Scientific Committee Descriptions
ASGCT's Scientific Committees are responsible for developing scientific symposia sessions presented at the Annual Meeting based around the following subject matter
Cancer Gene and Cell Therapy
The Cancer Gene and Cell Therapy committee is responsible for the development of ASGCT programming with a focus on the following topic areas: next generation cell and gene therapy approaches in cancer treatment, tumor vaccination strategies utilizing gene-based vaccines, novel oncolytic viruses and platforms for oncolytic immunotherapy, current updates on clinical successes and challenges in the field of cell and gene therapy in cancer treatment, and the newest advances in cell and gene therapy approaches in pre-clinical therapy evaluation.
Cardiovascular Gene and Cell Therapy
The Cardiovascular Gene & Cell Therapy Committee includes diverse investigators from academia and industry and is focused on the development of novel cell- and gene-based therapies for diseases of the cardiovascular system. Examples of scientific topics of interest include the development of viral and non-viral methods of therapeutic cargo to the heart and vessels, cellular and gene-based strategies to achieve cardiac regeneration and revascularization, and advanced biological therapeutics for diseases including atherosclerosis, heart failure, peripheral vascular disease, pulmonary hypertension, cardiac arrhythmias, and heritable cardiomyopathies. The main mission of the committee is to profile important scientific advances occurring in this rapidly advancing field, including through the development of cardiovascular-related programming for the ASGCT Annual Meeting.
Gene and Cell Therapy of Genetic and Metabolic Diseases
This committee is broadly focused on identifying and disseminating the most contemporary therapeutic strategies to treat genetic and metabolic disorders. Inborn errors of metabolism including PKU, urea cycle defects, lysosomal storage disorders, organic acidemias, glycogen storage disorders, diabetes, and hemophilia are some of the conditions that have traditionally been under our purview. Because genetic and metabolic disorders serve as important proof of concept targets for traditional and novel treatment approaches, the committee has remained agnostic of methodologies and focused on assessing submitted work based on results and impact, fully embracing viral and non-viral platforms, genome editing, and stem cell therapies.
Genome Editing
The Genome Editing Committee designs annual ASGCT programming related to the development and application of genome editing technologies. This includes the optimization of safe and effective DNA and RNA editors, the continued discovery and innovation of naturally occurring enzymes that can be turned into useful tools, the development of precision editors capable of mutating individual bases or generating larger sequence edits such as exon or gene-sized insertions or replacements, methods to minimize unwanted off-target or genotoxic events, barriers facing broader adoption of these technologies, and the translation of these technologies into genetic medicines. Early genome editing clinical trials are already providing exciting results, and continued innovation in areas that span basic research, technology development, and pre-clinical studies will further allow these tools to help patients and their families.
Hematologic and Immunologic Gene and Cell Therapy
The Hematologic and Immunologic Gene and Cell Therapy scientific committee is focused on all aspects of gene and cell therapy for benign hematologic disorders as well as disorders of the innate and adaptive immune system. The scientific portfolio covers ex vivo modified hematopoietic stem cells (HSCs) with viral and non-viral platforms such as lentiviral and adenoviral vectors, RNA, gene addition and editing with genome editing systems. The scientific portfolio also includes marrow conditioning toxicity, and the fate and function of therapeutically modified autologous or allogeneic cells used to treat primary immunodeficiencies and benign hematological disorders.
Immune Responses to Gene and Cell Therapy
The committee is composed of a group of experts in the study of immune responses to cell and gene therapy. The committee reviews the latest research on immunological barriers and on immunology related toxicities to cell and gene therapy, selecting topics to be showcased at the Annual meetings and other workshops organized by the ASGCT. Topics include both preclinical and clinical studies on innate and adaptive immune responses to viral and non-viral gene transfer vectors and their encoded transgenes, immune response in cell therapy, ex vivo gene therapy, and gene editing. The scope of this committee also includes new treatments of potential strategies to mitigate immune-related adverse events.
Musculo-Skeletal Gene and Cell Therapy
The Musculo-Skeletal Gene and Cell Therapy committee creates programs for scientific symposia at the Annual Meeting to showcase research in the field of gene and cell therapy that advances our understanding or treatment of diseases affecting bone and skeletal muscles. The committee focuses on highlighting progress in the fields of vector biology, cell and gene delivery to musculo-skeletal tissues, and systemic immune responses to cell and gene therapies. The committee also emphasizes preclinical and clinical translation of advanced molecular therapies for musculo-skeletal diseases.
Nanoagents and Synthetic Formulations
The goal of the Nanoagents and Synthetic Formulations Committee is to provide a comprehensive overview of major trends, developments, and advances in the field of nonviral formulation of gene therapies. The committee covers topics in exosomes, liposomes, targeted assembly, and novel protein and peptide engineering approaches. In addition, the committee’s purview includes novel developments in the areas of nanoparticle self-assembly from natural materials such as proteins, RNA, and carbohydrates.
Neurologic and Ophthalmic Gene and Cell Therapy
The Neurologic and Ophthalmic Gene and Cell Therapy Committee educates members by promoting the dissemination of innovative scientific research, novel therapeutic approaches, and breakthrough technologies to advance the discovery and development of cell and gene therapies for neurological and ocular diseases. The Committee is responsible for the organization of the ASGCT Annual Meeting Symposia on gene and cell therapy for neurologic and ophthalmic disorders by developing the Symposia content and identifying and selecting the speakers. The Committee may also propose the organization of other educational events (Workshops, Insight Series) that will be scheduled during the year.
Nonviral Therapeutic Delivery
The Nonviral Therapeutic Delivery Committee is responsible for the development of ASGCT programming on several areas of focus. Gene therapies delivered by physical methods (electroporation, hydrodynamic delivery, ultrasound, laser) as well as newer delivery technologies such as exosomes are included. Novel RNA- and DNA-based expression vectors including transposons and integrases, minicircles, genome editors, and artificial chromosomes are a second focus. Innovative targeting methods for organ, cellular and intracellular nonviral therapeutic delivery are in the scope of the committee. Therapeutic application is emphasized using novel nonviral gene delivery methods and vectors for the treatment of genetic diseases and cancers. Finally, chimeric therapies combining viral, nonviral and cell-based methods are included.
Oligonucleotide and RNAi Therapeutics
The Oligonucleotide and RNAi Therapeutics committee develops ASGCT programming related to the latest developments and advances in the oligonucleotide therapeutics area from discovery to clinical applications, including new chemistry, novel targeted delivery approaches, and therapeutic applications. The committee focus also includes, but is not limited to, challenges in oligonucleotide therapeutics including toxicity and clinical development as well as emerging scientific areas that are amenable or relevant to oligonucleotide therapeutic modalities including ASOs, RNAi, aptamers, circular RNAs, RNA editing, mRNAs, and tRNAs.
Prenatal Gene and Cell Therapy
Responsible for planning an Annual Meeting symposium and other on-line seminars, highlighting the advancements of cell and gene therapy and other related approaches to treat genetic diseases prior to birth. The committee will address matters pertaining to basic and translational science investigating age-appropriate cell therapies, developing age-safe gene delivery systems, and gaining a full understanding of the risks and benefits of employing current therapeutics to the fetal population. It is the goal of this committee to promote research that can fill critical gaps in knowledge in fetal therapies and create paths to bring these therapies to fruition.
Respiratory and GI Tract Gene and Cell Therapy
The Respiratory & GI Tract Gene & Cell Therapy Committee is responsible for the creation of the Annual Meeting Symposium on the topic of gene and cell therapies for respiratory and gastrointestinal diseases. Understanding how molecular, biochemical, and cellular processes contribute functionally to disease phenotypes is important for the development of these therapies and for the selection of appropriate patient populations who will most likely respond to them. There is a pressing need for the development of novel gene and cell therapeutic technologies that are suitable for the efficient and targeted delivery of genetic payloads to the lung and GI tract. Further, translationally relevant in vitro assays and non-clinical in vivo models are instrumental for proof-of-concept studies and ultimately for the selection of therapeutics for clinical development. The goal of the symposium is to provide a platform for open information sharing across diverse disciplines for the benefit of patients in need.
Stem Cell
The Stem Cell committee is responsible for the creation of an Annual Meeting symposium and other programs with a focus on stem cell biology and stem cell applications that are relevant for cell and gene therapies. The committee’s scope includes the following areas: using allogeneic or autologous pluripotent stem cells as a target for ex vivo gene and cell therapies, using allogeneic or autologous adult stem cells as targets for ex vivo and in vivo gene and cell therapies (for example, hematopoietic stem cells), and applying basic knowledge from stem cell and regenerative biology to target non-stem cells but to achieve durable treatment effects.
Translational Science
The Translational Science Committee covers the process of turning gene and cell therapy observations in the laboratory, clinic, and community into even better interventions that improve the health of patients. We focus on the two most critical junction points in the discovery and development of new gene or cell therapies: translating initial scientific discoveries from academia to industry and advancing preclinical programs to the clinic and through subsequent registration. Each stage builds upon and informs the other. We support and recommend topics covering all stages of this progression, including the process of applying discoveries generated in preclinical laboratory research to experimental studies in animals and human participants, as well as technology innovations and regulatory strategies for investigators to extend often limited clinical observations from early trials into approved drugs.
Viral Gene Transfer Vectors
The Viral Gene Transfer Vectors Committee develops educational sessions across therapeutic areas and stages of development. The main goal of the committee is to highlight the recent advances and challenges of using viral vectors to deliver therapeutic genetic material. The committee aims to design symposia that provide researchers with insights into leveraging different viral vector modalities (DNA & RNA viruses, replicative and non-replicative, commonly used & newly discovered). Areas of focus include practical considerations for academic and industry professionals, including emerging technologies, manufacturing/product quality, and safety and efficacy during non-clinical and clinical development.