Gene Therapy

Interim 6-month results from Part B of the LIGHTHOUSE trial show structural and functional improvements consistent with earlier cohorts; pivotal Phase 3 enrollment now underway with BLA filing targeted for 2028.

The ongoing GARDian3 study is designed to assess whether a single subretinal administration of OCU410ST can slow structural disease progression in patients with ABCA4-associated retinopathy.

Chiesi Global Rare Diseases said the agency declined to approve its application and requested additional information for the proposed treatment of Leber hereditary optic neuropathy.

Gene and cell therapies plus AI imaging tools accelerate breakthroughs for rare retinal diseases, spotlighting new trials for RP and Stargardt patients.

Trial shows AAV8 PDE6A gene therapy doesn’t improve retinitis pigmentosa vision in a year and may cause retinal thinning and VA loss.

The trial is evaluating OPGx-MERTK gene therapy for MERTK-related retinitis pigmentosa (RP).

Japan fast-tracks MCO-010 optogenetic eye injection as studies show lasting safety and meaningful vision gains in inherited retinal disease.

The trial is evaluating OCU410 (AAV5-RORA) for the treatment of geographic atrophy (GA) secondary to dry age-related macular degeneration.

OCU410ST uses an adeno-associated virus delivery platform for the retinal delivery of the RORA gene.

The Part B dose-expansion portion is evaluating SB-007 for the treatment of Stargardt disease.

CoTx-101 is intended for the treatment of retinal vascular diseases, such as wet age-related macular degeneration (wAMD) and diabetic macular edema (DME).

RUSH2A is an ongoing natural history study for patients with mutations in the USH2A gene causing Usher syndrome type 2A or nonsyndromic retinitis pigmentosa (RP).

This acquisition will include Adverum’s lead candidate, Ixo-vec, a gene therapy treatment for wet AMD.

AMD treatment may become unrecognizable as gene therapies evolve.

According to the company, FALCON aimed to provide “a better understanding of how autosomal dominant optic atrophy (ADOA) disease parameters change over time to inform potential future interventional clinical trials.”

As ophthalmic technologies move at supersonic speed, AI and gene therapy take center stage.

The FDA describes the RDEA pilot program as a program to support novel endpoint efficacy development for drugs that treat rare diseases.

The company can now initiate the Opti-GAIN (Optimized Geographic Atrophy INterventional) phase 1/2 clinical trial.

Additionally, the company also received Clinical Trial Authorisation (CTA) approval from the UK's Medicines and Healthcare Products Regulatory Agency (MHRA).

Kwangdong, a top 5 pharmaceutical and healthcare company in Korea, is actively involved in research and development innovation, including “transformational late-stage, high-impact technologies.”

In both trials, Beacon’s lead program, laru-zova, was found to be well-tolerated by SKYLINE participants through month 36 and DAWN patients at month 9 or longer.

Alfredo Sadun, MD, PhD, details an unexpected outcome from a gene therapy in development for Leber hereditary optic neuropathy (LHON).

Kriya is developing gene therapies targeting chronic diseases such as geographic atrophy, trigeminal neuralgia, and type 1 diabetes.