Gene Therapy

Viridian Therapeutics unveils positive data from its ongoing Phase 1/2 trial evaluating low dose VRDN-001 in patients diagnosed with TED.

According to the company, gene and protein expression of cytokines and chemokines after latanoprost+benzalkonium chloride treatment administered via the company’s microdose formulation decreased inflammation from preserved glaucoma solutions compared to drops.

FT-001 is administered by a one-time injection into the subretinal space of the eye that delivers a functional copy of the human RPE65 gene to the nuclei of the patient’s retinal cells.

The technique provides model for studying genesis of age-related macular degeneration and other eye diseases.

Janey Wiggs, MD, PhD, associate chief of Ophthalmology Clinical Research at Massachusetts Eye & Ear discusses genetics of glaucoma, providing an overview of what is known about genes and gene mutations that can cause or contribute to glaucoma.

According to a news release, Opus will advance preclinical development programs for BEST1– and RHO-related retinal diseases. The deal expands the company’s addressable patient population for its novel treatments for rare inherited retinal diseases.

The Cell and Gene Therapy Center will combine a team of 100 researchers to capitalize on the company’s 35 years of cell and gene therapy experience.

According to researcher, the treatment offers improved retinal sensitivity, visual function, and mobility.

OPGx-001 is Opus’ first program to enter clinical evaluation and is designed to address vision loss due to mutations in the LCA5 gene, which causes one of the most severe forms of early-onset blinding disease Leber congenital amaurosis.

Investigators who are examining end-stage disease see significant visual recovery in 2 patients.

The therapy offers improved retinal sensitivity, visual function, as well as improvements in mobility.

Investigators who are examining end-stage disease see significant visual recovery in 2 patients.

The novel genetic engineering approach, tested in mice and laboratory-grown nerve and light-receiving cells, will initially have research applications.

According to National Institutes of Health/National Eye Institute researchers, the study may help lead to gene therapy for rare inherited blinding disease.

Replay is launching Eudora, an HSV gene therapy company that will target genetic retinal diseases.

A previously uncharacterized area of the protein known as RPE65 spontaneously turns spiral-shaped when it encounters intracellular membranes, or thin structures that surround different parts of a cell.

Jeff Nau, PhD, MMS, President and CEO of Oyster Point Pharmaceuticals, provides an appraisal of the company's pipeline as well as updates on recent innovations to hit the market.

According to University of California, Irvine researchers, current preclinical successes and clinical genome editing approaches for treating inherited retinal degenerative disease and stresses there is hope that in vivo gene editing will be the future treatment paradigm for these diseases.

Nanoscope Therapeutics Inc. announced the FDA has granted Fast Track Designation to MCO-010, an ambient-light activatable multi-characteristic opsin optogenetic monotherapy to restore vision in blind patients, for the treatment of retinitis pigmentosa via intravitreal injection.

According to a study by researchers at the Scheie Eye Institute in the Perelman School of Medicine at the University of Pennsylvania, patients’ low-light sensitivity improved by factors of thousands in a clinical trial.

Nanoscope Technologies LLC announced this week it has received Direct Phase II SBIR grant from National Institutes of Health for developing an innovative approach to autonomously regulate pressure for treatment of Glaucoma in a gene-agnostic manner.

In an announcement at the American Academy of Ophthalmology’s 2022 annual meeting at McCormick Place in Chicago, the company noted that results from Phase 1/2 MGT009 study demonstrate safety profile of investigational gene therapy botaretigene sparoparvovec (AAV-RPGR) and suggest sustained vision improvement in patients with X-linked retinitis pigmentosa.

Recent reports of retinal atrophy have raised concerns on potential long-term safety.

LUNA trial will evaluate the 2x10^11 vg/eye (2E11) and a new lower 6x10^10 vg/eye (6E10) dose of Ixo-vec, with enhanced prophylactic steroid regimens in patients requiring frequent anti-VEGF injections. Interim data anticipated throughout 2023.

Nanoscope Therapeutics’ Phase 2 STARLIGHT open-label trial enrolled 6 subjects with advanced vision loss due to a clinical or genetic diagnosis of Stargardt disease.