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Molecular Therapy Oncology

Formerly Molecular Therapy Oncolytics

Molecular Therapy Oncology is an international, online-only, open-access journal focusing on the development and testing of cancer therapies in pre-clinical and clinical settings. While providing a unique forum for work in the burgeoning fields of biologic cancer therapies including viral, bacterial, cellular, and gene therapy, the journal will consider all top-quality, cutting-edge research that employs innovative approaches to cancer treatment. The scope also encompasses the identification and validation of predictive biomarkers of response. Because the journal is focused on cancer therapies, most accepted papers will have translational relevance.

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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

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Molecular Therapy Nucleic Acids

Molecular Therapy Nucleic Acids is an international, open-access journal publishing top-quality basic, translational, and clinical research in the broad fields of nucleic-acid-based therapeutics to treat and/or correct genetic and acquired disease. As an official journal of the American Society of Gene & Cell Therapy, Molecular Therapy Nucleic Acids builds upon the success of Molecular Therapy in publishing important peer-reviewed research, cutting-edge reviews, and commentaries targeted to advances in gene- and oligonucleotide-based therapies.

Subject areas include but are not limited to: development of therapeutics based on nucleic acids and their derivatives, vector development and design for the delivery of RNA-based therapeutics, applications of gene-modifying agents including triplex-forming oligonucleotides and enzymes such as Zn finger nucleases, pre-clinical target validation, safety/efficacy studies, and clinical trials.

To accommodate the rapid growth of Molecular Therapy Nucleic Acids, starting in 2022, articles will be submitted and published in one of six subsections covering key topics.

Subsections include:

Subsection	Topics covered	Guidelines
Bioinformatics	AI NGS mathematical modeling phylogenetic oligo arrays	Manuscripts presenting new software, algorithms, databases, web servers, or web services must clearly demonstrate the validity of these tools in proof-of-principle experiments or analyses; authors should also describe how these tools improve upon current approaches. New software, algorithms, databases, web servers, or web services must be open access, with a direct link to the host site or database provided within the main text of the manuscript. Novel software and source code must be provided in a version-controlled repository, such as GitHub or GitLab.
Delivery strategies	chemistry ligands lipid nanoparticles localized delivery applications polymeric nanoparticles systemic delivery targeted delivery viral vectors	Studies can cover the broad field of targeting and clinical applications. Examples include different routes of delivery (e.g., localized, systemic, subcutaneous, intrathecal), targeted vs. non-targeted delivery applications, lipid nanoparticles (LNPs), polymeric nanoparticles (PNPs), viral vectors, and chemistries. For targeted delivery approaches, there should be a clear demonstration of specificity with the inclusion of at least one negative control or control cell line. For nanoparticle-based approaches, the composition of the particle must be provided. Studies must include comprehensive data on the functional efficacy of the proposed strategy. Efficacy studies should be performed in at least two in vitro systems. Validation of the delivery approach in vivo is preferred but not required.
Non-coding RNAs	circRNAs lncRNAs miRNAs siRNAs regulatory guidelines	Studies should pioneer innovative research into non-coding RNAs involved in disease, potential therapeutic interventions targeting these disease elements, or using novel non-coding RNAs for treatment. Validation of the non-coding RNA should be robust and highly reproducible with compelling data supporting the molecular effects in the disease, which may include overexpression and silencing studies, binding interactions with molecular targets, deletion of the target site, or functional rescue, with data supporting significant downstream effects. Relevant controls should be included pertaining to the specific non-coding RNA under investigation, e.g., miRNA studies should have scrambled or non-target controls for experiments. Therapeutic interventions targeting non-coding RNAs should be validated in relevant disease models. The functional relevance of the non-coding RNA in disease must be established. Experimental data demonstrating that the perturbation of the non-coding RNA can augment the disease state should be present or at least a clear rationale described for therapeutic invention for a novel non-coding RNA. Top candidate non-coding RNAs identified with bioinformatic approaches must be extensively validated experimentally. Purely computational or bioinformatic approaches for non-coding RNAs should be submitted to the Bioinformatics section. Studies pertaining to the mechanisms of action and applications of synthetic oligonucleotides should be submitted to other sections.
Oligonucleotides: diagnostics and biosensors	aptamer-based biosensors molecular beacons nanostructured biosensors radiolabeled oligonucleotides rolling circle amplification-based biosensors	Studies should present novel research in the broad field of molecular diagnostics and biosensors involving nucleic acids and their application in medicine. Studies involving aptamers must provide their sequences and equilibrium dissociation constants. Aptamer binding must be validated by at least two methods with appropriate controls. The target(s) of the aptamers should be identified. The specificity, sensitivity, accuracy, and working/dynamic range of assays must be clearly demonstrated and calculated.
Oligonucleotides: therapies and applications	clinical studies CMC development discovery preclinical assessment regulatory RNAs	The efficacy of the proposed strategy should be reproducibly demonstrated in at least two disease-specific cellular models in vitro. Confirmation in at least one animal model in vivo is preferred but not required. Studies should include positive/benchmark controls as well as negative controls. Matched, scrambled oligonucleotides with similar chemistry should be included in studies characterizing the impact of new chemistries on the efficacy and safety of oligonucleotides. Studies on new oligonucleotide therapeutic modalities should include safety assessments (including the potential immunotoxicity of such strategies) at least in vitro. Safety studies can include immune cell viability testing and cytokine profiling, e.g., high-density peripheral blood mononuclear cell cultures for human-related reagents. Studies based on immunomodulatory oligonucleotides should address the broad immune activity of oligonucleotide-based reagents, both positive and negative regulation of immune responses, with clear relevance to therapy of human diseases. Early-stage clinical studies and studies of new oligonucleotide therapeutic modalities should include an overall safety assessment of these reagents.
RNA/DNA editing	ADAR CRISPR HDR NHEJ TALEN ZFN	Studies that integrate both biochemical and functional approaches are preferred. Studies that show the productive effects of editing must be confirmed in at least two independent experimental models. Approaches involving epigenetic mechanisms must address the specificity of the mechanism(s) involved. The specificity (potential off-target events) and efficiency of the RNA/DNA editing approach must be clearly demonstrated. For RNA editing, transcriptome-wide and bystander events should be assessed. For DNA editing, genome-wide off-target analysis should be performed.

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