Gene Therapy

Stoke Therapeutics announced enrollment of the first patient in a prospective natural history study of people ages 8 to 60 who are living with autosomal dominant optic atrophy.

Low-grade inflammation seems to be associated with baseline disease in inherited retinal dystrophies (IRDs), according to Christine N. Kay, MD, a surgeon at Vitreoretinal Associates, in Gainesville, Florida.

APOE4 gene associated with Alzheimer’s disease risk was found to protect mice from glaucoma. Research team also prevented retinal ganglion cell death by blocking the APOE signaling pathway, pointing to a potential treatment strategy for glaucoma.

X-linked retinoschisis is a congenital disorder that results in visual impairment in boys at an early age. No treatment is currently available, and surgical repair fails in many cases.

The study provided evidence of safety, visual acuity improvement and structural stability in a difficult-to-treat patient population.

According to a news release, the U.S. Food and Drug Administration has approved Coherus’ ranibizumab-eqrn (Cimerli) as an interchangeable biosimilar for all five indications of Lucentis.

A multi-institutional effort led by researchers at the University of Pennsylvania is taking steps to develop an effective technique to regenerate photoreceptors cells and restore sight in patients with vision disorders.

The discovery of molecular signatures of age-related macular degeneration will help with better diagnosis and treatment of this progressive eye disease.

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.