Gene Therapy

The trial marks the first-ever in vivo delivery of an experimental CRISPR gene editing medicine to a pediatric patient, with the company on track to complete dosing of the pediatric mid-dose cohort in the first half of 2022.

According to researchers at the University of California, Irvine, base editing may provide long-lasting retinal protection and prevent vision deterioration in patients with inherited retinal degeneration, specifically in Leber congenital amaurosis patients.

According to the company, the modifier gene therapy candidate is for the treatment of retinitis pigmentosa resulting from mutations in the nuclear receptor subfamily 2 group E member 3 and Rhodopsin genes.

The company’s announcement marks first clinical trial in humans of Ocugen’s modifier gene therapy platform.

A team of investigators at the Okinawa Institute of Science and Technology Graduate University in Japan have identified a gene necessary for the survival of retinal ganglion cells – a class of neurons located in the retina that are critical for vision.

Investigators find that hybrid cells could be a potential therapeutic strategy to treat retinal damage and visual impairment.

A team of investigators has found that nomacopan, a recombinant biologic derived from blood-feeding ticks, may be an option for the treatment of allergic eye disease due to its ability to down-regulate the LTB4/C5 pathways in experimental allergy conjunctivitis.

Investigators observe survival rate with orbital retinoblastoma improves substantially due to a combination approach that includes intensive sequential treatment comprised of chemotherapy, enucleation, and external beam radiation therapy.

According to the company, pegcetacoplan demonstrated continuous and clinically meaningful effects at month 18 in the studies, which also found that treatment effects in DERBY were comparable to OAKS during months 6 to 18. The combined 18-month data show the potential for improving treatment effects over.

A gift from Bronwyn Bateman, MD, will establish the UCLA Bronwyn Bateman Center for Ocular Genetics at the Stein Eye Institute.

A team of investigators has found that the eye disease is more prevalent than first thought.

Trial recruitment is enabled by the Foundation Fighting Blindness' patient registry, My Retina Tracker Registry, which includes gene

The clinical trial will evaluate the safety and tolerability of ADX-2191 in patients diagnosed with RP due to mutations of the rhodopsin gene, including the P23H gene mutation.

Christina Y. Weng, MD, MBA, an associate professor of ophthalmology and surgical retina fellowship program director at Baylor College of Medicine in Houston, recently shared some standout therapies for macular degeneration.

The companies note that the partnership will propel Ray Therapeutics’ lead optogenetics gene therapy to Phase 1-2 clinical trials.

Investigators at Trinity College Dublin have highlighted the SARM1 gene, a key driver in the damage that ultimately leads to impaired vision.

Carel B. Hoyng, MD, noted that investigators have developed an RNA therapy to stop the progression of the disease, which ultimately leads to legal blindness.

The team hopes the use of patient-derived stem cells will enable high-throughput drug screening for potential therapeutics for patients.

Cleveland Eye Bank Foundation (CEBF) will feature nine researchers from three eye institutions on February 15, from 3 to 5 pm Eastern, for its second annual virtual vision research symposium.

Researchers collected data on COVID-19 symptoms via online surveys from in excess of 1 million 23andMe participants who self-reported their COVID-19 symptoms.

A team of investigators at the RIKEN Center for Biosystems Dynamics Research in Japan have used a genetic modification to improve human-derived retina transplants grown in the lab.

A study finds acceptable outcomes, vision, and microperimetry improvements at higher doses.

Nanoscope Therapeutics has received IND clearance from the FDA to begin a Phase 2 trial of its Multi-Characteristic Opsin ambient-light activatable optogenetic monotherapy to restore vision in Stargardt patients.

AGTC-501 is a recombinant AAV vector-based gene therapy developed for the treatment of X-linked retinitis pigmentosa.

According to the company, 4D-150 is a dual-transgene, intravitreal gene therapy designed to inhibit four distinct angiogenic factors to prevent angiogenesis and reduce vascular permeability for the treatment of wet AMD.

Team at University of California, Irvine provides an understanding of the mechanisms underlying the pathologies of certain gene mutations.

The novel gene therapy product candidate is being developed as a potential single intravitreal administration for blue cone monochromacy by delivering a functional copy of the OPN1LW gene.

The grant will provide Steven DeVries, MD, PhD, the opportunity to travel to Kyoto, Japan and learn from scientists at Ritsumeikan University about the process of growing 3D retinal organoids from stem cells, a technique first developed in Japan.

Researchers have found that study participants with healthy eyes and no history of AMD had thinner retinas if they carried the genes that put them at risk.

In light of another unprecedented year filled with technological advancements and pivots as a result of the pandemic, Joshua Mali, MD, offers his top 5 predictions in ophthalmology for 2022.