- Triplex Therapeutics Inc. Headquarters, R&D center CEO and Representative Director (Chief of Department)
- University of California, Santa Barbara
Doctor of Philosophy (PhD)(The University of Tokyo)Master of Medical Science (MMS)(University of Tsukuba)Bachelor of Science (BS)(Yamagata University)
- J-GLOBAL ID
- External link
Dr. Takamitsu Yano is a founder, CEO and a scientist of a biopharmaceutical company that specializes in the research and development of innovative gene drugs to treat rare neuromuscular diseases.
Beginning with his research on the control mechanism of replication of mutant mitochondrial DNA in human cells in his doctoral program at the University of Tokyo, he developed "ML1 polyamide", a therapeutic substance which has a unique mechanism of pharmacological action for mitochondrial disease, MELAS (Mitochondrial myopathy, Encephalopathy, Lactic acidosis, and Stroke-like episodes), through his own study. In 2016, taking advantage of the establishment of this domestic patent, he launched Triplex Therapeutics, a biotech company from the University of Tokyo.
ML1 polyamide does not require any special drug deliveries and has the characteristic permeability for cell membranes and mitochondrial bilayers as a single substance without specific modifications or carriers for its delivery. ML1 polyamide is possible to increase the copy number of normal mitochondrial DNA in human cells by permeating cell membranes and mitochondrial bilayers, and binding sequence-specifically to the target sequence of normal type double-stranded mitochondrial DNA. Previous experiments have shown that there is no difficulty on the membrane permeability of myoblasts, which are a kind of affected cells , and its efficacy on mitochondrial DNA copy number in the patient's cells has been confirmed.
Aiming to develop new drugs with a fundamental therapeutic effect on mitochondrial diseases, the patent for substance and pharmaceutical composition of ML1 polyamide has already been acquired in Japan, the United States, Canada, and six European countries.
Currently, Triplex Therapeutics is preparing for in-vivo level tests towards clinical trials of MELAS, a type of lethal mitochondrial disease, and new R & D program for an entirely new genetic drug that has never existed before overcoming the conventional drugs of "exon skipping" is ongoing for neuromuscular diseases such as Duchenne Muscular Dystrophy (DMD) caused by mutations in dystrophin gene.
These genetic diseases are the most frequent and fatal diseases of humanity, and we are continuing our earnest research for a complete recovery and total patients' reintegration to our society beyond the mere “life support” that was the limit of usual medicines.
11 2016 - Present
11 2015 - 3 2016
Pyrrole-imidazole polyamide, a synthetic DNA-binding compound, is effective at increasing levels of wild-type mtDNA in both cybrid cells and MELAS patient-derived fibroblast cells with the MELAS A3243G mutation by a selective promotion of wild-type replicationMITOCHONDRION 13(6) 924 - 925 11 2013 Peer-reviewed
Loss of mutant mitochondrial DNA harboring the MELAS A3243G mutation in human cybrid cells after cell-cell fusion with normal tissue-derived fibroblast cellsINTERNATIONAL JOURNAL OF MOLECULAR MEDICINE 25(1) 153 - 158 1 2010 Peer-reviewed
Successful preferential propagation of wild‐type mitochondrial DNA in human cells harboring the mt tRNALeu(UUR) A3243G mutation by a synthetic DNA‐binding compoundUnited Mitochondrial Disease Foundation, Mitochondrial Medicine 2011: Chicago 6 2011 Invited
Normal fibroblast nuclear transfer causes selective loss of mutant mitochondrial DNA harboring the A3243G mutation in human cybrid cellsThe 7th Annual Conference of the Japanese Society of Mitochondrial Research and Medicine (J-mit) 12 2007
Preferential loss of mitochondrial DNA harboring the pathogenic mutation tRNALeu(UUR) 3243 A->G in human somatic cell hybridsThe 5th Annual Conference of the Japanese Society of Mitochondrial Research and Medicine (J-mit) 12 2005