| |
Sendai Virus (SeV) Vectors:
 |
New pseudo-typing Technology for Vectors (Simian Immunodeficiency Virus[SIV]) Vector):
 |
Selective Amplifier Genes (SAG):  |
| Design |
*Diversely designed vectors (Solvable to various clinical and manufacturer requirements)
| *Vector design safe and with good gene transfer properties to airway (
Union
of the merits of lentiviral vector and a SeV vector) |
*Developed fundamental technology for use in gene therapy(Artificial chimeric genes that improve performance and gene transfers to hematopoietic stem cells of existing vectors) |
| Manufacturing system |
*Built up a supply system of cGMP-responsive? vectors for clinical use through a tie-up with an overseas CRO.
* Inner manufacturing facility is working to supply vectors for use in cooperative researches and requests from outside companies in collaborations etc. |
*Inner manufacturing facility is working to supply the recombinant vectors for use in cooperative researches
*Received the assessment “The most influential gene therapy company with such vector in the world” from the UK Cystic Fibrosis Consortium |
*Inner manufacturing facility is working to supply recombinant vectors for use in cooperative researches |
| Stage of development |
*A group of candidates for development have appeared from the network.
*The studies for many-sided practical use of bio-products are advancing. |
*Built up a system of domestic and overseas clinical doctors for cooperative developments |
*Certified efficacy in animal experiments to use with retrovirus vectors (mice and monkeys)
*Highest level of gene transfer without bone marrow destruction in the world |
| Patent & know-how |
*Accumulated patents and know-how lead the world.
*Accumulated patents and know-how lead the world
*Fundamental patents certifiedhave veen approved in
USA
and
China
*
A system with diverse elemental technologies for vector mass-production t has been established |
*Accumulated patents and know-how lead the world
*Applied for a patent for technology giving a vector the ability to infect an airway penetrating well through mucus membrane |
*Accumulated patents and know-how lead the world
*Applied for patents of the gene and the technology for gene transfers without bone marrow destruction
*Patent of the gene is accepted in
China
and under inspection in
USA
|
| Target use |
Gene therapy for diverse diseases
*Gene vaccines |
*Gene therapy of respiratory disorders
*Gene therapy of nervous system disorders etc.(SIV vector). |
*Therapy of blood disorders such as hematopoietic stem cell gene therapy |
Usefulness of the Sendai Virus (SeV) Vectors
Of the 3 core technologies the development of
Sendai
Virus (SeV) Vectors has progressed the most rapidly. At first, SeV vector showed its high performance gene transfers and expression in vitro and in animal experiments in various types of cells and tissues including the respiratory system and the nervous system. For the former, it has not been possible to transfer any genes with existing vectors. and therefore, These excellence of the vectors make them very powerful tools for use in gene therapy and other bio-technologies such as function -analysis of genes and proteins, .
One more important point is that the SeV vector is a vector from the new concept, “Cytoplasmic RNA vectors”. Because SeV does not enter the cellular nucleus and express its gene in the cytoplasm, there is theoretically no risk of damaging of chromosomes or their functions in the nucleus and therefore it has an advantage, that is, higher safety than that of existing vectors from view point of genetic toxicity.
The superiority of SeV vector has been highly evaluated in universities and medical organizations, and a number of collaborative developments are in progress between our company and domestic and overseas medicine-related organizations, including pre-clinical studies in relation to gene therapy.