Gene Therapy

Scientists at the Louisiana State University Health New Orleans Neuroscience Center of Excellence have developed a new, experimental human cell line from retinal pigment epithelial cells.

According to the company, 6-month safety and efficacy data are expected in Q1 2023. MCO-010 gene therapy reprograms healthy retinal cells to make them photosensitive.

The findings could lead to a new understanding of unexplained causes of heritable retinal diseases.

Pediatric ophthalmologists may need background information when treating young patients.

Eyes of mice lacking protective protein show signs similar to age-related macular degeneration.

According to the company, the primary outcome of the MGT009 study is safety, and botaretigene sparoparvovec treatment was found to be generally safe and well-tolerated.

Jason Menzo is named CEO, while Russell Kelley, PhD, MBA, will become managing director of the RD Fund. Ben Yerxa, PhD, will take the helm of Opus Genetics, the first RD Fund- and foundation-backed spin-off company.

The research is the result of a national collaboration led by investigators from the Centre for Eye Research Australia at the University of Melbourne and University of Tasmania and the Garvan Institute of Medical Research.

Genetic and clinical research reveals new type of macular dystrophy, a cause of central vision loss.

According to the study by a team of researchers from the University of California Irvine and University of Southern California, treatment with Humanin G reduced protein levels of inflammation markers that become elevated in age-related macular degeneration.

At ARVO 2022 in Denver, Colorado, Timothy Blenkinsop, MD, presented “3D Eye Organoids with Distinct Cornea.” His presentation outlined how these organoids have developed sophisticated cornea structures.

Aurion noted that its first candidate is a cell therapy for the treatment of corneal edema secondary to endothelial dysfunction.

Variable TULP1 missense mutations in inherited retinal diseases.

Greg Kunst, president and CEO of Aurion BioTech, shares a company update on endothelial cell therapy trials, clinical plans and upcoming data.

John Berdahl, MD, discusses the technique and benefit of endothelial cell therapy. An early technology, this therapy method allows for up to 100 treatments out of a single human cornea.

Impact of native lipids on rhodopsin signaling and regeneration opens door to GPCR drug discovery in native membrane environments

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.