DNAVEC Corporation selected three most important technologies out of the numerous leading-edge technologies developed by the predecessor company DNAVEC Research, Inc. in its nine years of research. Our technologies have extended their applications in gene therapies for intractable diseases; gene vaccines for chronic diseases, cancer and infectious diseases; therapeutic antibodies; protein production; and gene function analysis.
Three Core Technologies of DNAVEC
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Sendai Virus vector
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Simian Immunodeficiency Virus vector
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Selective Amplifier Gene
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| Concept |
- Cytoplasmic RNA vector.
- Realization of “Cytoplasmic gene therapy” and “Cytoplasmic gene vaccine”.
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- A non-pathogenic lentivirus vector, which does not recombine with HIV (AIDS virus). Achieves persistent therapeutic gene expression suitable for the treatment of chronic diseases.
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- Artificial chimeric genes that improve the performance of existing vectors and increase the gene transfer efficiency to hematopoietic stem cells. Basic technology for gene therapies targeting blood cells.
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| Design |
- Multitude of designs possible, amenable to various needs of bedside and lab bench.
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- Vector design capable of achieving both efficient transduction and safety.
- Utilizes advantages of both lentivirus vector and Sendai virus vector.
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- Fusion of the gene for a receptor that dimerizes by extracellular stimulus and the gene for a protein that generates an intracellular growth stimulation signal.
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| Manufacture |
- GMP-compliant vectors for clinical use are supplied by an overseas contract manufacturer.
- Conventional grade vectors are manufactured by inside facility. Vectors for collaborative research use are also available.
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- Recombinant vectors for collaborative research use are manufactured by inside facility.
- Manufacturing of GMP-compliant vectors for clinical use by an overseas contract manufacturer is under development.
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- Vectors carrying SAG are manufactured by inside facility. Vectors for collaborative research use are also available.
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Development
Stage |
- Licensed out: Gene therapeutic medicine for Critical Limb Ischemia (CLI), AIDS gene vaccine.
- Under joint development with major pharmaceutical companies: gene vaccines for Alzheimer's and other diseases.
- Searching for candidates for clinical development through a network of collaborating clinicians, both domestic and overseas.
- Developing multifaceted bio-products jointly with Medical & Biological Laboratories Co., Ltd. (MBL).
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- Acknowledged by the Cystic Fibrosis Gene Therapy Consortium of UK as “the most potent gene therapeutic medicine in the world.”
- Jointly developing gene therapies for Retinitis Pigmentosa and Glaucoma with Kyushu University.
- Research and development underway in collaboration with clinicians domestic and overseas.
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- Proven effectiveness in mice and monkeys.
- World’s top level in vivo gene transfer efficiency in blood cells without bone marrow ablation.
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Patents
and
Know-hows
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- World-leading patent families and know-hows.
- Master patents granted in US, China, Japan, and Europe.
- Established technologies for various component processes as well as mass manufacture of vectors.
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- World-leading patent families and know-hows.
- Patent has been applied for technologies to make the vector capable of infecting respiratory tract.
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- World-leading patent families and know-hows.
- Patents have been applied for SAG technologies as well as technologies of nondestructive introduction of genes to bone marrow.
- Patent for SAG granted in China; under examination in US.
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| Usage |
- Broad range of applications in gene therapy.
- Broad range of applications in gene vaccine.
- Production of therapeutic antibodies.
- Production of proteins.
- Functional analysis of genes and proteins.
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- Gene therapy for respiratory diseases.
- Gene therapy for neurological disorders.
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- Therapies for blood disorders such as gene therapy for hematopoietic stem cells.
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