ASGCT 2021: Time to celebrate and expand
Authors: Roland W. Herzog, Robert Frederickson
Journal: Molecular Therapy (2021)

The annual meeting of the American Society of Gene and Cell Therapy (ASGCT), which took place in May 2021, set yet another record with nearly 7,000 registrants attending the virtual presentations. On the heels of successful gene therapies for genetic diseases, cellular immunotherapy and virotherapy for cancer, and the approval of multiple gene therapy drugs, comes a Nobel Prize for gene editing and highly efficacious vaccination against the infectious disease coronavirus disease 2019 (COVID-19) using messenger RNA (mRNA) delivery and viral vectors. The concept of gene therapy first emerged in the early 1970s, gained traction in the 1980s with the progress in molecular cloning, but only became feasible in the 1990s with the development of suitable gene transfer vectors. Recent progress in nanoparticle technologies has enabled the expansion of the concept of delivery of therapeutic nucleic acids to various RNA molecules such as mRNA. Molecular therapy now encompasses a much broader range of experimental therapeutics, which includes technologies such as viral and non-viral gene delivery, gene editing, siRNA for gene silencing, mRNA delivery, aptamers, gene modified cells, targeting of non-coding RNA, oncolytic viruses, and others. Applications are only limited by our imagination but already include genetic disease, cancer, acquired diseases, and, of course, vaccination. In many ways, the 2021 ASGCT meeting has been transformative. In part because of the pandemic that has impacted all of our lives, the general public has reached a greater understanding of the value of molecular therapies. Who would have thought that “mRNA” would become a frequent term heard in the mainstream media? In Germany, for instance, the scientists of Biontech are already highly decorated folk heroes. Prior to the pandemic, mRNA-based vaccines were unproven, and other genetic vaccines were not yet widely used. Now, vaccine development and protection from infectious disease are prominent components of the ASGCT meeting. At some point almost forgotten, cancer gene therapy is now a flourishing field with impressive clinical successes, and its promise was celebrated by ASGCT with the Outstanding Achievement Awards, given to two pioneers of immunotherapy based on chimeric antigen receptor (CAR) T cells, Drs. Carl June and Michel Sadelain, and with Robert Martuza’s lecture on oncolytic virotherapy at the presidential symposium. It is widely accepted that the precise changes in the human genome that can be accomplished by gene editing represent the future approach of choice in gene therapy. Jennifer Doudna, one of last year’s winners of the Nobel Prize in Chemistry for her discovery of the molecular machinery that enables gene editing, was asked during her fireside chat about more scientists joining in on this rapidly evolving technology. Her answer was clear: “There has never been a better time.” Fittingly, the Molecular Therapy journal family will present a special issue on gene editing later this year. Reflecting the growth of the annual meeting and the maturity of the society and the gene therapy field, the scope of the meeting has expanded beyond presentation of data and scientific achievements. The program now includes various aspects of career development (including alternative careers in academia, industry, and other entities), racial justice in the gene therapy field (a symposium organized by the Ethics & Diversity and Inclusion Committees), patient access to gene and cell therapy, questions of fairness and ethical aspects of drug development, as well as other areas related to regulatory aspects and science policy. These themes will be followed up during an ASGCT policy summit in September and are also covered on a regular basis in the form of commentaries and reviews in Molecular Therapy and its sibling journals. Indeed, these are heady times for the Molecular Therapy journal family as well. Submissions to the journals continue to have an exponential trajectory, and the number of papers published over past 12 months in the journal family nearly reached 1,000, roughly double the number just 2 years ago. All of the growth is due to the rapid expansion of the open access sibling journals—Molecular Therapy - Nucleic Acids, Molecular Therapy - Methods & Clinical Development, and Molecular Therapy - Oncolytics—which reflect the continued push of science publishing, in general, toward this platform. This impressive growth has allowed us to fulfil our mission of publishing high quality research for society members and non-members alike, while at the same time being more selective at Molecular Therapy itself, with a view to increasing its growing impact even further. Papers submitted to Molecular Therapy itself are being vetted carefully for general interest and potential impact and we have expanded the scope to cover all aspects pertinent to the development of molecular therapies, including identification of new therapeutic and diagnostic targets and related questions of disease mechanisms, biomarker development, novel molecular technologies, and approaches to the treatment of human disease.