Gene Therapy

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

Data show transfection in both eyes despite unilateral injection

Infrastructure across Europe needs a major overhaul to optimise education and patient access in groundbreaking treatments

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.

Bilateral distribution of the transgene seen with unilateral injection

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.

For retinal specialists grappling with diseases once thought “untreatable,” gene therapies present a wide range of viable options

In this EyePod® episode, David Hutton sits down with Shankar Musunuri, CEO of Ocugen to discuss updated data for OCU400.

Kriya is developing a one-time gene therapy designed to block complement C3 and C5, which are clinically validated substrates targeted by FDA approved therapies, to delay the progression of geographic atrophy.

Patients with HTG had significantly fewer mtDNA copies per nuclear DNA.

According to the companies, the collaboration has the potential to benefit patients who have suffered from specific, chronic ocular diseases.

According to the company, NFS-05, utilizes a gene therapy approach that delivers an AAV vector containing the OPA1 gene into the vitreous cavity.

ATSN-201 leverages novel spreading capsid to overcome challenges associated with intravitreally delivered AAVs in the treatment of XLRS.

VG901 is a gene therapy for CNGA1-associated RP.

Data may inform clinical associations, mechanistic research, trial design.

The gene therapy company announces its closure less than 2 years after the company launch in 2021.

According to the University of Barcelona, the agreement between DBGen Ocular Genomics and Novartis will allow the accurate diagnostics of the genetic bases of these two pathologies to be conducted by mass sequencing of gene panels designed by the UB spin-off.

For visual recovery to occur, there must be photoreceptors left that can be rescued.

The London-based company will use regenerative medicine to fight ocular disease.