Gene Therapy

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).

Presentation topics will include therapies for age-related macular degeneration, X-linked retinitis pigmentosa and other retinal pathologies

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

AMD treatment may become unrecognisable as gene therapies evolve

Best disease, or vitelliform macular dystrophy, is a rare, inherited retinal condition causing macular degeneration by mutations in the BEST1 gene, leading to progressive vision loss and, in some cases, blindness.

A 12-month follow-up indicated no dose limiting toxicity and no ocular or systemic adverse effects

AAVB-039 is the company’s gene therapy program for Stargardt disease secondary to biallelic mutation in ABCA4.

João Pedro Marques, MD, MSc, PhD discusses a retrospective study of 800 patients with inherited retinal diseases

In a press release, the National Institute for Health and Care Excellence announced the first-ever approval for a mitochondrial disease treatment

Ophthalmology celebrates 50 years of groundbreaking innovations, highlighting anti-VEGF therapies, OCT imaging, and future advancements in eye care.

The Global RDH12 Alliance unites inherited retinal disease advocacy groups including UK-based Eyes on the Future and the US-based RDH12 Fund for Sight

These updates support Ocugen in its efforts to pursue its goal of 3 biologics license applications (BLA) in the next 3 years.

The platform, developed by University College London and Heidelberg Engineering, demonstrated accuracy and precision in early tests

Krystal Biotech initiates EMERALD-1 trial for KB801, a redosable eye drop gene therapy.

AAV204 is a novel adeno-associated virus (AAV) capsid from the AIM capsid library licensed by Abeona from the University of North Carolina at Chapel Hill.

Through the program, gene therapies are developed to treat patients with retinitis pigmentosa caused by pathogenic variants in the MERTK gene.