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models / small-molecule / aiprotac
Small moleculeProtein
Tsinghua UniversityAgency for Science, Technology and Research (A*STAR)Gluetacs TherapeuticsCentral South UniversityChina University of Mining and TechnologyJiangnan UniversitySichuan UniversityReleased March 2025

AiPROTAC

PROTAC degradation prediction from molecular graphs of the target, linker, and E3 ligase, combining cross-attention with contrastive learning.

The short version

  • —Filters out weak degraders before chemists commit to synthesis
  • —Reads the target protein, the chimera, and the E3 ligase as one graph assembly
  • —Contrastive auxiliary tasks pull signal from unlabeled PROTAC-DB entries
  • —Guided design of the AR degrader GT19, confirmed active in prostate cancer cells
77Openness0Citations
11GitHub stars
Apache-2.0License

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Proteolysis-targeting chimeras degrade a protein rather than inhibit it, which lets them reach targets with no druggable pocket. Whether a given chimera actually works, though, depends on a ternary complex forming between the target protein, the bifunctional ligand, and an E3 ligase — a property that is hard to reason about from structure and expensive to establish experimentally. More than two decades after the first proof-of-concept PROTAC, no degrader has reached market approval, and degradation capacity remains the property that most often decides whether a series is worth pursuing.

AiPROTAC predicts that degradation capacity directly. Led by Li Zhang and Xing Chen with collaborators at Tsinghua University, A*STAR, Gluetacs Therapeutics, and several Chinese universities, and posted as a preprint in March 2025, it represents all three components as molecular graphs and passes them through dedicated encoders before a two-stage cross-attention decoder produces a binary degradation call. The design is deliberately shaped like the wet-lab logic it replaces: the first attention stage models the proximity the chimera induces between target and ligase, the second reads the resulting ternary representation from both partners' perspectives.

Labeled degradation data is scarce, and the model's second idea addresses that shortage. Two auxiliary contrastive branches, active only during training, learn from graph augmentations of unlabeled PROTAC-DB molecules and proteins, so the supervised classifier benefits from data that carries no degradation label at all. Where SynPROTAC generates candidate chimeras, AiPROTAC scores them.

#Key Features

  • Tri-graph input: The target protein, the E3 ligase, and the chimera each enter as a molecular graph, so the model sees the whole assembly rather than a concatenation of descriptors.
  • Edge-aware encoders: Proteins use an edge-weighted graph convolutional network and chimeras a Convolution with Edge-Node Switching encoder, both in residual form, letting physicochemical information on graph edges contribute to the representation.
  • Two-stage cross-attention: Attention first derives target-ligand and ligase-ligand representations, then exchanges them to produce target-viewed and ligase-viewed ternary features that are concatenated for classification.
  • Contrastive use of unlabeled data: Auxiliary protein and ligand branches share the encoder with the classifier and train on augmented subgraphs, extending the effective training set beyond the labeled samples.
  • Prospective design validation: The trained model guided a campaign that produced GT19, an androgen receptor degrader confirmed by Western blot in a prostate cancer cell line.

#Technical Details

Evaluation used PROTAC-DB 2.0 plus PROTAC-ZL, a manually curated set assembled by the authors from molecules not present in PROTAC-DB 2.0. Labeled PROTAC-DB samples were split 8:2 into training and test sets across five independent runs with different random seeds. Averaged over those runs, AiPROTAC reached accuracy 0.8502, precision 0.8298, recall 0.8467, F1 0.7935, AUROC 0.9192, and AUPR 0.8278, with standard deviations at or below 0.0505 on every metric. On the fully external PROTAC-ZL set it held accuracy 0.7928, AUROC 0.8156, and AUPR 0.7504; the mean of those three metrics, 0.7862, compares with 0.5450 for DeepPROTACs. Ten baselines were implemented for comparison, including random forest and SVM models over MACCS keys and Morgan fingerprints, DeepPROTACs, and four ablated AiPROTAC variants that swap the encoder for a GCN or GAT or strip out the attention and contrastive machinery.

In the prospective campaign, 18 cereblon-recruiting androgen receptor degraders were designed, synthesized, and assayed in LNCaP cells; AiPROTAC's predictions matched 8 of 12 in the first series and 5 of 6 in the second. A nineteenth compound, GT19, was designed by moving the linker attachment point on the cereblon ligand's benzene ring, and was predicted and confirmed to degrade the androgen receptor — at 100 nM within 24 hours, outperforming bavdegalutamide (ARV-110), with a cereblon binding IC50 of 0.15 μM against greater than 10 μM for a methylated negative control. Across all 19, 14 labels were called correctly, a 74% hit rate.

#Applications

The intended use is triage. A medicinal chemistry group designing a degrader series can score every candidate before committing to synthesis, discarding the ones predicted not to degrade and carrying forward only the plausible ones — the AR campaign is a worked example of exactly that loop. Because the input is three molecular graphs, the model applies to any target and any E3 ligase for which structures or sequences are available, not only the cereblon and VHL systems that dominate published data.

#Impact

AiPROTAC is a preprint and has not been peer reviewed. The code, including the training scripts, model definitions, preprocessed PROTAC-DB and PROTAC-ZL data, and predictor entry points, is public under Apache-2.0; the predictor loads a saved state dictionary, but no trained checkpoint ships with the repository, so users must retrain before running inference. The prospective AR result is the entry's strongest evidence — a degrader designed with model guidance, synthesized, and shown by Western blot to outperform a clinical-stage comparator — while the benchmark margins rest on datasets of a few hundred labeled chimeras, which is the standing constraint on every model in this area.

At a glance

Released
March 2025
Category
Small molecule
License
Apache-2.0
Organizations
Tsinghua University / Agency for Science, Technology and Research (A*STAR) / Gluetacs Therapeutics / Central South University / China University of Mining and Technology / Jiangnan University / Sichuan University

Related models

  • SynPROTAC

    Sun Yat-sen University

  • SynGlue

    Indraprastha Institute of Information Technology Delhi / Aurigene Oncology

  • Ubicon

    Institute of Science Tokyo

  • AiPP

    University of Maryland School of Pharmacy / Purdue University

  • ProtLigand

    Technion – Israel Institute of Technology / Microsoft

Links

GitHub RepositorybioRxiv Preprint

Tags

cancercontrastive_learningdrug_discoverygraph_neural_networkvirtual_screening

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