REV-0100
A novel approach to treating a high-impact rare disease with no currently available treatments…
REV-0100 is in preclinical development as a treatment for Stargardt disease. Our candidate is a repurposed, GRAS (generally recognized as safe) compound delivered intravitreally that could work as a first-line therapy or in combination with other products currently in development.
REV-0100 was awarded both Orphan Drug Designation (ODD) and Rare Pediatric Disease (RPD) designation by the US Food and Drug Administration. REV-0100 works by reducing levels of a toxic lipid called lipid bisretinoid, which accumulates within the retina in material called lipofuscin. This accumulation happens much more quickly in people with Stargardt disease, leading to retinal cell death and retinal degeneration, which eventually results in permanent central blindness. By reducing levels of the abnormal accumulation of toxic lipid bisretinoid, REV-0100 is being developed as a treatment to stave off or prevent central blindness in people diagnosed with the ABCA4 gene mutation that leads to Stargardt disease.
REV-0100 is unique…
Other products in development to treat Stargardt disease have different mechanisms of action, some of which may retard the visual cycle, which may negatively impact vision. REV-0100 works differently, clearing the toxic lipid without impact on the visual cycle.
REV-0100 is versatile…
Because REV-0100 works by clearing toxic lipids, it is being considered for multiple uses. It may be effective as a first-line monotherapy, adjuvant treatment in combination with other oral therapies, or as a temporary treatment prior to or during such as gene therapy.
Therapeutic Opportunity
Stargardt disease qualifies as an Orphan indication in the United States, Europe, and Japan, and Stargardt’s is expected to meet the criteria for orphan status in other countries that recognize such a designation. The number of people affected in the United States is currently about 32,000, with 355 new patients diagnosed annually. Estimates in Europe are around 50,000 diagnosed patients, with about 500 new diagnoses annually. The incidence rate is 1 in 10,000 worldwide. Until an effective gene therapy is developed and made accessible, chronic treatments, such as REV-0100, will be the only option for disease management. Even with the approval of a gene therapy, accessibility and success rate will still be barriers to treatment for a number of patients. REV-0100 may also be effective as an adjunctive therapy until full efficacy of gene therapy is achieved.
Partnering Opportunity
reVision is currently seeking partners to advance our lead compound, REV-0100. reVision has secured an exclusive supply agreement and rights to reference the drug master file for the active molecule in our product.
Please submit a Contact form to learn more about our global and regional partnering opportunities.