Expanded Opportunities for Methods and Clinical Development

Abstract

Now well into its third year, Molecular Therapy — Methods & Clinical Development (MTM) is firmly established as one of our Society's successful open-access journals. Its first special issue has just been published, providing in-depth coverage of recent advances in manufacturing of gene therapy vectors. At the core of the issue, which was edited by two leading experts in the manufacturing of gene therapy products, are six comprehensive reviews on the methodology of the production, purification, formulation, and quality control of major gene transfer vectors, with emphasis on process development for their clinical use. These molecular medicines include adeno-associated viral, lentiviral, and baculoviral vectors, oncolytic viruses, nanoparticles, and aptamers. Two articles each were written by authors from institutions in North America, Europe, and Asia. In addition, the issue contains numerous original research articles that reflect the cutting edge of vector production. Another special issue, on the development of cell therapy products, is in the pipeline for early 2017. Beyond these special issues, the journal has regularly been publishing more than 60 articles per year, although this number is likely to substantially increase now that MTM is indexed in all major databases, including PubMed, and on track to receive its first Impact Factor. To cope with the increasing demand to publish in MTM, several geographically diverse deputy editors have been appointed. Furthermore, timely review articles in methodology and clinical development of gene and cell therapies, of other closely related molecular therapies, of genetic vaccine development, and of immunotherapies, as well as insightful commentaries are now being published in MTM on a regular basis. Each month, one research article is highlighted at the journal's home page in the form of a “Featured Image” with legend, and a link to an archive of these images is provided. Finally, the array of different types of articles that can be published within MTM has been expanded to include two additional categories. First, recognizing the ever-growing number of clinical trials that utilize gene and/or cell therapies and the potential importance of observations in clinical medicine that are made in individual patients, we decided to offer publication of case reports (presubmission inquiry is encouraged for this type of manuscript, however). Second, the journal will publish two or three invited protocols per year to provide the community with thorough technical instructions on laboratory methods that are widely used in the field of gene and cell therapy. Currently in preparation are protocols that detail specific methods for the purification of adeno-associated viral vectors and the in vivo delivery of nonviral DNA vectors. In a closer look at the research articles published in MTM over the past two years, major themes that emerge include vector production and purification, vector development and optimization, studies in small- and large-animal models (such as models of disease or nonhuman primates), development of data that are critical on path to clinical trial (including safety data), data from clinical studies, immune-response studies, vaccine development, and physical and surgical methods to monitor or deliver gene therapy. Other examples include papers on various cell therapies (including induced pluripotent stem cells), genome editing, targeting of vectors to specific tissues, and regulated gene expression. MTM provides an opportunity for investigators in industry to publish on process development and other technical aspects of manufacturing, which are often underrepresented in other journals. Likewise, it is the ambition of the editors to further expand the range of topics covered in MTM to include more cell therapies, tissue engineering, monoclonal antibody therapies, and development of engineered protein therapeutics, among others. However, none of these themes are unique to MTM. On the contrary, many research subjects fall within the scope of more than one journal within the Molecular Therapy family, sometimes even within all four current journals. Whereas Molecular Therapy has the widest impact and strives to publish papers that are of the broadest interest, MTM and the other open-access siblings have a more specialized focus (methods and clinical development, oligonucleotides and nonviral vectors, and oncolytic/cancer gene therapy). Nevertheless, there is also overlap in their scope, thus providing authors with choices for submission. Therefore, the editorial teams of these journals closely collaborate and coordinate, attempting to find the best home for scientifically sound manuscripts, while also considering the authors' requests and preferences. Clearly, Molecular Therapy — Methods & Clinical Development has been a strong addition to the gene and cell therapy journals that is strongly embraced by the scientists of our field, and we will continue to expand the opportunities to publish and make contributions to these areas.