Molecular Therapy: Methods & Clinical Development

Molecular Therapy – Methods & Clinical Development is an international, open-access journal publishing important peer-reviewed methods and procedures as well as translational advances in the broad array of fields under the Molecular Therapy umbrella. Focus areas are advances in cell and gene therapy and how they are translated and applied in the clinical setting. Novel methods of gene delivery into immune cells and hematopoietic and other stem cells using integrating, non- integrating vectors, non-viral vectors, and gene editing methods are featured, manufacturing of such products under Good Manufacturing Practice (GMP) conditions, including regulatory science and possible commercialization of these novel products are additional focus areas.

Topics of particular interest within the journal's scope include:

• Early-phase clinical trials that investigate a novel biological product with a novel mechanism of action

• Systematic reviews and meta-analyses of gene and cell therapies in preclinical and clinical development stages and in different indications

• Translational and clinical scale-up and Good Manufacturing practice procedures and process development

• Post-marketing observational studies to investigate long-term safety and efficacy of gene and cell therapies

• Clinical trial protocols of gene and cell therapies, combined medical devices, and other biological products

• Studies focusing on regulatory science and analyzing the regulatory approval process

• Methods for cell isolation, engineering, culture, expansion, and transplantation

• Cell processing, storage, and banking for therapeutic application

• Quality Control (QC) assay development

• IND-enabled preclinical studies comprising preclinical pharmacology, toxicology, metabolism, and bio distribution

• Pharmacology/toxicology studies of new and next-generation vectors

• Methods for gene and cell vector delivery

• Methods for targeted genome editing and engineering

• Methods and technology development for cell reprogramming and directed differentiation of pluripotent cells

• Development of biomaterials and nanoparticles for applications in gene and cell therapy and regenerative medicine

• Computational and bioinformatic methods for analysis, modeling, or visualization of biological data

Visit the Cell Press website for more information about Molecular Therapy – Methods & Clinical Development: https://www.cell.com/molecular-therapy-family/home.