Drugs Promoting Retinal Rod Photoreceptor Survival

Unmet Need
Retinitis pigmentosa (RP) is a genetic disorder that can result from more than 60 mutations that cause severe rod photoreceptor degeneration and death. It is estimated to affect 1 in 4000 people worldwide, with approximately 100,000 cases in the United States. Symptoms begin in childhood with a loss of night vision, and eventually RP leads to significant vision loss and blindness in adulthood. Presently, RP has no cure or effective treatment. Life-style modifications and disability services can improve quality of life and retinal implants are available for those with profound disease. Consequently, there is a great need for novel neuroprotective therapeutics to treat RP and preserve rod photoreceptor cells throughout a patient’s life. 
 
Technology Overview
Johns Hopkins researchers have demonstrated novel repurposing of certain human approved drugs as neuroprotective compounds for the treatment of RP. The compounds were identified through a large-scale phenotypic screen in a zebrafish model of RP. Top hits from the screen in zebra fish that preserved rod photoreceptor cell survival were validated using mouse photoreceptor cell culture and retinal explant cultures from the retinal degeneration 1 (rd1) RP mouse model. Combinatorial assays also identified 7 additive compounds that target complimentary neuroprotective signaling pathways that offer additional treatment strategies for RP.
 
Stage of Development
The inventors have identified novel neuroprotective functions for four human approved therapeutics that can potentially be used to treat RP. Further research will be conducted to measure efficacy in preserving rod photoreceptor cell survival in humans and to optimize safe and effective dosing and extended release formulas for the human retina.

Patent Status: Provisional application pending

Inventors: Jeff Mumm
 

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