Posts in category: Medicine

Michel William Jeffrey Sadelain was born in Paris, France on 21 April, 1960. He received his baccalaureat degree in Math in France and MD degree from the University Paris in 1984, training with Pr. Gabriel Richet.  He continued his training in Immunology at the University of Alberta (Edmonton, Canada) with Dr. Thomas Wegmann and pursued his postdoctoral research at the Whitehead Institute for Biomedical Research at MIT (Cambridge, MA) with Dr. Richard Mulligan (1989-94). In 1994 Dr. Sadelain began his work at Memorial Sloan Kettering Cancer Center, where he founded the Center for Cell Engineering in 2007.

Dr. Sadelain’s research has been on the forefront of stem cell and T cell engineering, with the aim of devising safe, effective, and ultimately curative treatments for severe hereditary blood disorders and cancers. His lab conceived receptors for antigens that he named chimeric antigen receptors (CARs) and identified CD19 as an optimal therapeutic target, for which he provided the first experimental evidence by demonstrating that human T cells could be engineered to effectively target CD19+ lymphomas and leukemias in mice (Brentjens et al, 2003). His lab obtained the first FDA approval for a CD19 CAR therapy trial in the US. In 2013, his team was the first to report dramatic responses to CD19 CAR therapy in adults with relapsed and refractory acute lymphocytic leukemia (ALL) leading to long-term survival.  CAR therapies were approved by the FDA in 2017. The advent of CD19 CAR therapy has paved the way for a nascent global cell therapy industry that is poised to advance other cell-based medicines and regenerative medicine.

Michel Sadelain is an author of over 280 papers and his research has led to the creation of over 60 patents. He previously served on the NIH Recombinant DNA Advisory Committee, as President of the American Society for Gene and Cell Therapy, and is an elected Fellow of the American Association of Cancer Research.  He is the recipient of the Cancer Research Institute’s Coley Award for Distinguished Research in Tumor Immunology, the Sultan Bin Khalifa International Award for Innovative Medical Research on Thalassemia, the NYIPLA Inventor of the Year award, the Passano award, the Pasteur-Weizmann award, the Gabbay award, the INSERM International Prize Laureate, the ARC Foundation Léopold Griffuel award, the Outstanding Achievement Award from the American Society for Gene and Cell Therapy, the Clarivate Citation Laureate in Physiology or Medicine for breakthrough research advancing CAR therapy for the treatment of cancer, and in 2024 the Breakthrough Prize in Life Sciences and the Canada Gairdner Award.

Jerry R. Mendell, MD, is Emeritus Professor at Nationwide Children’s Hospital where he held the Dwight E. Peters and Juanita R. Curran Endowed Chair in Pediatric Research at the Abigail Wexner Research Institute at Nationwide Children’s Hospital. He has been elected to the National Academy of Medicine and recently received the ASGCT Translational Medicine Award that was named in his honor and has been given yearly to accomplished scientists.

He did Neurology residency at Columbia University’s New York Neurological Institute. His post-doctoral fellowship at the Medical Neurology Branch of NIH began his career in neuromuscular disease. He has published >400 articles with a focus on neuromuscular disease and authored books on Skeletal Muscle Disease, Peripheral Nerve Disorders, and Gene Therapy.

Early work in DMD described a vascular pathway responsible for muscle damage in Duchenne muscular dystrophy (DMD) now confirmed by the link to nNOS binding sites of muscle. The first breakthrough in treatment was described in 1989, as efficacy of corticosteroids in DMD (Mendell JR, et al. N Engl J Med 320:1592-1597, 1989). Prednisone or one its corticosteroid variants now standard of care for DMD. Since then, research has moved toward molecular-based strategies. In 1999 Dr. Mendell performed the first in-human clinical trial using AAV for gene transfer to skeletal muscle. In March 2007, Dr. Mendell’s gene therapy in limb-girdle muscular dystrophy type 2D, demonstrated sustained gene expression for more than 6 months, an important milestone for the field (Mendell JR Ann Neurol 2009;66:290-297). In a similar gene therapy approach for DMD, he demonstrated that expressing the transgene into deleted domain resulted in rejection of the gene product because of transgene immunity (Mendell JR et al N Engl J Med;363:1429-37).

He was instrumental in establishing the international incidence of DMD at birth at 1:5000 (Mendell JR, et al Ann Neurol 2012;71:304-313). Clinical Trials led by Dr. Mendell in exon skipping were noteworthy as the first therapeutic agent to show increased dystrophin expression following long-term exon skipping outcomes demonstrating slowing of disease progression (Mendell al. Ann Neurol 2013; 74:637-47; Ann Neurol 2016; 79:257-271). Eteplirsen (Exondys 51) is approved by the FDA for commercial use.

He was the principal investigator for SMA gene therapy, the first systemically delivered gene showing achieving safety and efficacy (Mendell JR, et al N Engl J Med Nov 2017). This was a major milestone saving the lives of infants with gene delivery by intravenous administration. This work received Science Magazine 2017 Breakthrough of the Year Award.  SMA gene therapy has now been approved by the FDA for clinical therapy as Onasemnogene Abeparvovec (Zolgensma®, Novartis, Inc). Based on SMA gene therapy, newborn screening is now established in 49 states throughout US.

Currently Dr. Mendell is actively engaged in systemic delivery of micro-dystrophin-DMD gene therapy. He is the Principal Investigator and as a result gene therapy has been approved by FDA treatment of DMD patients 4-5 years old (Elevidys®, Sarepta, Inc.). Studies are underway to obtain gene therapy treatment for all DMD patients.

Dr. Mendell now serves as a Senior Advisor for Sarepta Therapeutics.

This biography was written in the year the prize was awarded.

Dan Hung Barouch  received his undergraduate B.A. degree summa cum laude from Harvard College in 1993, his Ph.D. degree from Oxford University in 1995 on a Marshall Scholarship, and his M.D. degree summa cum laude from Harvard Medical School in 1999. He completed clinical training in Internal Medicine at Massachusetts General Hospital in 2001 and in Infectious Diseases at Brigham and Women’s Hospital in 2004. He founded and currently serves as the Director of the Center for Virology and Vaccine Research at Beth Israel Deaconess Medical Center, and he is the William Bosworth Castle Professor of Medicine at Harvard Medical School. Dr. Barouch’s laboratory focuses on studying the immunology and pathogenesis of viral infections and developing novel vaccine and treatment strategies. He pioneered the creation of a series of vaccine platform technologies, including novel recombinant adenovirus vectors such as Ad26. He utilized this technology to develop vaccine candidates for multiple pathogens of global significance, including HIV-1, Zika virus, tuberculosis, and most recently SARS-CoV-2. He utilized the Ad26 vector to develop a COVID-19 vaccine, which led to the Johnson & Johnson Ad26.COV2.S vaccine that has now been administered to hundreds of millions of people worldwide. This vaccine is stable without the need for subzero freezing and can be produced inexpensively, which are critical properties for a global COVID-19 vaccine. Dr. Barouch has authored over 350 research papers. He was elected to the National Academy of Medicine in 2020, and he is a member of the American Society for Clinical Investigation and the Association of American Physicians. He has received numerous awards for his work, including the Oswald Avery Award from the Infectious Diseases Society of America (2012), Bostonian of the Year (2016, 2021), Best Academic Research Team from the World Vaccine Congress (2019), Bloomberg 50 Most Influential People (2020), Global Citizen Hero from the American Red Cross (2021), STAT Madness Winner (2021), Ray Stata Leadership and Innovation Award from the Massachusetts High Technology Council (2021), George Ledlie Prize from Harvard University (2021), and Hero Among Us from the Boston Celtics (2021).

This biography was written in the year the prize was awarded.

Sarah Catherine Gilbert received her undergraduate degree in Biological Sciences (specialising in Microbiology) from the University of East Anglia with a prize for the highest final examination marks in the School of Biological Sciences, in 1983, and a PhD in Biochemistry from the University of Hull in 1986. She completed post-doctoral appointments at the Brewing Research Foundation and the University of Leicester before moving to Delta Biotechnology in 1989, working on the production of recombinant human blood proteins from yeast. She moved to Oxford University in 1994, as a senior post-doctoral researcher before being appointed University Research Lecturer (1999), Reader in Vaccinology (2004), Jenner Investigator (2006) and Professor of Vaccinology (2010). In 2021 Professor Gilbert was appointed to the Saïd Chair of Vaccinology and became Head of the Outbreak Pathogens Vaccine Group in the Pandemic Sciences Institute in the Nuffield Department of Medicine at Oxford University.

Professor Gilbert’s research has been on the development of vaccines against infectious diseases, including vaccine design, preclinical and clinical assessment of vaccines produced using viral vector platform technologies. This has included the initial development of the novel simian adenoviral vectored vaccine technologies ChAdOx1 and ChAdOx2. Her research has included the development of Good Manufacturing Practice (GMP) manufacturing processes and assays to allow ChAdOxvectored vaccines to be produced for clinical trials. She has led projects on the clinical development of ChAdOx1-vectored vaccines against influenza and Middle East Respiratory Syndrome (MERS), with clinical trials of the latter taking place in the UK and in the Kingdom of Saudi Arabia. In 2020 Professor Gilbert initiated and led the rapid production and development of a vaccine against SARSCoV-2 (Vaxzevria) which was licensed to AstraZeneca and is now in use in over 180 countries.

Professor Gilbert was made a Dame Commander of the British Empire in 2021 in recognition of her work during the 2020 pandemic. Her book, Vaxxers, describing the development of Vaxzevria was a Sunday Times bestseller. Other awards include the Royal Society of Medicine Gold Medal, the Princesa de Asturias Award for Technical and Scientific Research, 2021 and the Sunhak Peace prize 2022.

This biography was written in the year the prize was awarded.

David Liu graduated first in his class at Harvard College in 1994. During his doctoral research at UC, Berkeley, Liu initiated the first general effort to expand the genetic code in living cells. He earned his Ph.D. in 1999 and became an assistant professor of chemistry and chemical biology at Harvard University in the same year. He was promoted to an associate professor in 2003 and to a full professor in 2005. Liu became a Howard Hughes Medical Institute investigator in 2005 and joined the JASONs, academic science advisors to the U.S. government, in 2009. In 2016, he became a Core Institute Member and a Vice-Chair of the Faculty at the Broad Institute of MIT and Harvard, and the Director of the Chemical, Biology, and Therapeutics Science Program.

Professor Liu is the Richard Merkin Professor and the director of the Merkin Institute of Transformative Technologies in Healthcare, the vice chair of the faculty at the Broad Institute of Harvard and MIT, the Thomas Dudley Cabot Professor of the Natural Sciences at Harvard University, and a Howard Hughes Medical Institute (HHMI) investigator. Liu’s research integrates chemistry and evolution to illuminate biology and enable next-generation therapeutics. His major research interests include the engineering, evolution, and in vivo delivery of genome editing proteins such as base editors to study and treat genetic diseases; the evolution of proteins with novel therapeutic potential using phage-assisted continuous evolution (PACE); and the discovery of bioactive synthetic small molecules and synthetic polymers using DNA-templated organic synthesis and DNA-encoded libraries. Base editing, the first general method to perform precision gene editing without double-stranded breaks, and a Science 2017 Breakthrough of the Year finalist, as well as prime editing, PACE, and DNA-templated synthesis are four examples of technologies pioneered in his laboratory. These technologies are used by thousands of laboratories around the world and have enabled the study and potential treatment of many genetic diseases.

Liu has been elected as a member of the U.S. National Academy of Sciences, the U.S. National Academy of Medicine, and the American Association for the Advancement of Science. He has earned several University-wide distinctions for teaching at Harvard, including the Joseph R. Levenson Memorial Teaching Prize, the Roslyn Abramson Award, and a Harvard College Professorship. Liu has published more than 200 papers and is the inventor on more than 75 issued U.S. patents. His research accomplishments have earned distinctions including the Ronald Breslow Award for Biomimetic Chemistry, the American Chemical Society David Perlman Award, ACS Chemical BiologyAward, the American Chemical Society Pure Chemistry Award, the Arthur Cope Young Scholar Award, the NIH Marshall Nirenberg Lecturer, and awards from the Sloan Foundation, Beckman Foundation, NSF CAREER Program, and Searle Scholars Program. In 2016 and 2020, he was named one of the Top 20 Translational Researchers in the world by Nature Biotechnology, and was named one of Nature’s 10 researchers in the world and to the Foreign Policy Leading Global Thinkersin 2017. He is the founder or co-founder of several biotechnology and therapeutics companies, including Beam Therapeutics, Prime Medicine, Editas Medicine, Pairwise Plants, Exo Therapeutics, and Chroma Medicine.

This biography was written in the year the prize was awarded.

Stephen Strittmatter obtained an A.B. in Biochemistry in 1980 from Harvard College, and received his M.D. and PhD. In Pharmacology from Johns Hopkins University in 1986. He completed his medical internship and neurology residency at Massachusetts General Hospital. He joined Yale University faculty in 1993, and currently holds the Vincent Coates Professorship of Neurology and is Professor of Neuroscience. He is a Founding Director of Yale Cellular Neuroscience, Neurodegeneration and Repair Program, Yale Alzheimer Disease Research Center, and Yale Memory Disorders Clinic.

Professor Strittmatter is the author of over 240 original reports. He has been recognized by several prestigious awards and honors, including: the Ameritec Award, John Merck Scholar Award, Donaghue Investigator Award, McKnight Brain and Memory Disorders Award, Alzheimer Association Zenith Fellow Award, Senator Jacob Javits Award in the Neurosciences, and an NINDS Outstanding Investigator Award. He is a member of several editorial boards and scientific societies.

Professor Strittmatter has made outstanding contributions to the field of neural repair, including the identification of a Nogo Receptor pathway that plays a central role in determining the ability of axons to extend and reconnect after injury. He showed that glia-derived inhibitors bind axonal Nogo Receptor to activate RhoA and prevent neural plasticity, sprouting, regeneration, and recovery. His work revealed that soluble Nogo Receptor decoy therapy promotes recovery in preclinical spinal cord trauma and ischemic stroke, and led to clinical trials in chronic spinal cord injury.

This biography was written in the year the prize was awarded.

Robin Franklin obtained his BSc. in Neuroscience from University College London in 1985, a BVetMed in Veterinary Medicine from Royal Veterinary College London in 1988, and His PhD in Neuroscience from University of Cambridge in 1992. He spent most of his career at the University of Cambridge starting as research fellow in 1991, until he attained the professorship in 2005. He is currently at the Wellcome Trust-Medical Research Council Cambridge Stem Cell Institute, the Director of the UK MS Society Cambridge Center for Myelin Repair at the University of Cambridge, and a Professor of Stem Cell Medicine at the University’s Clinical School. He previously served as a Professor of Neuroscience in the University’s School of Biology.

Professor Franklin’s main research questions focused on how stem cells in the adult brain respond to injury, how they contribute to regeneration, and how they are affected by aging. He has made many outstanding original contributions to

in the University’s School of Biology.

myelin biology that have had important applications for clinical neurology, especially multiple sclerosis. He has been a pioneer in the biology of remyelination, an area in which he is widely acknowledged as the world’s leading expert, and where he has made many seminal contributions. These include: identifying the role of the innate immune response, the effects of aging and how they can be reversed, the activation and plasticity of CNS stem cells following injury, the transcriptional and epigenetic control of CNS stem cell differentiation, and the first demonstrations of remyelination by transplanted oligodendrocyte progenitor cells and olfactory ensheathing cells, the latter led to a successful clinical trial in spinal cord injury in which he played a central role.

His work opened the exciting possibility of pharmacological enhancement of remyelination, which has implications for a whole range of CNS conditions, not just MS. His studies on the remyelination enhancing properties of metformin and RXR are the basis of current clinical trials. His extensive publications of over 270 peer-reviewed papers in this area include many landmark studies for which he is recognized worldwide. He was the recipient of the Barancik International Prize for Research Innovation in 2017, and was elected as a Fellow of the Academy of Medical Sciences in 2016.

This biography was written in the year the prize was awarded.

Stuart Orkin received his B.S. degree from MIT and MD degree from Harvard Medical School (HMS) in 1972. He trained in general pediatrics and hematology/oncology at Boston Children’s Hospital, and served as a postdoctoral Research Associate at the NIH in the Laboratory of Philip Leder in 1973-1975. Following his appointment to the HMS faculty in 1978, he ascended to the rank of Professor in 1986. He served as the Chairman of the Department of Pediatric Oncology at Dana Farber Cancer Institute from 2000-2016. Currently, Professor Orkin is the David G. Nathan Distinguished Professor of Pediatrics at HMS, and an Investigator of the Howard Hughes Medical Institute.

Professor Orkin’s research has been on the forefront of molecular blood cell development and genetics for nearly 4 decades. His early work led to the first comprehensive molecular dissection of an inherited disorder (the thalassemia syndromes). He characterized genes responsible for other human blood disorders, including X-linked chronic granulomatous disease (the first positional cloning of a disease gene). Orkin identified the first hematopoietic transcription factors (the GATA family) and characterized their roles in blood cell development and cancer. His recent studies on BCL11A, a critical repressor of fetal hemoglobin (HbF), have illuminated regulation of the fetal-to-adult switch and improved prospects for HbF reactivation as genetic or pharmacological therapy of the thalassemias and sickle cell disease.

An author of 600 papers, Professor Orkin is an elected member of the National Academy of Sciences (NAS), National Academy of Medicine (NAM), American Academy of Arts and Sciences, and American Philosophical Society. He has been recognized with numerous awards, including the E. Mead Johnson Award of the American Academy of Pediatrics, Warren Alpert Prize, Helmut Horten Foundation Prize, Distinguished Research Award of the Association of American Medical Colleges, E. Donnall Thomas, Dameshek and Basic Science Mentor Awards of the American Society of Hematology, Jessie Stevenson Kovalenko Medal of the NAS, William A. Allan Award of the American Society of Human Genetics, George M. Kober Medal of the American Association of Physicians, and the Mechthild Esser Nemmers Prize in Medical Science.

This biography was written in the year the prize was awarded.

Steven Teitelbaum obtained his B.A. from Columbia College in New York and his M.D. from Washington University in St. Louis in 1964, where he rose through academic ranks to become Wilma and Roswell Messing Professor of Pathology, Immunology, and Medicine.

Professor Teitelbaum scientific studies included confirming the hematopoietic lineage of the osteoclast and the mechanisms by which the cell resorbs bone, which contributed to development of anti-osteoporosis drugs and understanding the importance of clinically arresting osteoclast function as opposed to formation. He documented that the αvβ3 integrin is central to osteoclast function and collaborated with industry to design the first specific inhibitor of the complex. His work has given major insights into the means by which inflammatory cytokines modulate osteoclast generation and its capacity to resorb bone contributing to effective therapy for joint destructive conditions such as rheumatoid arthritis. Additionally, he developed a new paradigm for the pathogenesis of glucocorticoid-induced osteoporosis eventuating in a novel approach to its treatment. Moreover, his recent work defined the relationship of osteoclast to obesity and established that a new family of drugs, which do not activate PPARϒ, effectively treat type 2 diabetes without the fracture-predisposing properties of presently used thiazolidinediones.

Professor Teitelbaum has published over 340 papers, and over 50 books and book chapters. He is a member of the editorial boards of several journals, including Cell Metabolism and Experimental Medicine. He held leadership positions in several professional organizations, including the American Society for Bone and Mineral Research. Professor Teitelbaum received numerous honors and awards including the Ann Doner Vaughan Kappa Delta Award, NIAMS Guru Award, and Gideon A. Rodan Award.

This biography was written in the year the prize was awarded.

Bjorn Olsen received his medical and doctoral degrees from the University of Oslo in 1967, where he became a faculty member at the Anatomical institute and conducted molecular studies on the structure of collagen. In 1971, he went to the United States to work with Professor Darwin Prockop, and one year later joined the faculty of the Department of Biochemistry, at Rutgers Medical School, now UMDNJ-Robert Wood Johnson Medical School, where he was promoted to the rank of professor in 1976. In 1985, Professor Olsen was appointed the Hersey professor of anatomy and cellular biology at Harvard Medical School, becoming later a Hersey professor of cell biology. Since 1996, he has also been a senior member of the staff at the Forsyth Institute and a professor of developmental biology at Harvard School of Dental Medicine (HSDM) since 2002. ​​He was the Chairman at the Harvard-Forsyth Department of Oral Biology between 1996-2002, and the Dean for research at the Harvard School of Dental Medicine between 2005-2017. research 2005-2017.

His work contributed to mapping the gene for craniofacial disorder Cherubism, the identification of the mutation in the myeloid signaling regulator SH3BP2, the mapping of the gene responsible for excess bone formation in craniometaphyseal dysplasia, and the identification of mutations in pyrophosphate transporter ANK and its regulation of bone mass.

Professor Olsen’s work on the genetics of vascular syndromes has led to identification of mutations governing pyrophosphate transport (ANK) and vascular endothelial growth factor receptor 2. These findings have led to unraveling of complex developmental and disease mechanisms at the intersection between skeletal and vascular biology, as well as highlighting the role of vascular endothelial growth factor in the differentiation that mesenchymal stem cells to osteoblasts and bone marrow adipocytes

Professor Olsen has published over 400 papers. He is a member of the editorial boards of several journals, including the Journal of Cell Biology, Molecular Biology of the Cell, Journal of Biological Chemistry, and Bone and Development. He has been editor-in-chief of Matrix Biology, and founder and editor-in-chief of BioMed Central’s Journal of Negative Results in Biomedicine. He held leadership positions in several professional organizations, including the International Society for Matrix Biology. Professor Olsen has received numerous honors and awards including Fell-Muir Award and Humboldt Research Award.\

This biography was written in the year the prize was awarded.