Gene Therapy

OCU410ST is a modifier gene therapy candidate being developed for Stargardt disease, which affects approximately 100,000 people in the United States and Europe combined.

The IND approval will allow the company to initiate a Phase I/IIa clinical trial for its gene therapy treatment targeting wet Age-related Macular Degeneration (AMD) including Polypoidal Choroidal Vasculopathy (PCV).

Ocugen is developing OCU410 as a 1-time gene therapy for the treatment of geographic atrophy. It utilizes an AAV delivery platform for the retinal delivery of the ROR Related Orphan Receptor A gene.

Prevent Blindness provides free resources on Stargardt disease, including a webpage, fact sheets, expert interview video and social media graphics, to educate the public and professionals on the inherited retinal disease.

Researchers note the G90D mutation in rhodopsin is known to produce background electrical noise to desensitize rods, but the nature of the ‘noise’ and its precise molecular source have not been resolved for almost 30 years.

Simply slowing disease progression is no longer enough.

Despite recognition of lenticular glutathione’s importance in lens formation for decades, why it declines with age remains a mystery.

The data will be presented in oral session at the 2024 American Society of Gene & Cell Therapy meeting May 7-11 in Baltimore, Maryland.

CRISPR manipulation presents opportunities and challenges.

Shift could lead to changes in the current management of retinal diseases

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.

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.

OCU410 is a modifier gene therapy candidate being developed for geographic atrophy, an advanced stage of dry age related macular degeneration.

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.

According to the company, its proprietary non-viral gene therapy platform with minimally invasive delivery technology is providing long lasting gene expression and favorable distribution in the retina.

ECP105 has been developed for the recovery of post-surgical treatment of glaucoma.

The trial of esonadogene mvoparvovec (Opvika) will take place in the US for the treatment of Leber hereditary optic neuropathy caused by ND4 mutation (ND4-LHON).

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

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.

Genetic testing is proving to be an evolving technology for ophthalmologists.

David A. Eichenbaum, MD, discusses the promising aspects of gene augmentation for treating common retinal diseases, addresses concerns about perpetual protein production, and considers the potential positive impact on patients by reducing treatment burdens.

David Eichenbaum, MD, discussed the status of the technology at the Retina 2024 meeting in Maui.

According to the company, OCU410 is a modifier gene therapy product candidate being developed for dry AMD

The improvement of accessibility and education could drive changes in outcomes.

ATSN-101, a gene therapy for GUCY2D-associated Leber congenital amaurosis, has demonstrated clinically meaningful improvements in vision at the highest dose with no drug-related serious adverse events 6 months post-treatment in ongoing Phase I/II clinical trial.

According to the company, OCU410ST uses an AAV delivery platform for the retinal delivery of the RORA (RAR Related Orphan Receptor A) gene.

The issuance provides patent protection in the U.S. for therapeutics incorporating the optimized ELOVL2 transgene until 2041. The patent is being prosecuted in the European Union and other countries around the globe. The company said the patent adds value to its lead program in dry AMD.

Technology allows partitioning of sequencing reads into 2 parental genome data sets.