Congratulations to the 2022 Award Recipients!

Grant Holley, PhD - April 11, 2022

The American Society of Gene & Cell Therapy is proud to announce the winners of the Outstanding Achievement Award, the Outstanding New Investigator Award, the Sonia Skarlatos Public Service Award, and the Jerry Mendell Award for Translational Science.

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.

Donald Kohn, MD

Professor of microbiology, immunology and molecular genetics, and pediatrics (hematology/oncology)
University of California Los Angeles

Dr. Kohn is a professor and a member of the Broad Stem Cell Research Center, the Jonsson Comprehensive Cancer Center and the UCLA Children’s Discovery and Innovation Institute. The research in his lab focuses on the development of new methods to treat genetic diseases of blood cells by gene modification of hematopoietic stem cells. Dr. Kohn’s group was the first to perform a clinical trial with gene transfer to umbilical cord blood CD34+ cells for a genetic disorder (ADA-SCID in 1993) and the first in the U.S. to initiate clinical trials of gene therapy for pediatric HIV/AIDS using bone marrow stem cells. A Past President of ASGCT (2003-2004), Dr. Kohn also served as president of the Clinical Immunology Society (2013-2014), and as a member (2010-15) and chair (2013-15) of the Recombinant DNA Advisory Committee (RAC) for the NIH Office of Biotechnology. 

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.

Lindsey George, MD

Director of clinical in vivo gene therapy
Children's Hospital of Philadelphia

Dr. George is a physician-scientist with clinical expertise in disorders of hemostasis and thrombosis with a particular interest in hemophilia and hemophilia gene therapy. Her basic science laboratory studies the molecular basis of coagulation that in diminished or excess functional states leads to disorders of hemostasis and thrombosis, respectively. The current focus of the George lab is to merge mechanistic studies aimed at understanding the regulation of factor VIII cofactor function with translational efforts in hemophilia A gene therapy. Her group is additionally interested in understanding the mechanistic basis of questions that have emerged from current hemophilia gene therapy clinical trials as well as general studies of adeno-associated viral vectors (AAV). Dr. George was previously the lead clinical principal investigator of multiple early phase hemophilia A and B adeno-associated virus-mediated gene addition trials.

Matthew Hirsch, PhD

Associate professor in the department of ophthalmology
University of North Carolina

Dr. Hirsch's lab investigates the use of AAV vectors for therapeutic gene delivery and gene editing towards the treatment of several genetic diseases. Hirsch and his colleagues developed a novel technique (OAGR) and characterized existing approaches that exploit host DNA repair pathways for intracellular large transgene reconstruction. During this work, they identified host DNA repair proteins and pathways necessary for episomal genetic engineering with the understanding that enhancements at the basic science level will aid in the generation of a safer and more efficient clinical reagent. Towards disease therapy, the team evaluated different AAV large gene delivery contexts in disease models of dysferlinopathy and hemophilia A. Hirsch's lab has also focused on emerging technologies using DNA endonucleases to genetically engineer human and mouse chromosomes, and on the the AAV vector transduction of different types of stem cells.

Morgan Maeder, PhD

Director of payload sciences
Chroma Medicine

When Dr. Maeder entered the field as a research technician in 2006 in Dr. Keith Joung's lab, her research spanned many technological platforms, from zinc-finger nucleases to TALENs to CRISPR. At the time, genome editing was tedious and expensive, but Dr. Maeder published 31 papers in field-leading journals. She demonstrated the first ZFN-induced correction of a disease-causing mutation in human-induced pluripotent stem (iPS) cells, as well as the first use of the CRISPR/Cas9 system for targeted gene activation in human cells. Dr. Maeder then joined Editas as its first scientist, where she led the development of EDIT-101 to treat Leber congenital amaurosis 10 (LCA10) from inception through IND-enabling studies. The BRILLIANCE Phase 1/2 clinical trial of EDIT-101 was the first in vivo use of a CRISPR-based medicine. Dr. Maeder went on to serve as a consultant to Third Rock Ventures before joining Chroma Medicine in 2020. At Chroma, Dr. Maeder established lab operations, onboarded technology, and designed and executed the company's initial scientific plan before it launched in November 2021. She now leads teams in the payload science group focused on technology development and optimization of the Chroma platform.

Christopher Peterson, PhD

Staff scientist, clinical research division
Fred Hutchinson Cancer Research Center

Dr. Peterson has an extensive backgroun in eukaryotic cell culture, gene expression, and in vivo disease models. For the last 10 years, he has worked in the lab of Dr. Hans-Peter Kiem, which he joined with the specific interest to pursue research in HIV. When he became a research assistant professor in 2016, Dr. Peterson developed an independent research program in HIV focusing on net-generation CAR T-cell technology for HIV. He has applied two primary strategies in this work: refining techniques for gene editing, most recently utilizing the CRISPR/Cas9 platform, and employing CAR T cells. He has incorporated CRISPR/Cas9 gene editing approaches both to enhance CAR T-cell function, and to protect CD4+ T cells, the primary targets of HIV-1, against infection.The ultimate goal of his research is to provide a curative therapy that is safer and more affordable than lifelong ART and can be applied anywhere in the world. In 2020 he published as senior author a seminal paper in Blood showing that co-administration of antigen presenting cells can improve anti-HIV CAR T-cell function and persistence. This was demonstrated in a highly clinically relevant NHP model and was the first time that anti-HIV CAR T cells were able to persist at the levels shown in this paper with control of SHIV in some animals. This work was featured as part of the ASGCT Presidential Symposium in 2020.

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.

P.J. Brooks, PhD

Acting director, office of rare diseases research, NCATS
National Institutes of Health

Dr. Brooks joined NCATS’ office of rare diseases research (ORDR) as a program director in August 2018 and was appointed deputy director in September 2021. Prior to his tenure in the ORDR, he worked in NCATS’ division of clinical innovation, where he was the lead program director for the clinical and translational science awards (CTSA) program collaborative innovation awards, designed to fund projects that will result in novel and creative approaches to overcoming roadblocks in translational science. Dr. Brooks' research on rare genetic diseases gave him the opportunity to meet with patients and their families, which had a powerful impact on his view of the importance of translational science in rare diseases. He believes that nucleic acids have clear therapeutic potential for single-gene disorders, and he developed funding opportunities (PAR-20-098 and PAR-20-109) to support innovative approaches to delivering genome editors to target cell types. is interested in accelerating clinical trials in rare diseases by moving beyond “one disease at a time” approaches. Examples include the development of therapeutics that target shared molecular mechanisms underlying multiple rare diseases and the implementation of platform vector gene therapy trials via the NCATS Collaborative Rare Disease Platform Vector Gene Therapy Trial cooperative agreement.

Jerry Mendell Award for Translational Science

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. Supported by a generous grant from Dr. Suku and Ann Nagendran.

Kathy High, MD

President of therapeutics

Dr. High joined AskBio in January 2021 as president of therapeutics and a member of the AskBio board of directors. In this role, she's responsible for driving the strategic direction and execution of the company’s preclinical and clinical programs. Most recently, she was a visiting professor at Rockefeller University. Previously, she served as president, head of research and development, and a member of the board of directors at Spark Therapeutics, where she directed the development and regulatory approval of Luxturna®, the first gene therapy for genetic disease to obtain regulatory approval in both the U.S. and Europe. She was a longtime member of the faculty at the University of Pennsylvania and medical staff at The Children’s Hospital of Philadelphia, where she was also an investigator of the Howard Hughes Medical Institute. She served a five-year term on the FDA advisory committee on cell, tissue and gene therapies and is a Past President of ASGCT.

Dr. Holley is the director of science and education at ASGCT.