Konstantin Petrukhin
Columbia University, NY·Pharmacology
Several blinding eye diseases have few or no treatments. These include dry age-related macular degeneration (AMD), Stargardt disease and exfoliation glaucoma.
- Fields
Pharmacology
Medicine & Clinical Research
Chemistry
- Topics
age-related macular degeneration
dry AMD
Stargardt disease
- Grants
- 3 known grants, through Jun 2027
What they work on
- Advance the bispecific RBP4/TTR ligand (R)-ACPHS-62 toward an IND application for dry AMD. This compound lowers retinol delivery to the eye while stabilizing TTR, and the work will include GLP-compliant IND-enabling safety studies.
- Use medicinal chemistry to optimize the starting compounds RPE65-61 and RPE65-71 into bispecific agents for Stargardt disease. These agents would partly inhibit the visual-cycle enzyme RPE65 and trap retinaldehydes, the precursors of toxic bisretinoids.
- Identify safe yet effective doses of these bispecific compounds using visual-cycle markers, rod ERG suppression and a serum aldehyde-trapping biomarker. Then test efficacy and chronic retinal toxicity in Abca4 mutant mouse models of Stargardt disease.
- Optimize a starting maxi-K+ potassium channel agonist through SAR studies and test its in vivo efficacy as eye drops for exfoliation glaucoma. Explore a new in vivo biomarker to support preclinical and clinical development.
More about the research
Across three projects, the lab designs and develops small-molecule drugs for these conditions. Two projects target lipofuscin, a toxic waste material that builds up in the retina. Its harmful components are bisretinoids, which form as by-products of the vitamin A chemistry used for vision. The third project aims to create eye drops that help cells clear protein clumps that block fluid drainage in glaucoma.
Why it matters
Patients with dry AMD, Stargardt disease and exfoliation glaucoma face high risks of vision loss with limited or no treatment options. These projects could yield an oral drug candidate for dry AMD that is safer for people predisposed to TTR amyloidosis. They could also produce a new compound class for Stargardt disease and eye drops that may slow exfoliation glaucoma.
Known grants
From NIH and NSF research grants. Labs often have other funding we can't see.
Advancing the small molecule drug candidate with dual specificity as a therapy for dry AMD
NIH · Jul 2024 – Jun 2027
Pharmacological modulation of ion currents for treatment of exfoliation glaucoma
NIH · Jun 2024 – May 2027
Polypharmacological approach to treatment of Stargardt disease
NIH · Mar 2023 – Feb 2027
Closest labs
Shuliang Jiao
Florida International University, FL·Biomedical Engineering
Build a VIS-OCT platform combining 480 nm visible-light OCT, 840 nm near-infrared OCT and an autofluorescence detector. Use an A2E-stained reference target to obtain absolute fundus autofluorescence values.
Paul Bernstein
University of Utah, UT·Biochemistry
Test whether orally delivered synthetic VLC-PUFAs, retina-specific fats made by the enzyme ELOVL4, protect photoreceptor function, using mouse models, cell culture and model membranes.
Katelyn Swindle-Reilly
Ohio State University, OH·Biomedical Engineering
Synthesize and characterize an investigational anti-inflammatory drug and load it into ROS-responsive polydopamine nanoparticles that release more drug under oxidative stress.
The school
Columbia University: 89 labs on PI Pages, $1.4B a year on research, 625 research doctorates a year.
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