Stargardt's Disease Therapy

A novel approach

Our lead product, ready for partnering, is a therapy for patients with Stargardt’s Disease that is designed to clear a toxic lipid, called A2E, that leads to cell death and retinal degeneration. No other product in development removes accumulated A2E. Researchers from Weill Cornell Medicine first reported removing accumulated A2E deposits from the eye in a mouse model of Stargardt’s.

Stargardt's is a rare genetic disorder that affects central vision. The disease often manifests in childhood and leads to legal blindness within the second or third decade of life. There is no approved treatment for Stargardt’s Disease. Our unique approach is based on the use of an FDA-approved, GRAS molecule with extensive safety data. A few companies are developing other therapies that could potentially slow disease progression or treat the genetic cause. Given its unique mechanism of action, reVision’s therapeutic could be useful as an adjuvant with these other therapies if they are approved, in addition to use as a first line therapy.

Stargardt's Disease

Stargardt’s disease is caused by mutations of the ABCA4 gene that lead to abnormal accumulation of fluorescent metabolic lipid byproducts of the visual cycle, A2E or lipofuscin, which exhibit toxicity when they accumulate. A2E accumulation in human retinal pigmented epithelial (RPE) cells has a direct correlation with RPE cell death, which leads to macular degeneration.  

reVision is developing CVZ002 for intravitreal injection, following demonstrated efficacy in pre-clinical studies.  

Therapeutic Opportunity

Stargardt's Disease qualifies as an Orphan indication. The number of people affected in the US is currently about 32,000, with 355 new patients annually. Estimates in Europe are around 50,000 patients, with about 500 new patients annually. The incidence rate is 1 in 10,000.

Partnering Opportunity 

reVision is currently seeking partners to help advance our lead compound, CVZ002, into the clinic. Given Stargardt’s low incidence and recognized unmet medical need, the product is expected to receive Orphan drug designation and qualify for Pediatric exclusivity. 

reVision's Stargardt's Therapy in a Nutshell

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.

reVision has secured an exclusive supply agreement and rights to reference the drug master file for the active molecule in our product.