In Silico Discovery of Pharmacological Chaperones of P23H Rhodopsin to Treat Retinitis Pigmentosa

Overview

Retinitis pigmentosa (RP), affecting about 1 in 4,000 people, is the most common inherited retinal disease. A common cause of RP is the P23H mutation in rhodopsin, which causes the protein to misfold and be destroyed. This leads to photoreceptor death and progressive vision loss. Unfortunately there are no approved treatments. I developed a computational drug discovery pipeline using AutoDock Vina and GROMACS to identify molecules that stabilize P23H rhodopsin. The system screens for strong binding at the retinal pocket and tests stability in a realistic membrane using molecular dynamics simulations. After screening 17,375 compounds, hnRP94450 emerged as the top candidate molecule. hnRP94450 restored near-normal stability causing treated proteins to behave like healthy rhodopsin over 500 ns. hnRP94450 could be patented as a novel therapy and become a first-in-class treatment to slow or halt RP progression.

Awards (2)

• Silver Medal
• Selected for CWSF 2026

Competition history

• CWSF 2026 Disease & Illness

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