Beacon Therapeutics reported positive results from the Phase 3 pivotal trial of laru-zova, its potential gene therapy treatment for X-linked retinitis pigmentosa. A significant proportion of patients improved dim-light vision by 15 or more letters after treatment with either high or low doses of laru-zova compared to the group that was not treated, according to preliminary results from the VISTA clinical trial. Beacon plans to begin seeking FDA approval later this year. If approved, this would be the first treatment available for XLRP, one of the more severe forms of retinitis pigmentosa that predominantly affects males.

The U.S. Food and Drug Administration accepted Dallas-based Nanoscope Therapeutics' biologics license application for MOGENRY, an emerging treatment for adults with retinitis pigmentosa and severe vision loss. The investigational treatment does not require a genetic diagnosis or specific gene mutation, giving MOGENRY the potential to become the first approved gene-agnostic treatment to improve vision in people with severe vision loss from RP.

Two non-profit professionals talk about plugging into community and finding low-vision resources through the Foundation

Sumitomo Pharma America treated its first patient in a Phase 1/2a clinical trial for an emerging retinitis pigmentosa (RP) treatment called DSP-3077. The clinical trial plans to treat 12 adults with one of two different doses to evaluate safety and effectiveness of the treatment among patients who have RP.

Tarsus Pharmaceuticals announced plans to acquire Alkeus Pharmaceuticals, including its emerging oral treatment for Stargardt disease, gildeuretinol (ALK-001), in a deal valued up to $800 million. Stargardt disease is the most common form of inherited macular degeneration and is often diagnosed in children or teens. There are no current treatments.

In Europe, Science Corporation has commercially launched PRIMA, a retinal implant designed to restore central vision in people with geographic atrophy (GA). The treatment has been shown in clinical trials to restore functional central vision in people with GA who until now had no way to get lost central vision back. While PRIMA is available in Europe, it is still going through FDA approvals in the U.S. Geographic atrophy is an advanced form of age-related macular degeneration (AMD) that leads to progressive vision loss. AMD is the most common cause of blindness in individuals over the age of 55 in developed countries.

Belite Bio’s tinlarebant could become the first-ever approved treatment for Stargardt disease

Dr. Alicia Kemble explains how the Foundation is using venture philanthropy to drive treatments for inherited retinal diseases even closer to the finish line.

Sepul Bio recently announced that it completed enrollment of its Phase 2b safety and efficacy clinical trial for ultevursen – an investigational RNA therapy to treat patients who have retinitis pigmentosa (RP) associated with mutations in the USH2A gene.

FDA approves Lumvoa, a new treatment for thyroid eye disease by Foundation Fighting Blindness