Introducing a New ASGCT Forum for Publication of Methodology and Clinical Development in Gene, Cell, and Oligonucleotide Therapies

Abstract

Though this be madness, yet there is method in’t. —William Shakespeare Methodology, technology, and clinical development studies form the core of the fields of cell and gene therapy. Whether focusing on vector engineering, cellular reprogramming, or strategies to efficiently and specifically deliver complex biologics to diseased tissues or cells, workers in the fields under the molecular therapy umbrella are pioneering the development of new ways to treat and cure disease. This constant focus on technology and methodology is fundamental to all stages of the process—from initial proof of concept in animal models through preclinical studies and early product development—because it ensures the ultimate success of the therapy. And, as has been discussed in these pages in numerous commentaries and editorials, the translational path that links the various stages of the development of novel therapies is fraught with methodological, procedural, and regulatory challenges. It is in this light that we are thrilled to announce this month the establishment of a new Methods section in Molecular Therapy's sibling journal, Molecular Therapy Nucleic Acids (MTNA). The section will focus specifically on methodology and technology development in gene-, cell-, and oligonucleotide-based therapy as well as on clinical development studies essential for safely moving these techniques into the clinic. Paradoxically, it was a journal focusing on methodology that we initially intended to launch two years ago as our first experiment with the online-only, open-access model on which MTNA is based. However, we were persuaded to launch MTNA instead, on the basis of strong interest and support from the oligonucleotide therapy community in the creation of such a title. The decision has paid off, as the journal has exceeded expectations with regard to both numbers and quality of submissions and published articles. As of the writing of this editorial, more than 100 papers have been submitted to the journal, roughly half of which were transferred from MT because the editors or reviewers felt that the paper would probably be of more specific interest to researchers in the fields of oligonucleotide therapy and gene modification. Molecular Therapy will continue to publish papers describing novel, high-impact methods and technology development when presented in the context of a therapeutic strategy being evaluated in a preclinical model or in patients. However, the new Methods section of MTNA will enable us to publish a greater volume of such studies and to capture more early-stage technology development in gene and cell therapy in general. It will also afford us the opportunity to report significant improvements to established research techniques in basic, translational, and clinical cell and gene therapy. We envisage several other areas falling within the purview of the new section, including vector development and production, translational scale-up and good manufacturing procedures, pharmacology and toxicology studies, quality control and assurance, computational and bioinformatics analyses, and regulatory issues and studies. Cellular reprogramming and engineering will also be featured in the section. Given the current strong interest in methods and technology to direct stem cell differentiation and modify cellular phenotypes, we encourage submissions in this area as well. Also falling within the purview of the section are methods for cell isolation and processing, culture and expansion, and storage and banking. Because MTNA is an online-only, open-access journal, papers published in the Methods section will be readily available to readers worldwide. Furthermore, these articles will be fully searchable in PubMed Central and are expected to be indexed in PubMed itself, hopefully by the end of the year. In 2013, following this interim launch period, the Methods section of MTNA will be spun out into a brand new journal focused on methodology and process development. More information will be forthcoming as we continue to develop this title.