U.S. National Committee for CODATA

Webinar

Lead Discovery in Terra-Scale Chemical Spaces with Modular Screening and Deep Learning Approaches

May 15, 2025

10:00 AM - 1:00 PM (EDT)

Past

Materials

Dr. Katritch Presentation Slides

About

As part of Advancing Drug Discovery: A Webinar Series of the National Academies of Sciences, Engineering and Medicine, a session on Lead Discovery in Terra-Scale Chemical Spaces with Modular Screening and Deep Learning Approacheswill be held on Thursday, May 15th, 2025 at noon (EDT). The 60-minute session will consist of a presentation from Dr. Vsevolod “Seva” Katritch, Professor of Quantitative & Computational Biology, Chemistry and Pharmacology, University of Southern California.

Abstract:

Recent technological advances, including (i) high accessibility of structural information for therapeutic targets, (ii) giga-scale on-demand and generative chemical spaces, and (iii) new computational tools, both physics-based and machine learning, have great potential to streamline and democratize early drug discovery. We have recently developed a new computationally driven platform, combining fast V-SYNTHES screening of billions of diverse drug-like compounds (Sadybekov et al, Nature 2022) and SAR/optimization tools, leveraging on-demand chemical spaces like Enamine REAL. The upgraded V-SYNTHES-DL platform with deep learning-based accelerator and RTCNN scoring function is now scaled up to cover trillions of compounds of the next-generation xREAL Space. The V-SYNTHES approach was initially validated in several target classes, including GPCRs and kinases, enabling discovery of new drug-like chemotypes with nanomolar affinities and potencies.

We now expand the application of this computationally driven platform to a broad range of highly challenging targets, in close collaboration with synthetic/medicinal chemists, structural/systems biologists, and pharmacologists as part of the CNT3D center at USC. In just 2 years, CNT3D advanced more than 20 pilot projects for challenging targets selected by disease experts, targeting pain and addiction, immune and neurodegenerative disorders, infectious diseases, and cancer. The results include the discovery of high-quality hits and lead series in most of these projects, with two optimized compounds advancing in preclinical development. These examples suggest that structure-based computationally driven platforms combining physics-based and deep-learning methods can provide fast, cost-effective quality entry points for drug development.

Short Speaker’s Bio:

Dr. Vsevolod “Seva” Katritch is a computational biologist and computational chemist interested in deciphering molecular function and structural pharmacology of membrane proteins, primarily the superfamily of G protein-coupled receptors (GPCRs). He also develops new physics-based and AI-based technologies facilitating discovery of new ligands and potential drugs for GPCRs and other clinically relevant targets, exploring giga- and terra-scale chemical spaces. His work has led to 9 patents and more than 150 papers, including the V-SYNTHES approach to giga-scale screening and review on computer-driven drug discovery, both published in Nature. He is named among Web of Science's Highly Cited Researchers in two categories: pharmacology/toxicology and biology/biochemistry.

Dr. Katritch graduated and received a Ph.D. in biophysics and molecular biology from Moscow Institute of Physics and Technology, followed by postdoctoral training at Rutgers University and The Scripps Research Institute. Before joining USC in 2015, he served as a director of computational chemistry at SIGA Technologies and held research faculty positions at the University of California, San Diego, and the Scripps Research Institute.

Organizing Project

Advancing Drug Discovery: A Webinar Series of the National Academies of Sciences, Engineering, and Medicine

Contact

Ana Ferreras

aferrerasfiel@nas.edu

Disclaimer

It is essential to the National Academies mission of providing evidence-based advice that participants in any of our meetings or events avoid political or partisan statements or commentary and maintain a culture of mutual respect. The statements and presentations during our meetings or events are solely those of the individual participants and do not necessarily represent the views of other participants or the National Academy of Sciences, which is a non-partisan, tax exempt organization that includes under its Charter the National Academy of Engineering and National Academy of Medicine, and that operates the National Research Council.

Webinar

Quantum Computing-Enhanced Machine Learning Algorithm Unveils Novel Inhibitors for KRAS

April 28, 2025

12:00 PM - 1:00 PM (EDT)

Past

Materials

Dr. Gorgulla Presentation Slides

About

As part of Advancing Drug Discovery: A Webinar Series of the National Academies of Sciences, Engineering and Medicine, a session on Quantum Computing-Enhanced Machine Learning Algorithm Unveils Novel Inhibitors for KRAS will be held on Monday, April 25th, 2025 at noon (EDT). The 60-minute session will consist of a presentation from Dr. Christoph Gorgulla, assistant member (faculty) at St. Jude Children's Research Hospital and associate at Harvard University, with time for a short question and answer session at the end.

Abstract:

The discovery of promising small molecules remains one of the most resource-intensive stages in drug development, particularly for challenging targets such as KRAS, a key driver in many cancers. While classical computational approaches have improved in quality and efficiency, they still face limitations in exploring the vast and complex chemical space to discover the best possible molecules. In this talk, we will explore a quantum-classical generative modeling framework designed to enhance molecular design by leveraging quantum computing. By integrating quantum algorithms with established classical methods, the approach enables the generation of chemically meaningful candidates with therapeutic potential. The application to KRAS inhibitor design demonstrates how quantum resources can complement and extend current capabilities in drug discovery.

Short Speaker’s Bio:

Dr. Christoph Gorgulla's research mission is to contribute to the transformation of drug discovery and molecular biology by integrating high-performance computing, artificial intelligence, quantum chemistry, and quantum computing.
He holds a B.Sc. in Molecular Biology with a focus on bioinformatics, a B.Sc. in Mathematics, and an M.Sc. in Mathematics with focus on quantum physics. He earned his Ph.D. from Freie Universität Berlin, the Berlin Mathematical School and the Max Planck Institute for Molecular Genetics, focusing on computational drug design. This interdisciplinary training enables him to bridge theoretical method development, software engineering, and real-world biomedical applications.
Following his postdoctoral research at Harvard University, he joined St. Jude Children’s Research Hospital in 2023 to establish and lead a team in computational drug discovery. In 2024, he became a faculty member in the Center of Excellence for Data-Driven Discovery within the Department of Structural Biology of St. Jude, where his research group continues to develop next generation computational methods based on AI and quantum computing, and apply them to challenging problems in ligand discovery and therapeutic innovation.

Organizing Project

Advancing Drug Discovery: A Webinar Series of the National Academies of Sciences, Engineering, and Medicine

Contact

Ana Ferreras

aferrerasfiel@nas.edu

Disclaimer

It is essential to the National Academies mission of providing evidence-based advice that participants in any of our meetings or events avoid political or partisan statements or commentary and maintain a culture of mutual respect. The statements and presentations during our meetings or events are solely those of the individual participants and do not necessarily represent the views of other participants or the National Academy of Sciences, which is a non-partisan, tax exempt organization that includes under its Charter the National Academy of Engineering and National Academy of Medicine, and that operates the National Research Council.

Webinar

Unique Capabilities of Cryo-EM in Structure Determination and its Potential in Drug Discovery

March 25, 2025

12:00 PM - 1:00 PM (EDT)

Past

About

As part of Advancing Drug Discovery: A Webinar Series of the National Academies of Sciences, Engineering and Medicine, a session on Unique Capabilities of Cryo-EM in Structure Determination and its Potential in Drug Discovery will be held on Tuesday, March 25th, 2025 at noon (EDT). The 60-minute session will consist of a presentation from Dr. Eva Nogales, Distinguished Professor in Molecular and Cell Biology at the University of California, Berkeley, with time for a short question and answer session at the end.

Abstract:

Cryo-EM has emerged as a powerful structural technique that is generally applicable to samples spanning from single proteins to large viruses, including integral membrane proteins and large macromolecular assemblies, even when they are flexible or involve transient, regulatory interactions. The minimal requirement concerning sample amount and the capacity to describe conformational ensembles make cryo-EM the technique of choice when characterizing samples that challenge methods like NMR or crystallography. The resolution of the structures obtained by cryo-EM has been increasing with improvement in hardware and software, making it relevant in the drug discovery arena. Dr. Nogales will introduce the technique and demonstrate its capability in studies of the microtubule cytoskeleton, molecular machinery involved in gene expression regulation, and other biological systems that had defied structural characterization by more traditional methods.

Short Speaker’s Bio:

Eva Nogales is a Distinguished Professor in Molecular and Cell Biology at the University of California, Berkeley. She studied physics at the Universidad Autónoma de Madrid, did her doctorate in biophysics at the University of Keele, UK, and carried out her postdoctoral work at the Lawrence Berkeley National Laboratory (LBNL) where she obtained the structure of tubulin using electron crystallography. She joined the University of California, Berkeley in 1998 and has been a Howard Hughes Medical Institute Investigator since 2000. She is also a Senior Faculty Scientist at LBNL. Nogales is an elected member of the National Academy of Sciences of the USA and the American Academy of Arts & Sciences, and foreign member the Real Academia de Ciencias de España and of EMBO. In 2020 she served as President of ASCB. In 2023 she received the Shaw Prize in Life Science and Medicine. Her lab uses cryo-EM to describe the structure, dynamics and interactions of large biological assemblies essential to the life of all eukaryotic cells, including microtubules and transcription and epigenetic complexes.

Organizing Project

Advancing Drug Discovery: A Webinar Series of the National Academies of Sciences, Engineering, and Medicine

Contact

Ana Ferreras

aferrerasfiel@nas.edu

Disclaimer

It is essential to the National Academies mission of providing evidence-based advice that participants in any of our meetings or events avoid political or partisan statements or commentary and maintain a culture of mutual respect. The statements and presentations during our meetings or events are solely those of the individual participants and do not necessarily represent the views of other participants or the National Academy of Sciences, which is a non-partisan, tax exempt organization that includes under its Charter the National Academy of Engineering and National Academy of Medicine, and that operates the National Research Council.

Webinar

Imaging Life Across Scales

February 25, 2025

12:00 PM - 1:00 PM (EST)

Past

About

As part of Advancing Drug Discovery: A Webinar Series of the National Academies of Sciences, Engineering and Medicine, a session on Imaging Life Across Scales will be held on Tuesday, February 25th, 2025 at noon (EDT). The 60-minute session will consist of a presentation from Dr. Lucy Collinson, Head of the Electron Microscopy Science Technology Platform at The Francis Crick Institute, with time for a short question and answer session at the end.

Abstract:

In this talk, Dr. Collinson will introduce state-of-the-art electron microscope technology, and describe how they link light, X-ray and electron microscopes into multiscale imaging workflows to reveal how cells and tissues work in health and disease. Dr. Collinson describe how we are addressing the challenge of handling, analysing and extracting new knowledge from terabytes of complex image data. She will also talk about the importance of community in advanced imaging, how we are trying to democratize advanced imaging to make it available in lower resource settings, and invite you to contribute to our understanding of transplant rejection through our citizen science program.

Short Speaker’s Bio:

Dr Lucy Collinson is an electron microscopist with a background in medical microbiology and cell biology. She has run a series of EM core technology facilities, at University College London and then at Cancer Research UK, which became part of the Francis Crick Institute in 2015. With a team of electron microscopists and physicists, she oversees more than 100 research projects with more than 60 research groups within the Crick, imaging across scales from proteins to cells to tissues to organisms. Where barriers exist in microscope technology, her team develops new imaging hardware and software solutions. Her expertise covers light microscopy, X-ray microscopy, electron microscopy, correlative and multimodal imaging, cryo microscopy, image analysis, citizen science, and microscope design and prototyping. Recent projects include the democratisation of advanced imaging technology and the use of advanced imaging across scales to understand transplant rejection.

Organizing Project

Advancing Drug Discovery: A Webinar Series of the National Academies of Sciences, Engineering, and Medicine

Contact

Ana Ferreras

aferrerasfiel@nas.edu

Disclaimer

It is essential to the National Academies mission of providing evidence-based advice that participants in any of our meetings or events avoid political or partisan statements or commentary and maintain a culture of mutual respect. The statements and presentations during our meetings or events are solely those of the individual participants and do not necessarily represent the views of other participants or the National Academy of Sciences, which is a non-partisan, tax exempt organization that includes under its Charter the National Academy of Engineering and National Academy of Medicine, and that operates the National Research Council.

Webinar

Summer Research Experiences in Drug Discovery

January 28, 2025

12:00 PM - 1:00 PM (EST)

Past

Materials

Extended Q and A Responses

About

As part of Advancing Drug Discovery: A Webinar Series of the National Academies of Sciences, Engineering and Medicine, a panel of experts on Summer Research Experiences in Drug Discovery will be held on Tuesday, January 28th, 2025 at noon (EDT). The 60-minute session will consist of a diverse panel of experts from a variety of industries and sectors that will advise students and early careers how to obtain a summer research position. The panelists will share opportunities and provide tips on how to launch a successful career path in Drug Discovery. This session is designed for early careers and those interested in pursuing a position/role in Drug Discovery.

Moderator

- Sheila Zipfel, Consultant, Zipfel Drug Discovery, LLC

Panelists:

- Katherine Bay, Senior Scientist II, Academic Curriculum Designer, Schrödinger

- Lauren Cotnoir, Early Talent Pipeline and Senior Recruiter at AbbVie

- Sandra Gabelli, Executive Director of Discovery Chemistry and Global Head of Protein & Structural Chemistry at Merck

Dr. Katherine Bay is a Senior Scientist responsible for leading the Teaching with Schrödinger program. She develops academic curricula that integrates Schrödinger’s molecular modeling tools in the classroom with the goal of making computational tools accessible to all students. Prior to joining Schrödinger, she was an Assistant Professor of Organic Chemistry and earned her Ph.D. in Organic Chemistry from UCLA. Currently, she is a Co-PI for an NSF grant titled “Introducing Molecular Modeling Experiences to Underrepresented Students” and hopes to provide industry-grade opportunities not just for the next generation of scientists but for folks who come from all walks of life.

Ms. Lauren Cotnoir is an Early Talent Pipeline and Senior Recruiter at AbbVie. She has more than 13 years of experience within University Relations, as well as more than 3 years of Career Services/Employer Relations experience within Higher Education. She has a true passion for helping early career candidates translate their academic, extracurricular and internship experiences into a rewarding and fulfilling career at an organization that is just as invested in their success.

Dr. Sandra Gabelli is the Executive Director of Discovery Chemistry and Global Head of Protein & Structural Chemistry at Merck. In 2022, she joined Merck and Co, as the global head of the Protein and Structural Chemistry. Dr. Gabelli leads the group that enables structural studies at Merck using Xray, Cryo EM and cryo ET. She channels her passion to inspire others to have a STEM career reaching out to underserved population and underrepresented minorities.

Ms. Zipfel has been a Drug Discovery Consultant for more than 26 years and specializes in small molecule drug discovery and development. She has a broad spectrum of expertise areas that include medicinal chemistry, product management, and strategic consulting.

Organizing Project

Advancing Drug Discovery: A Webinar Series of the National Academies of Sciences, Engineering, and Medicine

Contact

Ana Ferreras

aferrerasfiel@nas.edu

Disclaimer

It is essential to the National Academies mission of providing evidence-based advice that participants in any of our meetings or events avoid political or partisan statements or commentary and maintain a culture of mutual respect. The statements and presentations during our meetings or events are solely those of the individual participants and do not necessarily represent the views of other participants or the National Academy of Sciences, which is a non-partisan, tax exempt organization that includes under its Charter the National Academy of Engineering and National Academy of Medicine, and that operates the National Research Council.

Webinar

Expanding the Druggable Proteome

November 14, 2024

12:00 PM - 1:00 PM (EST)

Past

Materials

Dr. Knapp Presentation Slides

About

As part of Advancing Drug Discovery: A Webinar Series of the National Academies of Sciences, Engineering and Medicine, a session on Expanding the Druggable Proteome will be held on Thursday, November 14th, 2024 at noon (EDT). The 60-minute session will consist of a presentation from Prof. Stefan Knapp, PhD, Professor of Pharmaceutical Chemistry at Goethe University Frankfurt am Main in Germany, with time for a short question and answer session at the end.

Abstract:

Over the past two decades, the Structural Genomics Consortium (SGC) has contributed key tools for target validation, including high-resolution protein structures, chemical probes, chemogenomic inhibitor sets and disease-relevant assay systems. In the next funding period (2025-2030) of the consortium, we aim to expand the druggable proteome by developing new protein-based screening technologies supported by machine learning. In this talk, I will summarize these efforts, which include ML-assisted DNA encoded library screening and affinity selection mass spectrometry (ASMS).

Speaker Bio:

Stefan Knapp studied chemistry at the University of Marburg (Germany) and the University of Illinois (USA). He received his Ph.D. in protein crystallography from the Karolinska Institute in Stockholm. He joined Pharmacia (Nerviano, Italy) in 1999 and left the company in 2004 to establish a research group at the Structural Genomics Consortium at the University of Oxford. From 2008 to 2015 he was Professor of Structural Biology at Oxford University (UK) and from 2012 to 2015 Director of Chemical Biology at the Target Discovery Institute at Oxford University. In 2015, he joined the University of Frankfurt as Professor of Pharmaceutical Chemistry. Since 2017, he is also the CSO of the SGC (Structure Genomics Consortium) node at Goethe University Frankfurt. His research interests are the elucidation of molecular/structural mechanisms of kinase regulation using high resolution structures, the design of selective kinase inhibitors, and the inhibition of protein interaction domains such as bromodomains, which are the main readers of the epigenetic acetylation code, and E3 ubiquitylating ligases.

Organizing Project

Advancing Drug Discovery: A Webinar Series of the National Academies of Sciences, Engineering, and Medicine

Contact

Ana Ferreras

aferrerasfiel@nas.edu

Disclaimer

It is essential to the National Academies mission of providing evidence-based advice that participants in any of our meetings or events avoid political or partisan statements or commentary and maintain a culture of mutual respect. The statements and presentations during our meetings or events are solely those of the individual participants and do not necessarily represent the views of other participants or the National Academy of Sciences, which is a non-partisan, tax exempt organization that includes under its Charter the National Academy of Engineering and National Academy of Medicine, and that operates the National Research Council.

Webinar

Accelerating Drug Discovery with Machine Learning

October 1, 2024

12:00 PM - 1:00 PM (EDT)

Past

Materials

Dr. Alpha Lee's Presentation

About

As part of Advancing Drug Discovery: A Webinar Series of the National Academies of Sciences, Engineering and Medicine, a session on Accelerating Drug Discovery with Machine Learningwill be held on Tuesday, October 1st, 2024 at noon (EDT). The 60-minute session will consist of a presentation from Dr. Alpha Lee, co-founder and Chief Scientific Officer of PostEra and a faculty member at the University of Cambridge, with time for a short question and answer session at the end. Dr. Miranda Lynch, Assistant InvestigatorAssistant Investigator at Hauptman-Woodward Medical Research Institute, will be moderating this session.

Abstract:

Advances in machine learning and its impacts on daily life have captured the imagination of headlines. However, significant challenges abound in accelerating drug discovery with machine learning, a field with many unknown unknowns. In the talk, Dr. Lee will discuss the work on machine learning approaches to small molecule drug discovery, systematically accelerating each step of the design-make-test cycle. A key theme of the approach is the development of novel computational approaches that unlock and generate new sources of data. Dr. Lee will illustrate the impact of machine learning drawing from the recent work on drug discovery against viruses on pandemic concern, and point out limitations and opportunities for data-driven drug discovery.

Speaker Bio:

Dr Alpha Lee is the co-founder and Chief Scientific Officer of PostEra, and a faculty member at the University of Cambridge. He is also a co-Principal Investigator of the NIH-funded Antiviral Drug Discovery (AViDD) center "AI-driven Structure-Enabled Antiviral Platform". Dr Lee's research focuses on developing machine learning technologies to accelerate medicinal chemistry for small molecule drug discovery. He is also at the forefront of translating machine learning into biopharma impact. He was trained at Harvard University (Fulbright Scholar and George F. Carrier Fellow), and University of Oxford (DPhil). Dr Lee has been named by Forbes as 30 under 30 in Science and Healthcare in Europe.

Organizing Project

Advancing Drug Discovery: A Webinar Series of the National Academies of Sciences, Engineering, and Medicine

Contact

Ana Ferreras

aferrerasfiel@nas.edu

Disclaimer

It is essential to the National Academies mission of providing evidence-based advice that participants in any of our meetings or events avoid political or partisan statements or commentary and maintain a culture of mutual respect. The statements and presentations during our meetings or events are solely those of the individual participants and do not necessarily represent the views of other participants or the National Academy of Sciences, which is a non-partisan, tax exempt organization that includes under its Charter the National Academy of Engineering and National Academy of Medicine, and that operates the National Research Council.

Webinar

The Awesome Power of Multispecific Drugs

September 18, 2024

12:00 PM - 1:00 PM (EDT)

Past

Materials

Dr. Raymond Deshaies Presentation

About

As part of Advancing Drug Discovery: A Webinar Series of the National Academies of Sciences, Engineering and Medicine, a session on The Awesome Power of Multispecific Drugswill be held on Wednesday, September 18th, 2024 at noon (EDT). The 60-minute session will consist of a presentation from Dr. Raymond Deshaies, Senior Vice President of Global Research at Amgen, Inc, with time for a short question and answer session at the end.

Abstract:
Collectively, the biopharmaceutical industry has had an enormous impact on human health through the discovery and development of numerous safe and effective molecular medicines, including both small molecules and biologics. This success has, inevitably, raised the bar on expectations for future medicines. To thrive going forward, our industry will have to make medicines that are even safer and more effective than what came before. This is a tall order, given the relatively small number of high-conviction, accessible targets in most diseases. Nevertheless, opportunities abound, provided that we can surmount the key barriers that restrain many drug development efforts. These include overcoming biological redundancy, managing ontarget toxicity/therapeutic index, and conquering so-called undruggable targets. Multispecific medicines that either attack multiple targets simultaneously, localize the action of a drug, or link targets to powerful natural effectors offer a new approach to vanquish these perennial challenges.

Dr. Deshaies will review the challenges and opportunities and describe approaches that Amgen and other leading innovators in the biopharmaceutical industry are taking to develop the next generation of multispecific small molecule and biologic medicines for the benefit of future patients.

Speaker Bio:
Prior to joining Amgen, Deshaies served as a professor at the California Institute of Technology (Caltech) and an executive officer in Caltech’s Division of Biology and Biological Engineering. He was also an investigator at the Howard Hughes Medical Institute. He has published over 150 papers on various subjects including discoveries of Sec61 translocon, cullin–RING ubiquitin ligases, and proteolysis-targeting chimeric molecules (Protacs).
In addition to his academic work, Deshaies co-founded Proteolix in 2003. In 2011, he cofounded Cleave Therapeutics.

Deshaies holds a bachelor's degree in biochemistry from Cornell University and a Ph.D. in biochemistry from the University of California, Berkeley. He is also a member of the American Academy of Arts and Sciences and National Academy of Sciences. In 2022, Fierce Pharma named Deshaies as one of the most influential people in biopharma. He received the 2023 Jacob and Louise Gabbay Award in Biotechnology and Medicine along with Yale University professor, Craig Crews, for their pioneering work on PROTACs, or proteolysis targeting chimeras.

Organizing Project

Advancing Drug Discovery: A Webinar Series of the National Academies of Sciences, Engineering, and Medicine

Contact

Ana Ferreras

aferrerasfiel@nas.edu

Disclaimer

It is essential to the National Academies mission of providing evidence-based advice that participants in any of our meetings or events avoid political or partisan statements or commentary and maintain a culture of mutual respect. The statements and presentations during our meetings or events are solely those of the individual participants and do not necessarily represent the views of other participants or the National Academy of Sciences, which is a non-partisan, tax exempt organization that includes under its Charter the National Academy of Engineering and National Academy of Medicine, and that operates the National Research Council.

Webinar

Protein Data Bank: From two epidemics & the global pandemic to mRNA vaccines & Paxlovid

June 4, 2024

12:00 PM - 1:00 PM (EDT)

Past

Materials

Stephen K. Burley Presentation

About

As part of Advancing Drug Discovery: A Webinar Series of the National Academies of Sciences, Engineering and Medicine, we will feature the talk titled Protein Data Bank: From two epidemics & the global pandemic to mRNA vaccines & Paxlovid. The 60-minute session will consist a presentation from Dr. Stephen K. Burley, M.D., D.Phil., University Professor and Henry Rutgers Chair at Rutgers, The State University for New Jersey and a Q+A session.

Abstract

Structural biologists around the world and the Protein Data Bank (PDB) played decisive roles in combating the COVID-19 pandemic. This talk will explain how global three-dimensional (3D)biostructure data was turned into global knowledge, allowing scientists and engineers around the world to understand the inner workings of coronaviruses and develop effective SARS-CoV-2 counter measures. State-of-the-art mRNA vaccines, initially designed with guidance from single-particle cryo-electron microscopy structures of the SARS-CoV and MERS-CoV Spike Proteins, benefited more than five billion individuals around the world by preventing infections entirely or significantly reducing morbidity and mortality. Structure-guided drug discovery efforts at Pfizer, first initiated in the 2000s in response to SARS-CoV and reactivated in 2020 early in the global pandemic, yielded nirmatrelvir -- a potent inhibitor of the SARS-CoV-2 Main Protease. This targeted antiviral drug received Emergency Use Authorization from the United States Food and Drug Administration in December 2021, less than two years following public release of the viral genome sequence. It is used clinically for the treatment of acute SARS-CoV-2 infections in a fixed dose combination with ritonavir and sold under the brand name Paxlovid. Bolstered by open access to research data generated with public and private monies, particularly 3D structures of coronavirus proteins archived in the PDB, basic and applied researchers made a difference when the world desperately needed them to succeed. To underscore the importance of these contributions, I quote Dr. Anthony Fauci, former head of the National Institute of Allergy and InfectiousDisease, “Show me a person who’s vaccinated, got infected, took Paxlovid and died. I can’t find anybody.”

Speaker bio

Stephen Kevin Burley is an expert in structural biology, biophysics, computational biology, data science, structure/fragment-based drug discovery, and clinical medicine/oncology. Burley is the Director of the RCSB Protein Data Bank (rcsb.org). Within Rutgers, The State University of New Jersey he serves as University Professor and Henry Rutgers Chair, Founding Director of the Institute for Quantitative Biomedicine, and a Member of the Rutgers Cancer Institute of New Jersey, wherein he Co-Leads the Cancer Pharmacology Research Program.

Organizing Project

Advancing Drug Discovery: A Webinar Series of the National Academies of Sciences, Engineering, and Medicine

Contact

Ana Ferreras

aferrerasfiel@nas.edu

Disclaimer

It is essential to the National Academies mission of providing evidence-based advice that participants in any of our meetings or events avoid political or partisan statements or commentary and maintain a culture of mutual respect. The statements and presentations during our meetings or events are solely those of the individual participants and do not necessarily represent the views of other participants or the National Academy of Sciences, which is a non-partisan, tax exempt organization that includes under its Charter the National Academy of Engineering and National Academy of Medicine, and that operates the National Research Council.

Webinar

AI in Drug Design: Part II – Foundation and Generative AI

April 23, 2024

12:00 PM - 1:00 PM (EDT)

Past

About

As part of Advancing Drug Discovery: A Webinar Series of the National Academies of Sciences, Engineering and Medicine, this is a two part series on AI in Drug Design. The 60-minute session will consist a presentation from Dr. Marinka Zitnik, Assistant Professor at Harvard in the Department of Biomedical Informatics, and a Q+A session.

Part II – Foundation and Generative AI: Foundation and generative AI models are poised to reshape drug discovery by enabling AI to learn on its own and acquire knowledge autonomously. The speaker will discuss the development of these AI models for target discovery and drug design. Instead of training a separate model for each task in drug discovery, it is now possible to adapt large pretrained foundation models, such as language models or geometric deep learning models, for multiple tasks through fine-tuning and few-shot prompting techniques. Central to this approach is building comprehensive and diverse datasets, training self-supervised models to learn general encoders of data across scales and data modalities, and adjusting the models for an array of tasks with little to no additional training required. The speaker will also introduce Therapeutics Commons, an initiative to facilitate the use of AI across therapeutic areas (such as small molecules, macromolecules, cell and gene therapies) and the drug discovery process, from molecule design to efficacy and safety assessment, including drug repurposing. The Commons provides benchmarks, leaderboards, and access to pretrained models and datasets large enough to develop foundation models.

Speaker bio

Marinka Zitnik (https://zitniklab.hms.harvard.edu) is an Assistant Professor at Harvard in the Department of Biomedical Informatics with additional appointments at the Kempner Institute for the Study of Natural and Artificial Intelligence at Harvard University, Broad Institute of MIT and Harvard, and Harvard Data Science. Zitnik investigates the foundations of AI that contribute to the scientific understanding of medicine and therapeutic design, eventually enabling AI to learn on its own and acquire knowledge autonomously. Her research won several best paper and research awards, including Kavli Fellowship of the National Academy of Sciences, awards from International Society for Computational Biology, International Conference in Machine Learning, Bayer Early Excellence in Science, Amazon Faculty Research, Google Faculty Research, Roche Alliance with Distinguished Scientists, and Sanofi iDEA-iTECH Award. She founded Therapeutics Data Commons, a global open-science AI foundation to advance therapeutic science and is the faculty lead of the AI4Science initiative.

Organizing Project

Advancing Drug Discovery: A Webinar Series of the National Academies of Sciences, Engineering, and Medicine

Contact

Ana Ferreras

aferrerasfiel@nas.edu

Disclaimer

It is essential to the National Academies mission of providing evidence-based advice that participants in any of our meetings or events avoid political or partisan statements or commentary and maintain a culture of mutual respect. The statements and presentations during our meetings or events are solely those of the individual participants and do not necessarily represent the views of other participants or the National Academy of Sciences, which is a non-partisan, tax exempt organization that includes under its Charter the National Academy of Engineering and National Academy of Medicine, and that operates the National Research Council.

Webinar

AI in Drug Design: Part I – Molecular Drug Discovery

March 26, 2024

12:00 PM - 1:00 PM (EDT)

Past

Materials

Zitnik Presentation Slides

About

As part of Advancing Drug Discovery: A Webinar Series of the National Academies of Sciences, Engineering and Medicine, we will now feature a two part series on AI in Drug Design. The 60-minute session will consist a presentation from Dr. Zitnik, Assistant Professor at Harvard in the Department of Biomedical Informatics, on molecular drug discovery and a Q+A session.

The first part of this workshop introduces how AI is advancing molecular drug discovery. Large language models and generative AI are profoundly transforming drug design. The discussion includes self-supervised learning, which utilizes extensive unlabeled datasets, and geometric deep learning, which leverages the geometry and structure of biochemical data. These approaches give rise to generative models that help design useful molecules by generating molecular structures that maximize binding affinity with biological targets, serve as optimal binders, and have specific biochemical properties. For drugs to be effective, they must act on biological targets in relevant biological contexts. The session describes PINNACLE, a graph neural network that identifies optimal cellular contexts for drug action. PINNACLE models perform an array of tasks, including enhancing 3D structural protein representations critical in immune-oncology, predicting the effects of drugs across cell-type contexts, and nominating therapeutic targets in a cell-type specific manner.

Registration and information about AI in Drug Design: Part II – Foundation and Generative AI on April 23.

Speaker bio

Marinka Zitnik (https://zitniklab.hms.harvard.edu) is an Assistant Professor at Harvard in the Department of Biomedical Informatics with additional appointments at the Kempner Institute for the Study of Natural and Artificial Intelligence at Harvard University, Broad Institute of MIT and Harvard, and Harvard Data Science. Zitnik investigates the foundations of AI that contribute to the scientific understanding of medicine and therapeutic design, eventually enabling AI to learn on its own and acquire knowledge autonomously. Her research won several best paper and research awards, including Kavli Fellowship of the National Academy of Sciences, awards from International Society for Computational Biology, International Conference in Machine Learning, Bayer Early Excellence in Science, Amazon Faculty Research, Google Faculty Research, Roche Alliance with Distinguished Scientists, and Sanofi iDEA-iTECH Award. She founded Therapeutics Data Commons, a global open-science AI foundation to advance therapeutic science and is the faculty lead of the AI4Science initiative.

Moderator bio

M. Madan Babu is the George J. Pedersen Endowed Chair of Biological Data Science and the Director of the Center of Excellence for Data-Driven Discovery in the Department of Structural Biology at St Jude Children’s Research Hospital. He is also the Senior Vice President for Data Science and Chief Data Scientist at St Jude. Before joining St Jude, Madan was a Programme Leader at the MRC Laboratory of Molecular Biology in Cambridge, UK (2006-2020).

Madan’s research group develops data science approaches to make biological discoveries with a particular emphasis on understanding how the precise structure and intrinsically disordered regions of proteins contribute to cellular function. His interests and publications span diverse areas in life sciences relevant to human diseases and medicine. The work from his group has been recognized with national and international awards, including the Royal Society’s Francis Crick Medal and the Blavatnik Award for his work elucidating the functions of key proteins in the human genome. He was awarded the 2019 EMBO Gold Medal for his fundamental contributions to the field of computational molecular biology, specifically for his discoveries in the areas of GPCR signaling and intrinsically disordered proteins.

Madan is the Chief Editor of Molecular Systems Biology and an executive editor of Nucleic Acids Research. He is an elected member of EMBO (2016), a Fellow of the Royal Society of Chemistry (2017), a Fellow of the UK Academy of Medical Science (2021), and a Fellow of the Royal Society (2023).

Organizing Project

Advancing Drug Discovery: A Webinar Series of the National Academies of Sciences, Engineering, and Medicine

Contact

Ana Ferreras

aferrerasfiel@nas.edu

Disclaimer

It is essential to the National Academies mission of providing evidence-based advice that participants in any of our meetings or events avoid political or partisan statements or commentary and maintain a culture of mutual respect. The statements and presentations during our meetings or events are solely those of the individual participants and do not necessarily represent the views of other participants or the National Academy of Sciences, which is a non-partisan, tax exempt organization that includes under its Charter the National Academy of Engineering and National Academy of Medicine, and that operates the National Research Council.

Precision Medicine: An Innovative Approach to Treating Cancer

February 1, 2024

12:00 PM - 1:00 PM (EST)

Past

Materials

About

As part of Advancing Drug Discovery: A Webinar Series of the National Academies of Sciences, Engineering and Medicine, a session on Precision Medicine: An Innovative Approach to Treating Cancer will be held on Thursday, February 1st, 2024 at noon (EDT). The 60-minute session will consist a presentation from Dr. Gordon Mills, Director, Precision Oncology at the Knight Cancer Institute.

Precision medicine, also known as personalized medicine, is an innovative approach to tailoring disease prevention and treatment that takes into account differences in a patient’s genes, environments, and lifestyles. This approach has led to powerful new discoveries and FDA-approved treatments. This webinar will introduce precision medicine and discuss the results of the SMMART Clinical Trial. This trial tracks a patient’s cancer over time through biopsies and tests and uses that information to adjust drug combinations to stay one step ahead of the cancer. The impact of personalized medicine on the FDA approval process will also be discussed.

The next webinar in the series will be AI in Drug Design: Part I – Molecular Drug Discovery on March 26 and AI in Drug Design: Part II – Clinical Drug Development on April 23 with Dr. Marinka Zitnik, Assistant Professor at Harvard in the Department of Biomedical Informatics.

Organizing Project

Advancing Drug Discovery: A Webinar Series of the National Academies of Sciences, Engineering, and Medicine

Contact

Ana Ferreras

aferrerasfiel@nas.edu

Disclaimer

It is essential to the National Academies mission of providing evidence-based advice that participants in any of our meetings or events avoid political or partisan statements or commentary and maintain a culture of mutual respect. The statements and presentations during our meetings or events are solely those of the individual participants and do not necessarily represent the views of other participants or the National Academy of Sciences, which is a non-partisan, tax exempt organization that includes under its Charter the National Academy of Engineering and National Academy of Medicine, and that operates the National Research Council.

Beyond the Microscope: Cryo-EM's Impact on Accelerating Drug Discovery

November 16, 2023

12:00 PM - 1:00 PM (EST)

Past

Materials

Gabelli Presentation Slides

About

As part of Advancing Drug Discovery: A Webinar Series of the National Academies of Sciences, Engineering and Medicine, a session on Beyond the Microscope: Cryo-EM's Impact on Accelerating Drug Discovery will be held on Thursday, November 16th, 2023 at noon (EDT). The 60-minute session will consist a presentation from Dr. Sandra Gabelli, Head of Structural biology at Merck & co, formally Executive Director of Protein and Structural Chemistry. The talk will be moderated by Melanie Adams-Cioaba, Senior Director/General Manager at Thermo Fisher Scientific.

Over the past decade, single particle cryo electron microscopy (cryoEM) has transitioned from a niche technique to a powerful tool for structural biology. This is due, in part, to technological breakthroughs which have made it relatively routine to achieve near atomic resolutions for protein targets of pharmacological interest in complex with drug candidates. The pharmaceutical industry has historically relied heavily on X-ray crystallography to enable structure-based drug design (SBDD); however, today cryoEM is playing an ever-increasing role in that process, especially for challenging samples like large multimeric complexes, proteins that are flexible, or proteins that are difficult to express or purify. Dr. Sandra Gabelli describe the strategy and present examples were cryoEM has impacted the pipeline at Merck by enabling biology, guiding SBDD, driving protein engineering of biologics and impacting small molecule formulations with micro ED. Dr. Gabelli will also explore the potential offered by upcoming technological advancements of the technique.

Sandra Gabelli, Executive Director of Protein and Structural Chemistry at Merck. She majored in Computer Sciences from Universidad Nacional del Sur, Argentina and obtained her Ph.D. in Biophysics from Johns Hopkins University (JHU) as an NSF graduate fellow. In 2011, she was recruited as a PI to the Cardiology Division at JHU where her research was aimed at elucidating the molecular mechanism of proteins involved in signaling pathways dysregulated in diseases. Sandra channeled her passion to inspire others to have a STEM career reaching out to underserved population and underrepresented minorities. In 2022, she joined Merck and Co, as the global head of the Protein and Structural Chemistry. Sandra leads the group that enables structural studies at Merck using xray, Cryo EM and cryo ET.

Organizing Project

Advancing Drug Discovery: A Webinar Series of the National Academies of Sciences, Engineering, and Medicine

Contact

Ana Ferreras Fiel

aferrerasfiel@nas.edu

Disclaimer

It is essential to the National Academies mission of providing evidence-based advice that participants in any of our meetings or events avoid political or partisan statements or commentary and maintain a culture of mutual respect. The statements and presentations during our meetings or events are solely those of the individual participants and do not necessarily represent the views of other participants or the National Academy of Sciences, which is a non-partisan, tax exempt organization that includes under its Charter the National Academy of Engineering and National Academy of Medicine, and that operates the National Research Council.

Induced Proximity to Reprogram Transcription

October 24, 2023

12:00 PM - 1:00 PM (EDT)

Past

About

Genes that drive the proliferation, survival, invasion and metastasis of malignant cells have been identified for many human cancers. Independent studies have identified cell death pathways that eliminate cells for the good of the organism. The coexistence of cell death pathways with driver mutations suggests that the cancer driver could be rewired to activate cell death using chemical inducers of proximity (CIPs). Here we describe a new class of small molecule based molecular glues called transcriptional/epigenetic CIPs (TCIPs) that recruit the endogenous cancer driver, or a downstream transcription factor, to the promoters of cell death genes, thereby activating their expression. We show proof-of-concept for this novel modality by making compounds that induce proximity between Brd4 and Bcl6.

Nathanael Gray is a Professor of Chemical and Systems Biology at Stanford, Co-Director of Cancer Drug Discovery, Co-Leader of the Cancer Therapeutics Research Program, Member of Chem-H, and Program Leader for Small Molecule Drug Discovery for the Innovative Medicines Accelerator (IMA). His research uses the tools of synthetic chemistry, protein biochemistry, and cancer biology to discover and validate new strategies for addressing anti-cancer targets. Dr. Gray’s research has had broad impact in the areas of kinase inhibitor and degrader design and in circumventing drug resistance. Dr. Gray’s generalized strategy for structure-based design of inhibitors that stabilize the inactive kinase conformations (type II) has been widely adopted by the research community and has had a significant impact on the development of numerous inhibitors of tyrosine kinases that are currently undergoing clinical development. Dr. Gray received his PhD in organic chemistry from the University of California at Berkeley in 1999 after receiving his BS degree with the highest honor award from the same institution in 1995.

Organizing Project

Advancing Drug Discovery: A Webinar Series of the National Academies of Sciences, Engineering, and Medicine

Contact

Ana Ferreras

aferreras@nas.edu

Disclaimer

It is essential to the National Academies mission of providing evidence-based advice that participants in any of our meetings or events avoid political or partisan statements or commentary and maintain a culture of mutual respect. The statements and presentations during our meetings or events are solely those of the individual participants and do not necessarily represent the views of other participants or the National Academy of Sciences, which is a non-partisan, tax exempt organization that includes under its Charter the National Academy of Engineering and National Academy of Medicine, and that operates the National Research Council.

Summit Closed

U.S. Research Data Summit

October 10 - 11, 2023

Past

About

The National Academies of Sciences, Engineering, and Medicine appointed the U.S. Research Data Summit Planning Committee, under the auspices of the U.S. National Committee for CODATA, Board on International Scientific Organizations, to convene U.S. leaders of research data organizations across sectors who can shape and influence U.S. research data policies and practices.

The objectives of the convening are to:

increase coherence of interests and activities among the cross-sector research data organizations
increase communication and collaboration across sectors on shared priorities,
reduce duplication of effort, and
position the United States to be well-represented in international discussions on research data.

Summit invitees include research data organization leaders in academia, government, industry, and related nonprofits who can effect change in the near future.

The Summit outcomes will reflect the experience, knowledge, discussion, recommendations, and commitment to the objectives of the participating leaders. They will be informed by the results of focus groups with experts convened by the US Research Data Planning Committee from April - July 2023. The focus groups covered six prioritized topics: Cross-Sector Access to Research Data; Data for Disruption: Responding to Disasters and Shocks in Disadvantaged Communities; Data Needs for Decarbonization; Indigenous Data Governance; Justice, Equity, Diversity, and Inclusion (JEDI) Research Data Integration; and Organizational Approaches to AI Inputs and Outputs. The topics were selected based on input from the 2022 survey by the Association of Research Libraries and the U.S. National Committee for CODATA, discussions with the U.S. National Committee for CODATA, and refinement by the U.S. Research Data Summit Planning Committee.

The Summit proceedings in brief will be shared with the public.

Organizing Project

U.S. Research Data Summit

Contact

Tom Arrison

(202) 334-3755
tarrison@nas.edu

Location

National Academy of Sciences Building

2101 Constitution Ave NW

Washington DC 20418, USA

Disclaimer

It is essential to the National Academies mission of providing evidence-based advice that participants in any of our meetings or events avoid political or partisan statements or commentary and maintain a culture of mutual respect. The statements and presentations during our meetings or events are solely those of the individual participants and do not necessarily represent the views of other participants or the National Academy of Sciences, which is a non-partisan, tax exempt organization that includes under its Charter the National Academy of Engineering and National Academy of Medicine, and that operates the National Research Council.

Log In

Sign in to access your saved publications, downloads, and email preferences.

Former MyNAP users: You'll need to reset your password on your first login to MyAcademies. Click "Forgot password" below to receive a reset link via email. Having trouble? Visit our FAQ page to contact support.

Members of the National Academy of Sciences, National Academy of Engineering, or National Academy of Medicine should log in through their respective Academy portals.

Email (required)
Password (required)

Don't have an account?

Register

Email (required)
New password (required)
Retype password (required) Password must be at least 8 characters
I accept the terms of use
terms of use

Thank You

Thank you for creating a MyAcademies account!

Enjoy free access to thousands of National Academies' publications, a 10% discount off every purchase, and build your personal library.

Forgot Password

Enter the email address for your MyAcademies (formerly MyNAP) account to receive password reset instructions.

Reset Requested

We sent password reset instructions to your email . Follow the link in that email to create a new password. Didn't receive it? Check your spam folder or contact us for assistance.

Reset Password

New password (required)
Retype password (required) Password must be at least 8 characters

Reset Password

Your password has been reset.

Log In

Verify email

Verify Your Email Address

We sent a verification link to your email. Please check your inbox (and spam folder) and follow the link to verify your email address. If you did not receive the email, you can request a new verification link below

Subscribe

First Name
Last Name
Email (required)
I agree to receive emails from the National Academies (required)

We respect your privacy and will never share your information.

My Academies

U.S. National Committee for CODATA

Lead Discovery in Terra-Scale Chemical Spaces with Modular Screening and Deep Learning Approaches

Quantum Computing-Enhanced Machine Learning Algorithm Unveils Novel Inhibitors for KRAS

Unique Capabilities of Cryo-EM in Structure Determination and its Potential in Drug Discovery

Imaging Life Across Scales

Summer Research Experiences in Drug Discovery

Expanding the Druggable Proteome

Accelerating Drug Discovery with Machine Learning

The Awesome Power of Multispecific Drugs

Protein Data Bank: From two epidemics & the global pandemic to mRNA vaccines & Paxlovid

AI in Drug Design: Part II – Foundation and Generative AI

AI in Drug Design: Part I – Molecular Drug Discovery

Precision Medicine: An Innovative Approach to Treating Cancer

Beyond the Microscope: Cryo-EM's Impact on Accelerating Drug Discovery

Induced Proximity to Reprogram Transcription

U.S. Research Data Summit