Gene Therapy

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.

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.

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

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

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

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.

The trial is evaluating KB803 for the treatment and prevention of corneal abrasions in dystrophic epidermolysis bullosa

Ixo-vec shows sustained efficacy and reduces injection burden through 4 years in clinical trials

Siegfried Priglinger, MD discussed research he presented focused on pediatric patients with inherited retinal diseases with an emphasis on studies into the RPE65 gene.