Gene and Cell Therapies in Expansion Mode: ASGCT 2016
Authors: Cynthia E Dunbar
Journal: Molecular Therapy (2016)

There was palpable excitement at the 2016 American Society of Gene and Cell Therapy (ASGCT) annual meeting in Washington, DC, this past May. Attendance was up 20% from the highly successful 2015 meeting in New Orleans, and has nearly doubled since the 2013 meeting. The number of exhibitors and sponsors jumped dramatically, and included a number of “big pharma” firms for the first time. Noticeably well-dressed investors and business analysts prowled the halls and huddled with investigators in the Marriott hotel lobby. Unfortunately, capacity was insufficient at the Marriott venue—booked four years earlier—and the scientific and education sessions were jam-packed because of the high quality of the programming and the intensity of interest in the new information being conveyed. Presentations spanned from cutting-edge genome editing basic science through encouraging results from multiple mature clinical trials. The day before the meeting, our workshop on commercialization was fully subscribed. The Society's membership has also grown 33% since the beginning of 2014. The success of our 2016 meeting mirrors the overall renewed academic and commercial interest in gene and cell therapies, stimulated by recent gene therapy treatment successes, including those employing chimeric antigen receptor–engineered T cells for hematological malignancies, adeno-associated virus gene addition for hemophilia B and for inherited retinal degeneration, and lentiviral gene addition for a number of inherited immunodeficiencies and degenerative neurological disorders. Clinical trials of gene targeting via zinc-finger nucleases have begun to show encouraging results, and translational science resulting from the discovery of CRISPR/Cas9 technologies in 2012 has exploded, with myriad clinical applications of this highly flexible and efficient gene editing approach in development by companies and academic investigators around the world. The first clinical trial of induced pluripotent stem cell regenerative therapies has commenced in Japan. Given this encouraging climate, ASGCT leadership determined that it was important to hold a strategic planning retreat in order to carefully consider how to best serve the Society's mission and our membership. The leadership pondered the recent trend of presenting abstracts describing initial clinical gene therapy trial results at major medical society meetings––for instance, those sponsored by the American Society of Clinical Oncology or the American Society of Hematology––instead of at our annual meeting, and whether this phenomenon could be or should be reversed. Many argued that this phenomenon was evidence of the maturation of our field into the mainstream, with relevance to physicians more generally. These discussions led to a focus on the larger picture regarding the best role for the Society, our annual meeting, and our journals in the further development of gene and cell therapy. We concluded that the Society could best serve as a nidus for furthering the translation of basic science and new technology development into novel clinical therapies. Our meetings and journals should continue to function to bring together academics, regulators, and biotechnology and pharmaceutical partners to further the development of safe and effective gene and cell therapies. To facilitate these goals in the short and medium term, the strategic planning process prioritized improving the ability of the Society to serve as a source of reliable and timely information regarding gene and cell therapies, via upgrading of our website. In addition, communication between constituencies should be furthered by a digital presence, spearheaded by members of our newly created Trainee Committee. These initiatives are ongoing, and we hope to unveil both a revamped website and a more robust digital presence during the next year. We also prioritized further outreach and collaboration with patient advocacy groups and foundations, to provide reliable and unbiased education to these consumers regarding the current and future status of gene and cell therapies. We also brainstormed the need to communicate advances in gene and cell therapies to broader constituencies, and have presented “Gene Therapy 101” programs at basic science meetings, such as the American Society of Immunology's recent convention in Seattle. We are developing programs to present at clinical meetings and creating webinars to connect with both physician and patient groups. We have taken our exceedingly popular clinical trials course “on the road” to standing-room-only at the annual meeting of the International Society for Stem Cell Research, and partnered with the European Gene and Cell Therapy Society to present a similar course at last year's meeting in Helsinki. Finally, maturation of the field brings new and complex challenges regarding clinical trial funding, regulatory processes, and approaches to reimbursement for very expensive but potentially curative therapies. The board constituted a new Government Relations Committee to interact more actively with the National Institutes of Health, the US Food and Drug Administration, and the legislative branch around these issues. As a relatively small society, we realize the benefits of joining with other groups to further our common interests. We are active members of the Alliance for Regenerative Medicine, we are partnering with the Foundation for Accreditation of Cellular Therapy to focus on the process of combining genetic modification with cell therapies in GMP facilities, and we were invited to join the National Academy of Medicine's Forum on Regenerative Medicine. It is an exciting time for ASGCT to help bring gene and cell therapies to patients in the context of our increasing knowledge of the molecular basis of human disease, access to incredible new technologies for designing and delivering molecular treatments, and commitment from both the public and private sector to support initiatives in our fields.