Gene Therapy

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

ATSN-201 was given regenerative medicine advanced therapy designation to treat X-linked retinoschisis, for which there currently are no approved treatments.

The new data builds upon previously reported 6- and 12-month positive results from adult patients treated in the same study.

Neurologist warns about recurrent optic neuritis attacks.

ENCELTO is the first and only FDA-approved treatment for MacTel.

Researchers indicated that pediatric patients with Leber congenital amaurosis experienced improvements in vision.

Patients who underwent bilateral treatment experienced a higher rate of clinically relevant recovery, the authors reported.

The University of Bristol research team used animal models to compare patient responses to AAV therapy, studying male and female eye cells across different age groups (young, middle-aged and older patients)

Previous research has shown that voretigene neparvovec administered subretinally early in childhood for RPE65-mediated inherited retinal dystrophy achieved encouraging efficacious results.

Laru-zova is a gene therapy currently being investigated for the treatment of patients with X-linked retinitis pigmentosa.

The companies will work together by using the codon-optimized BBS1 AAV9 vector to minimize the vision loss caused by the genetic defects in the BBS1 gene.

The funding will support activities like formal pharmacology and toxicology testing.

ViGeneron also received approval for dose escalation in the ongoing phase 1b clinical trial.

Alon Kahana, MD, PhD, discusses the role of interleukin-6 in thyroid eye disease and the potential of tourmaline Bio's IL-6 inhibitor therapy.