Congratulations to Our 2021 Annual Meeting Award Winners

Outstanding Achievement Award

The Outstanding Achievement Award (OAA) recognizes an ASGCT Member who has achieved a pioneering research success, specific high-impact accomplishment, or lifetime of significant contributions to the field of gene and cell therapy. It is the Society’s highest honor. The recipient is celebrated at the Annual Meeting by presenting a plenary lecture highlighting their scientific accomplishments that led to the award.

Carl June, M.D.
Richard W. Vague Professor in Immunotherapy
University of Pennsylvania

Dr. June maintains a research laboratory that studies various mechanisms of lymphocyte activation that relate to immune tolerance and adoptive immunotherapy for cancer and chronic infection. In 2011, his research team published findings detailing a new therapy in which patients with refractory and relapsed chronic lymphocytic leukemia were treated with genetically engineered versions of their own T cells. The treatment has also now been used with promising results to treat children with refractory acute lymphoblastic leukemia, and adults with refractory lymphoma. CTL019, the CAR T cell developed in the June laboratory was the first gene therapy to be approved by the U.S. FDA in August 2017. June has published more than 400 manuscripts and is the recipient of numerous prizes and honors, including election to the Institute of Medicine in 2012 and the American Academy of Arts and Sciences in 2014, the Paul Ehrlich and Ludwig Darmstaedter Prize (shared w J. Allison), the Novartis Prize in Immunology (shared w Z. Eshhar and S. Rosenberg), the Karl Landsteiner Memorial award, the Karnofsky Prize from the American Society of Clinical Oncology, the Albany Medical Prize and a lifetime achievement award from the Leukemia and Lymphoma Society.

Michel Sadelain, M.D., Ph.D.
Director, Center for Cell Engineering
Memorial Sloan-Kettering Cancer Center

Sadelain’s research focuses on the mechanisms governing transgene expression, stem cell engineering, and genetic strategies to enhance immunity against cancer. His laboratory has made several seminal contributions to the field of chimeric antigen receptors (CARs), from their conceptualization and optimization to their clinical translation for cancer immunotherapy. His group was the first to publish dramatic molecular remissions in patients with chemorefractory acute lymphoblastic leukemia following treatment with autologous CD19-targeted T cells. Sadelain is the recipient of numerous awards, including the Inserm International Prize, the Jacob and Louise Gabbay Award in Biotechnology and Medicine, 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 NYPLA Inventor of the Year award, the Passano award and the Pasteur-Weizmann award. He previously served on the NIH Recombinant DNA Advisory Committee and as President of the American Society for Gene and Cell Therapy.

Sonia Skarlatos Public Service Award

Named for its inaugural co-recipient and tireless gene therapy advocate,the Sonia Skarlatos Public Service Award (PSA) recognizes a person or group that has consistently fostered and enhanced the field of gene and cell therapy through governmental agencies, public policy groups, public education, or non-governmental charitable organizations.

Larry Corey, M.D.
President and Director Emeritus
Fred Hutchinson Cancer Research Center

Lawrence Corey is past president and director of the Fred Hutchinson Cancer Research Center and professor of its Vaccine and Infectious Disease Division. He is also a professor of Medicine and Laboratory Medicine & Pathology at the University of Washington, was head of the UW Virology Division during 1978-2010, and led the AIDS Clinical Trials Group (ACTG) 1987-1992. He has been the Founder and PI of the HIV Vaccine Trials Network (HVTN) since its inception in 1999, and is an internationally renowned expert in virology, viral immunology and vaccine development. Most recently, he was asked to co-lead the COVID-19 Prevention Network (CoVPN) vaccine program, which is responsible for implementing multiple COVID vaccine efficacy trials in the U.S. and overseas.Corey was the first to demonstrate that an antiviral compound (acyclovir) could be safely and effectively administered to control a chronic viral infection. He pioneered the use of viral load as a clinical benefit predictor and his studies on early administration of cART helped reduce HIV morbidity and mortality globally. Under his leadership at the ACTG, highly active antiretroviral therapy (HAART) was clinically demonstrated and AZT was shown to reduce maternal-fetal transmission; stimulating the use of antiretrovirals to avert pediatric HIV infections internationally. As PI of the HVTN, Corey established an NIH-supported global network of scientists on five continents with over 70 clinical trial sites, 45 of which are in sub-Saharan Africa. The clinical trial sites, statistical unit and laboratories were an important component of the CoVPN. Corey received his BS and MD from the University of Michigan and his infectious diseases training at UW. He is a member of the National Academy of Medicine and the American Academy of Arts and Sciences, and was the recipient of the Parran Award for his work in HSV-2, the American Society of Microbiology Cubist Award for his work on antivirals, and the University of Michigan Medical School Distinguished Alumnus Award. He is one of the most highly cited biomedical researchers in the last 20 years and is the author, coauthor or editor of over 1000 scientific publications.

Kathleen Neuzil, M.D., MPH, FIDSA
Professor
University of Maryland School of Medicine

Dr. Kathleen Neuzil is the Myron M. Levine Professor in Vaccinology, Professor of Medicine and Pediatrics, and the Director of the Center for Vaccine Development and Global Health at the University of Maryland School of Medicine. She is an internationally recognized research scientist and advocate in the field of vaccinology. Throughout her career, Dr. Neuzil has conducted clinical and epidemiologic studies on vaccine-preventable diseases, yielding high-profile publications that inform policy decisions and public health actions. Dr. Neuzil’s work has spanned dozens of low-resource countries with multiple vaccines, including influenza, rotavirus, human papillomavirus, Japanese encephalitis, typhoid conjugate vaccines, and most recently, COVID-19 vaccines. Dr. Neuzil is central to the domestic and global response to COVID. As a co-PI of the NIH-funded Leadership Group for the Vaccine and Treatment Evaluation Unit network, Dr. Neuzil is part of the strategic team evaluating COVID vaccines and therapeutics in the US and was part of the study team who designed the first COVID-19 clinical vaccine trial in the U.S. The CVD is likewise participating in COVID treatment and post-exposure prophylaxis trials. Dr. Neuzil also directs TyVAC, the Typhoid Vaccine Acceleration Consortium, with the goal to accelerate the introduction of typhoid conjugate vaccines into low-resource countries. She has more than 230 scientific publications on vaccines and infectious diseases. Dr. Neuzil's research capabilities are complimented by 20 years of involvement in domestic and international policy, including past membership on the US Centers for Disease Control and Prevention’s Advisory Committee on Immunization Practices. She is a member of the World Health Organization Strategic Advisory Group of Experts on Immunization and is a member of the prestigious National Academy of Medicine.

Outstanding New Investigator Award

Winners of the Outstanding New Investigator (ONI) Award come from academia, research foundations, government, or industry, and are recognized each year based on their contributions to the field of gene and cell therapy. Each awardee will present a plenary lecture at the ASGCT Annual Meeting highlighting their scientific accomplishments that led to the Award.

Marcela Maus, M.D., Ph.D.
Director of Cellular Immunotherapy
Massachusetts General Hospital

Marcela Maus, M.D., Ph.D. Marcela Maus, M.D., Ph.D., is currently an Associate Professor at Harvard Medical School, the Paula O’Keefe Chair in Oncology and Director of Cellular Immunotherapy at Massachusetts General Hospital (MGH) Cancer Center, an Attending Physician in the Hematopoietic Cell Transplant and Cell Therapy division of Oncology at MGH. She is an Associate Member of the Broad Institute of Harvard and MIT, and an Associate Member of the Ragon Institute of MGH, MIT, and Harvard. Dr. Maus is internationally known for her work as a translational physician-scientist in the field of immunology, particularly as it relates to T-cell immunotherapies and cellular therapies in the treatment of cancer. Her laboratory focuses on the biology of human T cell activation, costimulationm, and memory, and on the application of human T cell therapies to human disease, including forward and reverse translation of engineered T cell therapies in early-phase clinical trials. She has authored over 100 papers indexed in pubmed and holds three NIH R01 grants and several Investigational New Drug appplications (INDs). Dr. Maus holds an undergraduate degree from Massachusetts Institute of Technology (MIT) and graduate degrees (M.D., Ph.D.) from University of Pennsylvania. Dr. Maus trained in internal medicine at University of Pennsylvania and in hematology and medical oncology at Memorial Sloan Kettering Cancer Center, is board-certified in these three disciplines, and practices medical oncology. She also serves on several scientific and clinical advisory boards for the biotechnology industry as well as external academic medical centers

Benjamin Kleinstiver, Ph.D.
Assistant Professor of Pathology
Massachusetts General Hospital

Ben Kleinstiver is a biochemist and genome editor with interests in protein engineering and genome editing technology development, and a long-term goal of translating these technologies into molecular medicines. He received his Ph.D in Biochemistry from the University of Western Ontario, and completed his postdoctoral studies at Massachusetts General Hospital (MGH) and Harvard Medical School. Within the Center for Genomic Medicine at MGH, the Kleinstiver laboratory develops scalable methods for molecular engineering to accelerate the development of CRISPR technologies. The major goals of the research in his laboratory are to address limitations of existing technologies, to develop new capabilities that will help solve important research questions at the forefront of the genome editing field, all with the hope of providing safe and effective treatments for patients.

Natalia Gomez-Ospina, M.D., Ph.D.
Assistant Professor of Pediatrics
Stanford University

Dr. Gomez-Ospina was born and raised in Medellin, Colombia. She began her undergraduate studies in petroleum engineering at the Universidad Nacional de Colombia before moving to Colorado. She double majored at the University of Colorado Boulder and graduated summa cum laude with bachelor’s degrees in Molecular Cellular and Developmental Biology as well as Biochemistry. She then completed her combined M.D., Ph.D. at Stanford Medical School, where her Ph.D. work focused on understanding novel functions of voltage-gated calcium channels. After completion of her dual degrees, Dr. Gomez-Ospina pursued residency in Medical Genetics at Stanford. Her post-doctoral research was in Pediatric Stem Cell transplantation, where she began to develop genome editing-based strategies in stem cells as therapies for genetic diseases. Dr. Gomez-Ospina is currently an Assistant Professor in the Department of Pediatrics. For her clinical practice, she sees patients with suspected genetic disorders. Despite, her young career, she has been the lead author in research studies in the New England Journal of Medicine, Cell, Nature Communications, and American Journal of Medical Genetics. Dr. Gomez-Ospina has championed the idea of commandeering the hematopoietic system to express proteins needed in other organs, including the brain. She established an adaptable platform for the treatment of lysosomal enzyme deficiencies and performed a first-of-its-kind preclinical study to support the clinical development of autologous transplantation of genome-edited cells to treat patients with Mucopolysaccharidosis type I. Beyond delivering lysosomal enzymes, this platform has implications for delivering many kinds of therapeutic proteins to the brain.

Annarita Miccio, Ph.D.
Lab Director
Imagine Institute

Dr. Miccio’s main interests are the transcriptional control of hematopoiesis, and the development of therapeutic approaches toβ-hemoglobinopathies. As a PhD student, she generated a lentiviral vector (LV) successfully used in an early clinical trial for β-thalassemia. As a post-doc and later as an assistant professor, she gained experience in the gene regulation during erythroid development and in evaluating the efficacy of gene therapy approaches for hematopoietic disorders. In 2014, she was appointed as a Lab Director at the Imagine Institute, where she pursued her studies on transcriptional regulation in normal and diseased stem cells, and their progeny. These basic research studies were instrumental in developing novel strategies for β-hemoglobinopathies. In particular, she optimized the design of a LV currently employed in a clinical trial for sickle cell disease and developed CRISPR/Cas9 strategies for β-hemoglobinopathies.

Jerry Mendell Award for Translational Science
Supported by Dr. Suku and Ann Nagendran

Named for the first person to study viral mediated gene therapy for muscular dystrophy in humans, and the principal investigator in the study that led to the FDA approval of Zolgensma to treat SMA, the Jerry Mendell Award for Translational Science recognizes the extensive work required to bring gene and cell therapies to clinical trial.

Jerry Mendell, M.D.
Professor of Pediatrics and Neurology
Nationwide Children’s Hospital

Jerry R.Mendell, M.D., is an attending neurologist at Nationwide Children’s Hospital, the Dwight E. Peters and Juanita R. Curran Endowed Chair in Pediatric Research atthe Abigail Wexner Research Institute at Nationwide Children’s Hospital andprofessor of Pediatrics and Neurology at Nationwide Children’s and The OhioState University. Dr. Mendell joined the Research Institute at Nationwide Children’s full time in 2004,recruited by Philip Johnson, the Head of the Center for Gene Therapy at that time. Shortly after, leadership of the Center changed and provided the opportunity to build and establish a program in Clinical Translation with emphasis on neuromuscular disease. At Nationwide Children’s, he holds academic positions as Professor of Pediatrics and Neurology and holds the Curran-Peters Chair of Pediatric Research.He is a clinician scientist with decades of experience. A career emphasis began with training in neuromuscular disease at the National Institutes of Health, directing laboratory projects that could be carried to the bedside. He was recruited to Columbus to direct the Neuromuscular Center at Ohio State. His life’s work has emphasized clinical translation. He has published more than 390 articles with a focus on neuromuscular disease and authored books on muscle disease, peripheral nerve disorders, and most recently, gene therapy for muscle disease. He was the principal investigator on the prednisone clinical trial in DMD in 1989 that has profoundly influenced clinical care. His study on newborn screening for DMD established incidence of disease at 1:5000, now universally accepted. Studies on myoblast transfer showed the limitations of methodology. His studies using eteplirsen for exon skipping in DMD (Exondys 51®) was the first antisense oligonucleotide approved by FDA for clinical use. This opened the door for further studies targeting other exons for DMD treatment. He was the PI on the first gene therapy trial for LGMD and his most recent trial in LGMD2E (R4) demonstrated unequivocal efficacy. For DMD gene therapy, his current micro-dys trials show promise including phase 1 (published in 2020) and phase 2 (DBRCT, n=41) now moving to phase 3. For SMA type 1, the most common fatal genetic disease of infancy, his clinical trial provided the path for first FDA approval for systemic gene delivery and also led to newborn screening for SMA resulting in very early of infants rescuing neurons prior to degeneration.

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