Project

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Summer 2026 challenge: phase 1 contestant

Rational Design of Isoform-Selective CA II Inhibitors through Chemical Space Exploration

Ernestas Urniežius, Institute of Biotechnology, Vilnius University, Vilnius, Lithuania

Carbonic anhydrase II (CA II) plays a key role in neuronal pH regulation and is a potential therapeutic target for intracranial pressure-related diseases. However, clinically used CA drugs such as acetazolamide and topiramate lack isoform selectivity, leading to off-target inhibition and potential side effects. Although selective benzenesulfonamides have been developed for some isoforms, e.g. CA IX (oncology target), no chemotype comprehensively validated for selective targeting of brain-relevant CA II currently exists. Analysis of the PLBD database suggests that structural variation beyond the benzenesulfonamide scaffold may be required to achieve CA II selectivity, motivating a scaffold-hopping strategy. We will explore chemical space for novel non-benzenoid sulfonamide scaffolds and benzenesulfonamide analogs and filter using integrated LBDD and SBDD approaches. Selected candidates will be experimentally tested to support the development of novel isoform-selective CA II inhibitors.
Ernestas intends to achieve the following milestones:
  1. Curate CA isozyme structures and reference ligands. Establish LBDD/SBDD workflows and objective criteria for comparative ligand selection.
  2. Screen Enamine REAL and other spaces with infiniSee and SpaceMACS for non-benzenoid sulfonamide scaffolds and benzenesulfonamide analogs.
  3. Analyze, rank, and select accessible sulfonamide candidates using CADD workflows; define initial subsets for rigorous follow-up testing.