The 2014 ASGCT Awards
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Setting the Standard for Distinguished Achievement: The 2014 ASGCT Awards

Summary by Terence R. Flotte, MD

The ASGCT annually recognizes outstanding individuals and organizations whose accomplishments exemplify the society’s overarching values and goals. At the 17th Annual Meeting of the society in Washington, DC this May, the ASGCT once again presented an Outstanding Achievement Award, a Distinguished Service Award and four Outstanding New Investigator Awards. Also at this meeting, it was announced that beginning in 2015, the society’s Distinguished Service Award will be named in memory of Dr. Sonia Skarlatos, a tireless champion of gene therapy and the inaugural co-recipient of the award in 2013.

The winner of the 2014 Outstanding Achievement Award was Dr. Luigi Naldini Dr. Naldini was recognized for a long series of accomplishments in the field, beginning with his landmark development of the first HIV-based vectors, which was followed by a series of critical improvements in the lentiviral vector (LV) technology which has enjoyed widespread success in applications in hematopoietic stem cells. Dr. Naldini has also pioneered methods for cellular targeting of these vectors using miRNA detargeting and other designs.  He has also identified a subset of monocytes whose transduction is pivotal for the correction of certain neurologic disorders, such as metachromatic leukodystrophy, in which the clinical outcomes have been quite remarkable.

Mr. Edward and Mrs. Barbara Netter were recognized as the 2014 Distinguished Service Award recipients for their role in founding the Alliance for Cancer Gene Therapy, or ACGT. In 2001, Mr. and Mrs. Netter founded the Alliance to support the development of cell and gene therapy to treat cancer. Over the past decade the Alliance, under the direction of Mr. and Mrs. Netter, has raised and granted research awards of over $23 million and provided funding to 39 investigators in 28 academic medical institutions aided by a Scientific Advisory Council comprised of recognized leaders in the field of cancer cell and gene therapy, who implemented a rigorous peer review process for grant evaluation and contributed their services voluntarily. Unfortunately, Mr. Netter passed away before receiving the award, but Mrs. Netter accepted it at the meeting.

The Outstanding New Investigator awards were presented to four individuals whose early achievements in the field already demonstrate remarkable distinction: Dr. Brian Brown from Mt. Sinai School of Medicine, Dr. Charles Gersbach from Duke University, Dr. Scott Harper from Ohio State University and Nationwide Children’s Hospital, and Dr. Daniel Powell from the University of Pennsylvania. 

Dr. Brown’s contributions include new approaches for cell-type specific targeting of vector expression and elucidation of the innate and adaptive immune responses to oligonucleotides. Dr. Gersbach has developed new methods to genome editing for correction of genetic diseases, for directing stem cell differentiation and for tissue regeneration. 

Dr. Harper has been integral in a number of milestone discoveries in the muscular dystrophy field, including the generation of micro-dystrophins and the development of RNAi-based therapies for neurodegenerative diseases, as well as making contributions to understanding the basic pathobiology of two types of muscular dystrophy, FSHD and LGMD1A. Dr. Daniel Powell has made important contributions to T cell-based cancer therapy. These include elucidation of the role of CD27 in human T cell memory, development of a novel dual signaling approach to CAR-T cell therapy to limit toxicity, and identification of CD137 as a marker of naturally occurring tumor-reactive T cells.

The ASGCT award recipients are nominated annually by the ASGCT Advisory Council, subject to final approval by the Board of Directors.  Information regarding past recipients and procedures for nominating future award candidates may be found on the ASGCT website.

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