A bottom-up approach to find lead compounds in expansive chemical spaces

Abstract Drug discovery starts with the identification of a “hit” compound that, following a long and expensive optimization process, evolves into a drug candidate. Bigger screening collections increase the odds of finding more and better hits. For this reason, large pharmaceutical companies have in...

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Main Authors: Álvaro Serrano-Morrás, Andrea Bertran-Mostazo, Marina Miñarro-Lleonar, Arnau Comajuncosa-Creus, Adrià Cabello, Carme Labranya, Carmen Escudero, Tian V. Tian, Inna Khutorianska, Dmytro S. Radchenko, Yurii S. Moroz, Lucas Defelipe, David Ruiz-Carrillo, Maria Garcia-Alai, Robert Schmidt, Matthias Rarey, Patrick Aloy, Carles Galdeano, Jordi Juárez-Jiménez, Xavier Barril
Format: Article
Language:English
Published: Nature Portfolio 2025-08-01
Series:Communications Chemistry
Online Access:https://doi.org/10.1038/s42004-025-01610-2
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author Álvaro Serrano-Morrás
Andrea Bertran-Mostazo
Marina Miñarro-Lleonar
Arnau Comajuncosa-Creus
Adrià Cabello
Carme Labranya
Carmen Escudero
Tian V. Tian
Inna Khutorianska
Dmytro S. Radchenko
Yurii S. Moroz
Lucas Defelipe
David Ruiz-Carrillo
Maria Garcia-Alai
Robert Schmidt
Matthias Rarey
Patrick Aloy
Carles Galdeano
Jordi Juárez-Jiménez
Xavier Barril
author_facet Álvaro Serrano-Morrás
Andrea Bertran-Mostazo
Marina Miñarro-Lleonar
Arnau Comajuncosa-Creus
Adrià Cabello
Carme Labranya
Carmen Escudero
Tian V. Tian
Inna Khutorianska
Dmytro S. Radchenko
Yurii S. Moroz
Lucas Defelipe
David Ruiz-Carrillo
Maria Garcia-Alai
Robert Schmidt
Matthias Rarey
Patrick Aloy
Carles Galdeano
Jordi Juárez-Jiménez
Xavier Barril
author_sort Álvaro Serrano-Morrás
collection DOAJ
description Abstract Drug discovery starts with the identification of a “hit” compound that, following a long and expensive optimization process, evolves into a drug candidate. Bigger screening collections increase the odds of finding more and better hits. For this reason, large pharmaceutical companies have invested heavily in high-throughput screening (HTS) collections that can contain several million compounds. However, this figure pales in comparison with the emergent on-demand chemical collections, which have recently reached the trillion scale. These chemical collections are potentially transformative for drug discovery, as they could deliver many diverse and high-quality hits, even reaching lead-like starting points. But first, it will be necessary to develop computational tools capable of efficiently navigating such massive virtual collections. To address this challenge, we have conceived an innovative strategy that explores the chemical universe from the bottom up, performing a systematic search on the fragment space (exploration phase), to then mine the most promising areas of on-demand collections (exploitation phase). Using a hierarchy of increasingly sophisticated computational methods to remove false positives, we maximize the success probability and minimize the overall computational cost. A basic implementation of the concept has enabled us to validate the strategy prospectively, allowing the identification of new BRD4 (BD1) binders with potencies comparable to stablished drug candidates.
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spelling doaj-art-1f4fa385d19746d1925360b072e86c612025-08-20T03:04:26ZengNature PortfolioCommunications Chemistry2399-36692025-08-018111110.1038/s42004-025-01610-2A bottom-up approach to find lead compounds in expansive chemical spacesÁlvaro Serrano-Morrás0Andrea Bertran-Mostazo1Marina Miñarro-Lleonar2Arnau Comajuncosa-Creus3Adrià Cabello4Carme Labranya5Carmen Escudero6Tian V. Tian7Inna Khutorianska8Dmytro S. Radchenko9Yurii S. Moroz10Lucas Defelipe11David Ruiz-Carrillo12Maria Garcia-Alai13Robert Schmidt14Matthias Rarey15Patrick Aloy16Carles Galdeano17Jordi Juárez-Jiménez18Xavier Barril19Unitat de Fisicoquímica, Departament de Farmàcia i Tecnologia Farmacéutica, i Fisicoquímica. Facultat de Farmàcia i Ciències de l’Alimentació, Universitat de Barcelona (UB)Unitat de Fisicoquímica, Departament de Farmàcia i Tecnologia Farmacéutica, i Fisicoquímica. Facultat de Farmàcia i Ciències de l’Alimentació, Universitat de Barcelona (UB)Unitat de Fisicoquímica, Departament de Farmàcia i Tecnologia Farmacéutica, i Fisicoquímica. Facultat de Farmàcia i Ciències de l’Alimentació, Universitat de Barcelona (UB)Institut de Recerca Biomèdica (IRB Barcelona) and Barcelona Institute of Science and Technology (BIST). c/ Baldiri i ReixacUnitat de Fisicoquímica, Departament de Farmàcia i Tecnologia Farmacéutica, i Fisicoquímica. Facultat de Farmàcia i Ciències de l’Alimentació, Universitat de Barcelona (UB)Unitat de Fisicoquímica, Departament de Farmàcia i Tecnologia Farmacéutica, i Fisicoquímica. Facultat de Farmàcia i Ciències de l’Alimentació, Universitat de Barcelona (UB)Upper Gastrointestinal and Endocrine Tumor Group, Vall d’Hebron Institute of Oncology (VHIO), Vall d’Hebron Barcelona Hospital CampusUpper Gastrointestinal and Endocrine Tumor Group, Vall d’Hebron Institute of Oncology (VHIO), Vall d’Hebron Barcelona Hospital CampusEnamine Ltd.Enamine Ltd.Enamine Ltd.European Molecular Biology LaboratoryEuropean Molecular Biology LaboratoryEuropean Molecular Biology LaboratoryCenter for Bioinformatics, University of Hamburg ZBHCenter for Bioinformatics, University of Hamburg ZBHInstitut de Recerca Biomèdica (IRB Barcelona) and Barcelona Institute of Science and Technology (BIST). c/ Baldiri i ReixacUnitat de Fisicoquímica, Departament de Farmàcia i Tecnologia Farmacéutica, i Fisicoquímica. Facultat de Farmàcia i Ciències de l’Alimentació, Universitat de Barcelona (UB)Unitat de Fisicoquímica, Departament de Farmàcia i Tecnologia Farmacéutica, i Fisicoquímica. Facultat de Farmàcia i Ciències de l’Alimentació, Universitat de Barcelona (UB)Unitat de Fisicoquímica, Departament de Farmàcia i Tecnologia Farmacéutica, i Fisicoquímica. Facultat de Farmàcia i Ciències de l’Alimentació, Universitat de Barcelona (UB)Abstract Drug discovery starts with the identification of a “hit” compound that, following a long and expensive optimization process, evolves into a drug candidate. Bigger screening collections increase the odds of finding more and better hits. For this reason, large pharmaceutical companies have invested heavily in high-throughput screening (HTS) collections that can contain several million compounds. However, this figure pales in comparison with the emergent on-demand chemical collections, which have recently reached the trillion scale. These chemical collections are potentially transformative for drug discovery, as they could deliver many diverse and high-quality hits, even reaching lead-like starting points. But first, it will be necessary to develop computational tools capable of efficiently navigating such massive virtual collections. To address this challenge, we have conceived an innovative strategy that explores the chemical universe from the bottom up, performing a systematic search on the fragment space (exploration phase), to then mine the most promising areas of on-demand collections (exploitation phase). Using a hierarchy of increasingly sophisticated computational methods to remove false positives, we maximize the success probability and minimize the overall computational cost. A basic implementation of the concept has enabled us to validate the strategy prospectively, allowing the identification of new BRD4 (BD1) binders with potencies comparable to stablished drug candidates.https://doi.org/10.1038/s42004-025-01610-2
spellingShingle Álvaro Serrano-Morrás
Andrea Bertran-Mostazo
Marina Miñarro-Lleonar
Arnau Comajuncosa-Creus
Adrià Cabello
Carme Labranya
Carmen Escudero
Tian V. Tian
Inna Khutorianska
Dmytro S. Radchenko
Yurii S. Moroz
Lucas Defelipe
David Ruiz-Carrillo
Maria Garcia-Alai
Robert Schmidt
Matthias Rarey
Patrick Aloy
Carles Galdeano
Jordi Juárez-Jiménez
Xavier Barril
A bottom-up approach to find lead compounds in expansive chemical spaces
Communications Chemistry
title A bottom-up approach to find lead compounds in expansive chemical spaces
title_full A bottom-up approach to find lead compounds in expansive chemical spaces
title_fullStr A bottom-up approach to find lead compounds in expansive chemical spaces
title_full_unstemmed A bottom-up approach to find lead compounds in expansive chemical spaces
title_short A bottom-up approach to find lead compounds in expansive chemical spaces
title_sort bottom up approach to find lead compounds in expansive chemical spaces
url https://doi.org/10.1038/s42004-025-01610-2
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