Gene Therapy

Mark Pennesi, MD, PhD, reports during ARVO 2021 that preliminary results have shown that AGTC-401 and AGTC-402 seem safe and well tolerated in patients with ACHM.

Friederike Kortuem, MD, MSc, discusses how treatment with voretigene neparvovec-rzyl led to a short-term change in the foveal morphology in a patient with visual impairment that included nyctalopia and decreased visual acuity in early childhood.

According to Applied Genetic Technologies Corp., groups 5 and 6 had a 50% response rate among patients who met the inclusion criteria for the Skyline and Vista trials.

Investigators use an adeno-associated viral vector in a clinical trial to deliver a normal functioning copy of the RPGR gene via subretinal injection.

Approach can unravel causes in MYOC and TBK1 glaucoma.

Studying zebrafish helps unravel mysteries of photoreceptor regeneration .

Studies are uncovering a range of potential treatment options for disorders.

Options for physicians are increasing in availability with fewer burdens for patients.

Physician offers patient counseling pearls for selected retinal diseases.

Option demonstrates stable to improved visual acuity and retinal thickness.

Investigators continue to break new ground, giving hope to patients, physicians.

Investigators observe dramatic decrease in treatment burden seen in OPTIC study.

Treatment may facilitate sight in end-stage retinitis pigmentosa.

Baseline diabetic retinopathy severity is an important predictor of its progression in patients.

Gene therapies to mitigate the effects of mutations that cause blindness are undergoing rapid development. Investigators have found that novel gene vectors reduce the risks associated with these procedures.

Study examining the role of IL-17A in patients with diabetes.

Investigators focus on biophysical method to study protein-protein interactions .

The FDA has granted Fast Track designation to MeiraGTx's AAV-CNGA3 gene therapy product candidate for the treatment of achromatopsia (ACHM).

Neurophth Therapeutics, Inc (Neurophth) and AAVnerGene Inc have announced the launch of a strategic partnership that will grant Neurophth global rights to mutually select adeno-associated virus (AAV) capsids for the creation of the next-generation ophthalmic gene therapy.

A new gene therapy for this patient population targets the ND4 gene mutation.

BIGH3 is an extracellular matrix protein that can act in an autocrine, paracrine manner.

Two teams of researchers from Gyroscope Therapeutics and the University of Pennsylvania are joining forces to explore gene therapy targets for three specific serious eye diseases.

The investigational RNA therapy is designed to address the underlying cause of Leber congenital amaurosis 10 due to a mutation in the CEP290 gene.

ViGeneron and Biogen this week have announced a global collaboration and licensing agreement in which the partners will develop and commercialize gene therapy products based on adeno-associated virus (AAV) vectors that treat inherited eye disease.

Nine pediatric patients with Leber congenital amaurosis show visual improvement in clinical trials.