Gene Therapy

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.

Clinical judgment is not always sufficient to determine whether a patient has an inherited corneal disorder.

Groundbreaking treatment for patients with bullous keratopathy approved in Japan.

According to the company, its new 20,000-square-foot facility in Hayward, California, includes cGMP suites, developmental and testing laboratories, equipped with single use bioreactors.

Surgeon details training, experience with procedure that offers hope to patients.

According to data presented at the Association for Research in Vision and Ophthalmology annual meeting in New Orleans, composite efficacy data validate MCO-010 as a potential treatment for RP.

Under a compassionate use program, the topical application of B-VEC to the eye was well tolerated and patient showed significant improvement of visual acuity and complete corneal re-epithelization. The company presented data at the Association for Research in Vision and Ophthalmology annual meeting in New Orleans.

In a presentation at ARVO 2023 in New Orleans, the company noted data in a nonhuman primate model of non-arteritic anterior ischemic optic neuropathy demonstrates the ability to restore visual function after delivery of a novel gene therapy.

Physician outlines the latest emerging technologies to treat the condition.

A team of researchers has uncovered a mechanism that causes blindness, could lead to targeted therapies.

According to National Eye Institute researchers, the variants generate malformed proteins that alter the stability of the membrane attack complex, which may drive a chronic inflammatory response in the retina.

Ophthalmologist looks ahead amid company’s advances over past 18 months.

According to the company, it is the first approval in the world for allogeneic cell therapy to treat corneal endothelial disease.

Researchers are using a new, highly versatile form of CRISPR-based genome editing with the potential to correct a wide variety of disease-causing genetic mutations.