Gene Therapy

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.

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.

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.

Multidisciplinary ophthalmic genetics clinic adds efficiency to process.

A recently completed non-human primate study of VGX-0111 demonstrated good tolerability, provided strong transgene expression in the targeted region of the retina, and increased production of the lipids whose decline is associated with macular degeneration.

Data from IG-002, Intergalactic's lead program, demonstrate persistent ABCA4 expression in non-human primate retinas over six months after a single administration, and support plan to advance IG-002 into Phase 1 trials in 2024.

IIT Kanpur licensed the technology to Reliance Life Sciences Pvt. Ltd. and this marks the first time a gene therapy related technology has been developed and transferred from an academic institution to a company in India.