Mount Sinai Launches AI Small Molecule Drug Discovery Center
New center will use cutting-edge AI to accelerate the creation of life-saving medicines
Left to Right: Ethan Grimes, BS, and Paul Morgan, MD, conduct rational drug design at the newly launched AI Small Molecule Drug Discovery Center at the Icahn School of Medicine at Mount Sinai. As part of Mount Sinai’s robust AI research enterprise, the center exemplifies the synergy between AI-driven initiatives, collaborating with key programs such as the recently announced Center for AI in Children's Health, which could identify novel targets for the drug discovery center to explore and potentially develop into new therapies. Photo credit: Mount Sinai Health System
The Icahn School of Medicine at Mount Sinai has launched the AI Small Molecule Drug Discovery Center, a bold endeavor that harnesses artificial intelligence (AI) to revolutionize drug development. The new Center will integrate AI with traditional drug discovery methods to identify and design new small-molecule therapeutics with unprecedented speed and precision.
Unlike conventional drug discovery, which can take years and cost billions, AI-driven approaches enable researchers to rapidly navigate a vast chemical landscape, including natural products, to pinpoint promising drug candidates. By leveraging Mount Sinai’s world-leading expertise in machine learning, chemical biology, and biomedical data science, the Center aims to bring innovative treatments to patients faster—particularly for diseases with urgent unmet needs, including cancer, metabolic disorders, and neurodegenerative diseases.
“At Mount Sinai, we are dedicated to redefining the future of medical innovation,” says Avner Schlessinger, PhD, Professor of Pharmacological Sciences and Associate Director of the Mount Sinai Center for Therapeutics Discovery at the Icahn School of Medicine at Mount Sinai, who will lead the Center. “By integrating artificial intelligence with cutting-edge chemistry and biology, we can dramatically accelerate drug discovery and develop new treatments for some of the most complex and pressing diseases.”
The AI Small Molecule Drug Discovery Center will focus on three core areas: designing novel drug-like molecules using generative AI, optimizing existing compounds to enhance their efficacy and safety, and predicting drug-target interactions to repurpose known drugs or natural products for new indications.
Experts in computational drug design at the new center could revolutionize the drug discovery process by transforming traditional rational drug design approaches by guidance from AI-driven predictions. By training AI models on vast datasets of molecular structures and biological activity, they can anticipate the properties of new compounds before synthesis, potentially saving years of experimental work.
A major strength of this AI-powered strategy lies in its capacity to explore chemical space on a scale far beyond human capability. “Vast combinatory possibilities in optimal drug design lead to high costs, lengthy timelines, and low success rates,” says Ming-Ming Zhou, PhD, Dr. Harold and Golden Lamport Professor in Physiology and Biophysics and Chair of the Department of Pharmacological Sciences at the Icahn School of Medicine at Mount Sinai. “AI efficiently navigates this complex landscape, pinpointing the most promising candidates—an achievement unimaginable just a few years ago.”
The AI Small Molecule Drug Discovery Center will also foster collaborations with leading pharmaceutical companies, biotech firms, and academic institutions to drive drug development. Additionally, it provides hands-on training for the next generation of scientists through seminars, internship programs, and AI-driven drug discovery hackathons.
The new Center builds on other major recent Mount Sinai AI initiatives, including the launch of its new AI building and the recent announcement of the Center for Artificial Intelligence in Children’s Health.
“AI is fundamentally reshaping how we understand and target disease at a molecular level," says Alexander Charney, MD, PhD, Vice Chair, Windreich Department of Artificial Intelligence and Human Health, and Associate Professor of Artificial Intelligence and Human Health, Psychiatry, Genetics and Genomic Sciences, and Neuroscience at the Icahn School of Medicine at Mount Sinai. He is also Director of The Charles Bronfman Institute for Personalized Medicine. "By integrating artificial intelligence with genetic insights, we can move beyond conventional drug discovery to design precision therapeutics tailored to the underlying biology of neuropsychiatric and many other complex disorders.”
The center is guided by a distinguished Scientific Advisory Board, composed of leading experts in drug discovery and machine learning:
- Jian Jin, PhD, Mount Sinai Professor in Therapeutics Discovery and Director of the Mount Sinai Center for Therapeutics Discovery at the Icahn School of Medicine at Mount Sinai, a renowned expert in synthetic chemistry and drug development with extensive experience spanning both academia and industry.
- Dr. Zhou, who also serves as Co-Director of the Drug Discovery Institute and Professor of Oncological Sciences. His research focuses on the mechanisms of gene transcription in health and disease, with a strong emphasis on epigenetic drug discovery.
- Marta Filizola, PhD, Sharon and Frederick Klingenstein-Nathan Kase, MD Professor and Dean of the Graduate School of Biomedical Sciences, a leading authority in the computational biophysics of membrane proteins. With more than 25 years of experience, she has pioneered the use of AI and theoretical chemistry to advance drug discovery and development.
- Girish Nadkarni, MD, MPH, CPH, Chair of the Windreich Department of Artificial Intelligence and Human Health at the Icahn School of Medicine at Mount Sinai, where he is also Director of the Hasso Plattner Institute for Digital Health and Irene and Dr. Arthur M. Fishberg Professor of Medicine. A trailblazer in AI and digital health, he founded several companies and has several patents related to AI.
The Center will initially focus on building an advanced AI infrastructure and initiating key drug discovery projects. Over the next two years, Mount Sinai expects to make significant breakthroughs in AI-driven drug design, further cementing its role as a leader in biomedical innovation.
"The launch of the AI Small Molecule Drug Discovery Center reflects our commitment to pushing the boundaries of biomedical innovation,” Eric J. Nestler, MD, PhD, Director of The Friedman Brain Institute, Dean for Academic Affairs, and Nash Family Professor in the Nash Family Department of Neuroscience at the Icahn School of Medicine at Mount Sinai, and Chief Scientific Officer of the Mount Sinai Health System. “By harnessing the power of AI, we are transforming the way new medicines are discovered and developed, bringing hope to patients who need breakthrough treatments faster than ever before."
“We’re at the dawn of a new era in drug discovery,” Dr. Schlessinger added. “By combining AI, computational chemistry, and biomedical research, we’re not just making drug discovery faster—we’re making it smarter, more efficient, and more tailored to the biological complexity of human disease.”
Learn more about the AI Small Molecule Drug Discovery Center at: https://icahn.mssm.edu/research/ai-drug-discovery-center
About the Icahn School of Medicine at Mount Sinai
The Icahn School of Medicine at Mount Sinai is internationally renowned for its outstanding research, educational, and clinical care programs. It is the sole academic partner for the eight- member hospitals* of the Mount Sinai Health System, one of the largest academic health systems in the United States, providing care to New York City’s large and diverse patient population.
The Icahn School of Medicine at Mount Sinai offers highly competitive MD, PhD, MD-PhD, and master’s degree programs, with enrollment of more than 1,200 students. It has the largest graduate medical education program in the country, with more than 2,600 clinical residents and fellows training throughout the Health System. Its Graduate School of Biomedical Sciences offers 13 degree-granting programs, conducts innovative basic and translational research, and trains more than 500 postdoctoral research fellows.
Ranked 11th nationwide in National Institutes of Health (NIH) funding, the Icahn School of Medicine at Mount Sinai is among the 99th percentile in research dollars per investigator according to the Association of American Medical Colleges. More than 4,500 scientists, educators, and clinicians work within and across dozens of academic departments and multidisciplinary institutes with an emphasis on translational research and therapeutics. Through Mount Sinai Innovation Partners (MSIP), the Health System facilitates the real-world application and commercialization of medical breakthroughs made at Mount Sinai.
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* Mount Sinai Health System member hospitals: The Mount Sinai Hospital; Mount Sinai Beth Israel; Mount Sinai Brooklyn; Mount Sinai Morningside; Mount Sinai Queens; Mount Sinai South Nassau; Mount Sinai West; and New York Eye and Ear Infirmary of Mount Sinai.
About the Mount Sinai Health System
Mount Sinai Health System is one of the largest academic medical systems in the New York metro area, with 48,000 employees working across seven hospitals, more than 400 outpatient practices, more than 600 research and clinical labs, a school of nursing, and a leading school of medicine and graduate education. Mount Sinai advances health for all people, everywhere, by taking on the most complex health care challenges of our time—discovering and applying new scientific learning and knowledge; developing safer, more effective treatments; educating the next generation of medical leaders and innovators; and supporting local communities by delivering high-quality care to all who need it.
Through the integration of its hospitals, labs, and schools, Mount Sinai offers comprehensive health care solutions from birth through geriatrics, leveraging innovative approaches such as artificial intelligence and informatics while keeping patients’ medical and emotional needs at the center of all treatment. The Health System includes approximately 9,000 primary and specialty care physicians and 11 free-standing joint-venture centers throughout the five boroughs of New York City, Westchester, Long Island, and Florida. Hospitals within the System are consistently ranked by Newsweek’s® “The World’s Best Smart Hospitals, Best in State Hospitals, World Best Hospitals and Best Specialty Hospitals” and by U.S. News & World Report's® “Best Hospitals” and “Best Children’s Hospitals.” The Mount Sinai Hospital is on the U.S. News & World Report® “Best Hospitals” Honor Roll for 2024-2025.
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