with LCA4, a form of Leber congenital amaurosis caused by a mutation in the AIPL1 gene.

Our findings indicate that young children with AIPL1-related retinal dystrophy benefited substantially from subretinal administration of rAAV8.hRKp.AIPL1, with improved visual acuity and functional vi...

A genetic therapy to help patients with AIPL1-related retinal dystrophy gets its first in-human tryout, taking cocoa extract does not appear to reduce the rate of AMD occurrence, and OCT may have a use in diagnosing major depressive disorder.

Según Newsweek, un innovador tratamiento génico ha permitido a menores con LCA-AIPL1 recuperar la vista, transformando su calidad de vida.