Profile Information

Affiliation
CEO and Representative Director (Chief of Department), Headquarters, R&D center, Triplex Therapeutics Inc.
University of California, Santa Barbara
Degree
Doctor of Philosophy (PhD) (Lifesciences)(The University of Tokyo)

Researcher number
40625430
ORCID ID
 https://orcid.org/0009-0000-7888-6639
J-GLOBAL ID
200901086399535085
researchmap Member ID
5000090784

External link

Born in Japan. While PhD student at the University of Tokyo, he developed a polyamide compound that binds to mitochondrial DNA, which can increase the amount of normal mitochondrial DNA in cells, and based on the substance patent, he is engaged in research and development of therapeutic drugs for rare genetic diseases including mitochondrial diseases. In 2016, he launched a bio-venture company with friends, and is currently working hard to bring therapeutics to the market. Currently, he has also succeeded in developing a therapeutic molecule that induces "programmed ribosomal frameshift" (PRF), which corrects the misalignment of gene reading frames (ORF) caused by genetic mutations such as deletions, and aims to create a new drug that will enable patients with Duchenne muscular dystrophy to walk independently, which was not possible with conventional drugs.

 

In 2021, the development stage of a novel therapeutic molecule for Duchenne muscular dystrophy (DMD), which is caused by a mutation in the dystrophin gene, reached animal trials, and the company plans to raise funds for the animal trials through crowdfunding. The company started calling for funding through crowdfunding sites on February 3, 2021. As a result, the project was successfully completed on March 25, with more than 170% of the target amount raised.

 

Based on the support raised through this crowdfunding campaign, we have decided to conduct a small-scale animal study using muscular dystrophin mice in mid-August 2021. The purpose of this animal study is to confirm the expression of dystrophin protein in mouse muscle tissue by systemic administration of a novel therapeutic molecule to muscular dystrophin mice using a drug delivery system (DDS) that is already being used in clinical trials. This initial small-scale animal study on muscular dystrophin mice has just been successfully completed at the end of July 2022. Currently, we are working with experts to conduct a detailed analysis of the treatment effects based on the obtained image data of mouse muscle tissue and video data of muscular dystrophin behavior.

 

We have managed to successfully complete an animal study using Duchenne muscular dystrophy mice at the end of July this year, 2022. We are now planning to proceed with the analysis of the image data and other data with experts. I hope to be able to exchange opinions with those who wish to have individual discussions with me while showing them the video data of the mice and the image data of the muscle tissues that I have obtained. I would also like to send research reports to those who have requested them as soon as I receive comments from the experts.

 

However, I would be happy to discuss how to proceed with future muscular dystrophy drug research projects up to the clinical trial stage. I would appreciate it if you would follow and pay attention to the information that will be released in the future. Thank you in advance for your continued support. 

Academist Inc.'s crowdfunding site is at the URL below:

https://academist-cf.com/projects/210?lang=en (you can also find it by searching for "academist + muscular dystrophy" on google, etc.).

 

I have also begun disseminating information on social networking sites and Twitter:

Facebook: https://www.facebook.com/profile.php?id=100030928695425

LinkedIn: https://www.linkedin.com/in/takamitsu-yano-ph-d/

Twitter: https://twitter.com/takamitsu_yano (only account. (Only one account, no backstage accounts).

 

In addition. The Japan Muscular Dystrophy Association (JMDA) has introduced our activities for the animal study of a therapeutic molecule for Duchenne muscular dystrophy to JMDA. Thank you very much:

https://www.jmda.or.jp/members-only/medical/medical-info-2021021001/

JMDA's list of muscular dystrophy research trends (with links to us): https://www.jmda.or.jp/muscular-dystrophy/research-trend/


Papers

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Social Activities

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Media Coverage

  1