Generative design and validation of therapeutic peptides for glioblastoma based on a potential target ATP5A

Fuente: arXiv
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Hauptverfasser: Qian, Hao, You, Pu, Zeng, Lin, Zhou, Jingyuan, Huang, Dengdeng, Li, Kaicheng, Tu, Shikui, Xu, Lei
Format: Preprint
Veröffentlicht: 2025
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author Qian, Hao
You, Pu
Zeng, Lin
Zhou, Jingyuan
Huang, Dengdeng
Li, Kaicheng
Tu, Shikui
Xu, Lei
author_facet Qian, Hao
You, Pu
Zeng, Lin
Zhou, Jingyuan
Huang, Dengdeng
Li, Kaicheng
Tu, Shikui
Xu, Lei
contents Glioblastoma (GBM) remains the most aggressive tumor, urgently requiring novel therapeutic strategies. Here, we present a dry-to-wet framework combining generative modeling and experimental validation to optimize peptides targeting ATP5A, a potential peptide-binding protein for GBM. Our framework introduces the first lead-conditioned generative model, which focuses exploration on geometrically relevant regions around lead peptides and mitigates the combinatorial complexity of de novo methods. Specifically, we propose POTFlow, a \underline{P}rior and \underline{O}ptimal \underline{T}ransport-based \underline{Flow}-matching model for peptide optimization. POTFlow employs secondary structure information (e.g., helix, sheet, loop) as geometric constraints, which are further refined by optimal transport to produce shorter flow paths. With this design, our method achieves state-of-the-art performance compared with five popular approaches. When applied to GBM, our method generates peptides that selectively inhibit cell viability and significantly prolong survival in a patient-derived xenograft (PDX) model. As the first lead peptide-conditioned flow matching model, POTFlow holds strong potential as a generalizable framework for therapeutic peptide design.
format Preprint
id arxiv_https___arxiv_org_abs_2512_02030
institution arXiv
publishDate 2025
record_format arxiv
spellingShingle Generative design and validation of therapeutic peptides for glioblastoma based on a potential target ATP5A
Qian, Hao
You, Pu
Zeng, Lin
Zhou, Jingyuan
Huang, Dengdeng
Li, Kaicheng
Tu, Shikui
Xu, Lei
Biomolecules
Machine Learning
Glioblastoma (GBM) remains the most aggressive tumor, urgently requiring novel therapeutic strategies. Here, we present a dry-to-wet framework combining generative modeling and experimental validation to optimize peptides targeting ATP5A, a potential peptide-binding protein for GBM. Our framework introduces the first lead-conditioned generative model, which focuses exploration on geometrically relevant regions around lead peptides and mitigates the combinatorial complexity of de novo methods. Specifically, we propose POTFlow, a \underline{P}rior and \underline{O}ptimal \underline{T}ransport-based \underline{Flow}-matching model for peptide optimization. POTFlow employs secondary structure information (e.g., helix, sheet, loop) as geometric constraints, which are further refined by optimal transport to produce shorter flow paths. With this design, our method achieves state-of-the-art performance compared with five popular approaches. When applied to GBM, our method generates peptides that selectively inhibit cell viability and significantly prolong survival in a patient-derived xenograft (PDX) model. As the first lead peptide-conditioned flow matching model, POTFlow holds strong potential as a generalizable framework for therapeutic peptide design.
title Generative design and validation of therapeutic peptides for glioblastoma based on a potential target ATP5A
topic Biomolecules
Machine Learning
url https://arxiv.org/abs/2512.02030