Inherited Retinal Diseases

According to the company, VG901 is designed to deliver a functional CNGA1 gene to retinal photoceptor target cells in patients diagnosed with retinitis pigmentosa. It has been granted Orphan Drug Designation by FDA.

According to the company, the Phase 3 study will have a sample size of 150 participants—one arm of 75 participants with the RHO gene mutation and the other arm with 75 participants that are gene agnostic.

Stargardt disease is the most common inherited retinal dystrophy (causing blurring or loss of central vision) in both adults and children.

The new cohort will add patients living with IRDs caused by mutations in multiple genes to the already existing cohort of patients with IRDs caused by single gene mutations.

In this cohort of 3 adult patients, there was positive safety and efficacy data.

MCO-010 demonstrated a statistically significant improvement of best-corrected visual acuity (BCVA) at week 52.

In a recent study led by Steven Pittler, PhD, and his team at the University of Alabama Birmingham (UAB), the role of modifier genes in retinitis pigmentosa type 59 (RP59) was meticulously examined.

Gildeuretinol halts Stargardt disease progression for up to 6 years.

In the study, published in Nature Biotechnology, researchers used their new system to correct disease-causing mutations in the eyes of two mouse models of genetic blindness, partially restoring their vision.

Lowe syndrome, a genetic disease, affects about 1 in 500,000 people in the general population.

According to the company, Stargardt disease is an orphan blindness disease that affects approximately 35,000 people in the United States.

The authors suggested genetic mutation may relax the body’s defences and allow harmful bacteria to reach the eye.

The treatment is a minimally invasive intravitreal injection that can be performed with a topical anesthetic.

A new initiative from Prevent Blindness will kick off to provide free educational resources on retinopathy of prematurity.

Sruthi Arepalli, MD, discusses the case of a patient with syphilis and sheds light on the complexities of diagnosing a masquerading syndrome.

The free, online event will feature presentations from Rachel Huckfeldt, MD, PhD, and Rachelle Lin, OD.

According to the organization, GYROS results will help researchers design clinical trials for an emerging gyrate atrophy gene therapy.

According to the company, AGTC-501 was generally safe and well-tolerated and showed improvements in visual function at the 12-month analysis. The Phase 2/3 VISTA trial for AGTC-501 in XLRP expected to begin in in the first half of 2024.

According to the company, ACDN-01 is the first-ever RNA exon editor to enter clinical development and the only clinical-stage therapeutic targeting the genetic cause of Stargardt disease. Ascidian expects to initiate enrollment in Phase 1/2 STELLAR study in the first half of 2024.

According to the company, favorable safety and tolerability profiles were observed with the first 2 SPVN06 doses across 6 patients. The exploration of SPVN06 in geographic geographic atrophy is set to begin in 2024.

In his presentation, Eichenbaum defined gene therapy and described its use in common retinal disease.

The FDA previously granted Regenerative Medicine Advanced Therapy (RMAT) designation and orphan drug designation to ATSN-101 for the treatment of LCA1.

According to the company, all three patients treated for two years or more have remained free of symptoms and disease progression while taking gildeuretinol.

Updates include expected timelines for trials for the treatment of wet AMD, DME, GA, and inherited retinal diseases.

A specialist in the field of ophthalmology and gene therapy, Dr Girach’s appointment enhances SpliceBio’s leadership team as it accelerates lead program targeting Stargardt disease towards clinical development.

Catch up on a few of our top stories and ones you may have missed in 2023

During a multidisciplinary meeting with FDA, based on preliminary results from an ongoing Phase 1/2 study, the company received alignment on key points of the Phase 3 study design.

A genome topology map of human retina development lays the foundation for understanding diverse clinical phenotypes in simple and complex eye diseases.

According to the company, the Regenerative Medicine Advanced Therapy designation will help expedite the development of new regenerative medicines.

The partnership will further the effort to bring VGX-0111, a gene therapy treatment for AMD to patients and providers.