DNA Vectors
Unique and innovative non-viral DNA vectors of VTvaf17 and GDDT1.8NAS series
Introduction:
Since 2015, an international team of scientists, spearheaded by our company, has dedicated extensive intellectual resources and cutting-edge research efforts to this project, ultimately leading to the development of a groundbreaking universal platform solution — non-viral DNA vectors series VTvaf17 and GDDT1.8NAS — for creating advanced genetic tools in the rapidly evolving fields of biomedical and genetic technologies. These DNA vectors incorporate the unique RNA-out regulatory element from the Tn10 transposon, thus enabling antibiotic-free positive selection, and offering the following key advantages:
Maximum Safety
The absence of antibiotic resistance genes and viral genome sequences in our DNA vectors, in accordance with EMA and FDA recommendations, ensures the highest safety. This distinct combination in a non-viral DNA vectors makes our solution one-of-a-kind globally.
Superior Performance
By incorporating advanced delivery systems into our drug, we achieve therapeutically significant concentrations of target proteins.
Multiply Therapeutic Targets
Creation of a unique composition of genes empowers our drugs to simul-taneously target multiple therapeutic pathways, achieving a synergistic effect.
Nature-like Mechanism
The use of non-modified native genes ensures seamless integration with na-tural biological processes, minimizing the risk of adverse reactions.
Precision Expression
By integrating cell-specific and inflammation-activated promoters, our drugs achieve precise and effective expression of genes in target cells while minimizing undesirable side effects.
Technological Excellence
Implementation of high-tech manufacturing techniques optimizes production processes, achieving exceptional efficiency and significant cost reductions.
Strong Patent Protection
The intellectual property associated with this project is protected by more than 30 patents across various countries worldwide, highlighting the unique and innovative nature of the product.
Which delivery system is better? The "battle" between proponents of viraland non-viral vectors is less a confrontation and more an evolution of two approaches that address their unique challenges. The answer to this question is clear - each tool is suitable for its purpose. The flexibility and safety of non-viral vectors are ideal where targeted delivery, prolonged but limited expression period of the target protein, and minimal risks are essential.
Type | Viral vectors (lentivirus) | Viral vectors (retrovirus) | "Sleeping Beauty" non-viral system (transposon/transposase) | Existing vector plasmids | mRNA | Non-viral Gene therapy VTvaf17 and GDTT1.8NAS series vectors |
|---|---|---|---|---|---|---|
Plasmid part size (for plasmids) | - | - | >3 000 bp | >3 000 bp | >3 000 bp | 2 591 – 3 265 bp |
Antibiotic resistance gene (for plasmids) | - | - | Yes | Yes | - | No |
Selective agent (for plasmids) | - | - | Antibiotic | Antibiotic | - | Sucrose |
Type of selection marker (for plasmids) | - | - | Protein | Protein | - | RNA |
Tissue-specific transgene expression | No | No | Yes/No | Yes/No | No | Yes/No |
Preclinical / Сlinical trials currently underway worldwide | Yes | Yes | Yes | Yes | Yes | Yes |
Approved by regulators for use as a medicinal product | Yes | Yes | Yes | Yes | Yes | Clinical trials are in process |
Approved by regulators for use in CAR-T therapy | Yes | Yes | Clinical trials are in process | n/a | n/a | n/a |
Features of hardware equipment for transfection in CAR-T therapy | - | - | Electroporator | Electroporator | Electroporator | Electroporator |
Integration of a transgene into the genome | Yes | Yes | Yes | No | No | No |
Risk of alternative spontaneous oncological transformation | Moderate | Moderate | Moderate | Moderate | Absent | Absent |
Integral safety | Moderate | Moderate | Moderate | Moderate | Moderate | Highest |
Target product yield (for plasmids) | - | - | <10 mg/litre | 10 - 20 mg/litre | <10 mg/litre | >200 mg/litre |
Production scale-up | Yes | Yes | Limited | Limited | Yes | Yes |
Safety for the environment | Potentially hazardous | Potentially hazardous | Limited hazard level | Limited hazard level | Safe: Rapid degradation caused by exonucleases | Safe: Capable of replication only in a specific strain |
Production costs | Very expensive | Very expensive | Moderate | Moderate | Very expensive | Cheap |
Logistical aspects (low temperature shipping) | Expensive | Expensive | Cheap | Cheap | Expensive | Cheap |
Integral level of production and logistics costs | High | High | Low | Low | High | Minimal |
Patient affordability | Limited | Limited | Moderate | n/a | Moderate | High |
The Vector Development
Our Non-Viral DNA Vector: VTvaf17
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Patent Protection
A dedicated patent has been filed to safeguard VTvaf17’s intellectual property.
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Proven Safety
Toxicology tests confirm a favorable safety profile with minimal side-effect risk.
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Global Compliance
Strains have been deposited in an international repository under the Budapest Treaty of 1977.
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Universal Applicability
VTvaf17 can carry and deliver virtually any gene combinations, making it highly versatile for therapeutic development.
Strains used for the Vector Development:
The Escherichia coli SCS 110-AF strain serves as the basis for VTvaf17. This strain carries the target human gene(s) while excluding antibiotic resistance genes, in full compliance with stringent regulatory standards for medicinal product safety.
Patents
RU 2 678 756, International Application Number: РСТ/RU2018/000191, WO2019/039962, USA Application Number: 16636713; Title of Invention: “Gene therapy DNAvector VTvaf17, production method, strain Escherichia coli SCS110-AF, production method, strain Escherichia coli SCS110-AF/ VTvaf17, bearing gene therapy DNA-vector VTvaf17, production method”. Date of invention: 25.08.2017.
More information about patents:
https://patentscope.wipo.int/search/en/detail.jsf?docId=WO2019039962
Additional Gene Therapy Vectors
Alongside VTvaf17, our group of companies has developed and patented a range of non-viral DNA vectors, all free from viral genomes and antibiotic resistance genes.
These vectors adhere to the same safety-centric principles and can be utilized for a variety of therapeutic and research applications:
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VTvaf17-Cas9
Enables multiple genome-editing methods in human and animal cells.
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VTvaf17-Act1-Cas9
Developed for diverse genome-editing approaches in plant cells.
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GDTT1.8NAS12 (2,591 bp)
A platform solution closely related to VTvaf17, offering similar benefits in safety and efficacy.
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GDTT1.8NAS1 through GDTT1.8NAS11 (11 distinct vectors)
Designed for tissue-specific expression of target genes in muscle, skin, vascular endothelium, bone tissue, pulmonary tissue (bronchi and alveoli), nerve tissue, kidney podocytes, blood (hematopoietic cells, lymphocytes, or macrophages), and pancreatic beta cells.
Links to Publications of Patents of other our vectors:
Licensing Service:
We offer licensing of our proprietary vector technology to external partners. For more information on how to license our vector, please contact us here.
For more information,
see our presentation: download here
