Gene Therapy

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.

The TEASE-1 trial reveals gildeuretinol's potential to reduce vision loss in Stargardt disease, offering hope for future treatments.

Boyer discusses a potential treatment for diabetic retinopathy and macular degeneration using suprachoroidal injections, offering reduced risks and improved outcomes.

OCU400 is Ocugen’s novel modifier gene therapy for retinitis pigmentosa (RP).

Telomir-1 shows promising results in restoring vision and retinal structure in age-related macular degeneration, marking a significant advancement in treatment.

ABCA4-associated retinopathies include Stargardt disease, retinitis pigmentosa 19, and cone-rod dystrophy 3.

At Retina World Congress 2025, Dilsher S. Dhoot, MD, shares updates on the HELIOS trial and the potential future of tyrosine kinase inhibitors (TKIs)

Insights from a genome-wide association study identify novel genetic loci