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Therapeutic lead

RdrB allosteric modulators as chemical pharmacology probes for ADAR1 (host-directed A→I editing)

The bacterial RADAR complex (RdrA ATPase + RdrB deaminase) edits double-stranded RNA A→I, in direct functional homology with human ADAR1/ADAR2. Identify allosteric modulators of RdrB by in silico screening and test their transfer to ADAR1 — the host-directed target to potentiate viral recognition by MDA5.

Content

Initial intuition

Duncan-Lowey et al. (Cell 2023) characterized the bacterial RADAR supramolecular complex by cryo-EM—an RdrA ATPase associated with an RdrB deaminase that edits double-stranded RNA (A→I, identical to human ADAR1/ADAR2). This is one of the clearest functional parallels in the entire anti-phage arsenal: the same deamination chemistry, the same substrate (dsRNA), and structurally superimposable catalytic folds.

ADAR1 is a therapeutic target of major interest:

  • Its editing of human Alu transcripts masks endogenous RNA from MDA5, avoiding interferon-induced autoimmunity.
  • Reducing its activity in infected cells exposes viral RNAs to MDA5, potentiating the type I interferon response—this is a clean host-directed antiviral mechanism.
  • Selective ADAR1 inhibitors are actively sought (recent Phase I clinical trials: Synthekine, AIRNA, but few validated hits).

Hypothesis

The RdrB-ADAR1 structural conservation (Duncan-Lowey 2023) is sufficient for a virtual screening on bacterial RdrB to identify chemical scaffolds transferable to human ADAR1. The target is not the direct catalytic site (notoriously difficult to drug for deaminases) but allosteric pockets:

  1. RdrA-RdrB interface in the supramolecular complex—ADAR1 has an equivalent dsRNA binding domain
  2. dsRNA recognition site—competitive modulators
  3. Dimerization surface of the deaminase domain

An RdrB hit that transfers to ADAR1 with > 5× selectivity vs ADAR2 (the cerebral paralog, whose inhibition would be toxic) would be a first-class pharmacological probe for the community.

In silico plan

  1. Reference structures:

    • AlphaFold RdrB (A0ABU8PPW6, A0AAN0NNX4)—true RADAR positives according to UniProt
    • Human ADAR1 (PDB 6VFF, 5ED1, 5ED2)—crystallographic structures of the deaminase domain + dsRBD
    • Human ADAR2 (PDB 6VFF in complex with dsRNA—selectivity)
    • Complete RADAR cryo-EM (PDB 8UV4, Duncan-Lowey 2023) if available
  2. Allosteric pocket identification: FPocket / SiteMap on the non-catalytic surfaces of RdrB. Specifically target regions conserved with ADAR1 (structural alignment via Mol*).

  3. Virtual screening: drug-like ChEMBL library (~2M compounds) AutoDock Vina docking on 2-3 selected allosteric pockets. Top 1% redocking on the equivalent ADAR1 pockets.

  4. Selectivity and toxicity filters:

    • Docking score on ADAR2 (ratio > 5)
    • ADMET profile (DEREK, ADMET-AI) excluding hERG cardiotoxicity
    • No overlap with APOBEC inhibitors (other essential human deaminases)
  5. 100 ns MD on the top 30 hits to confirm the conformational stability of the binding modes.

Limitations and risks

  • ADAR allostery is poorly characterized: the few published inhibitors are either competitive (thus non-selective) or speculatively allosteric without mechanistic validation. In silico prediction of active allosteric pockets is notoriously noisy.
  • The RdrB → ADAR1 transfer assumes structural conservation of allosteric sites beyond the catalytic site. It is plausible but not guaranteed—enzymes can diverge more on regulatory surfaces than on active sites.
  • ADAR1 inhibition carries a significant autoimmunity risk (cf. ADAR1-deficient patients who develop Aicardi-Goutières type interferonopathies). An inhibitor must be used in short courses, in the context of an acute infection—not in chronic modulation. The screen should prioritize reversible hits with a short duration of action.

V1 Family RADAR (high translational priority, host-directed modality). Seminal paper Duncan-Lowey 2023 (Cell, DOI 10.1016/j.cell.2023.10.013). Strategy aligned with the family sheet: RdrB-ADAR1 structural alignment, prioritize compounds modulating host RNA editing to exacerbate viral recognition.

Note: this lead is explicitly host-directed (human ADAR1 target), not pathogen-directed. It falls into the same category as the Schlafen → SLFN11 lead, where the bacterial protein serves as a chemical template to reach a homologous human target.

Peer review

Local evaluation panel. Gesture e of the engagement report.

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