Molecular Therapy: At the Cutting Edge of Methodology and Clinical Development

Abstract

Molecular Therapy—Methods & Clinical Development (MTM) was launched in January 2014 as the second online-only open-access sibling journal to Molecular Therapy (MT). The enthusiasm with which the gene and cell therapy community has greeted this expanded network of journals and the unique opportunities that arise from the open-access format have been demonstrated by the stream of high-quality papers that have already been published by MTM and its predecessor sibling, Molecular Therapy—Nucleic Acids (MTNA). Going forward, we will further strengthen this effort with research articles, reviews, and commentaries at the cutting edge of methodology and clinical development of gene and cell therapy approaches. MTM fulfills a key role by specifically featuring emerging technologies in molecular medicine as well as translational research and clinical development. Innovative technologies have always been a major driver of the gene and cell therapy fields. Some combine gene transfer and cell therapies, such as use of gene-modified hematopoietic stem cells to treat genetic disease or the breakthrough in cancer immunotherapy that was made possible by the use of T cells engineered to express chimeric antigen receptors. The gene therapy community rapidly develops novel methodologies and quickly adapts emerging technologies from other fields. Genome editing and pluripotent stem cells are obvious examples, and surely other exciting technologies will emerge in the near and distant future. In this spirit, MTM will feature articles at the forefront of these developments. Gene therapy has enjoyed numerous clinical successes in recent years and is moving swiftly toward commercialization. These developments increase the demand for vector design, optimal and scalable methods for the production and purification of gene transfer vectors, biodistribution and toxicity studies, and clinical trial design. Consequently, MTM also welcomes articles in all these areas, including clinical protocols, trial outcomes, and case reports. These types of publications are critical to the field; by making the data freely available, they facilitate clinical development of vector systems. Similarly, as a result of successes with specialized cell therapies, academic centers are increasingly establishing facilities for the production of clinical-grade cell products, including gene-modified cells. Gene therapy laboratories are still heavily engaged in development of optimal disease models, and more recently in animal models that more accurately mimic gene transfer to humans, such as “humanized” mice and nonhuman primates. Hence, the journal will continue to feature novel laboratory and preclinical methods that are expected to drive clinical development of cell and gene therapy products. Open-access publishing has distinct advantages for authors. Methods and protocols can easily reach a large readership. Scientists at academic institutions with limited funds for journal subscriptions will nevertheless have access to these articles. Authors are more likely to reach not only fellow scientists but also physicians, patients, and those who hold corporate positions in management or research and development, or in the investment sector. We hope to attract not only a broad readership but also new authors from other fields who are developing new types of molecular therapies, such as novel chemistries for molecular conjugates or nucleic acid delivery, fusion proteins with prolonged half-lives, and monoclonal antibodies. At the same time, we expand the options for publication of methods that have traditionally been at the core of the gene therapy community, such as vector development. There is deliberate overlap between the scopes of the sibling journals, providing authors with choice of venue for their work. For example, a laboratory may have developed a novel method, based on a nonviral vector, that has shown efficacy in human cancer cells and thus has potential in cancer therapy. In addition to Molecular Therapy, this study would fit well into the more specialized subject areas of Molecular Therapy—Nucleic Acids, Molecular Therapy—Methods & Clinical Development, and Molecular Therapy—Oncolytics. The ultimate choice of venue will depend on the audience an author wishes to reach and the dialogue among the authors, the reviewers, and, of course, the editorial team. In addition, the functionality of our manuscript-tracking systems has been modified so as to facilitate transfer of both manuscripts and reviewer reports between the journals. Therefore, authors in the gene and cell therapy fields should view Molecular Therapy as a family of high-quality journals that provides them with exciting options for publication.