A-ju Science develops first-in-class therapeutics that directly eliminate mutant mitochondria — the root cause of mitochondrial disease.
Our technologyContactOur compounds recognize a specific mitochondrial DNA mutation, localize rapidly to mitochondria, and induce mitophagy of mutant mitochondria without damaging nuclear DNA, restoring respiratory function and energy production. No adverse effects have been observed in animal models including non-human primates.
The most common mitochondrial disease. Only symptomatic treatments exist today; no drug addresses the causative m.3243A>G mutation. This is where we start.
Leigh syndrome, mitochondrial myopathy, LHON and many others are caused by mtDNA mutations. Because our compounds are designed to the mutation, the same approach extends across these diseases.
The MELAS mutation is also found in about 1% of diabetes and some hearing loss, and mitochondrial alterations are seen in cancer cells. Our technology may extend to these conditions.
7 major markets = US, UK, DE, FR, IT, ES, JP. Patient numbers are approximate, based on public reports (IMARC, DelveInsight) and literature (GeneReviews et al.). Our compounds are in research and development; efficacy and safety will be evaluated in future clinical trials.
| Program | Indication | Stage | IND (planned) |
|---|---|---|---|
| CCC-018-TPP | Mitochondrial disease (MELAS) | Nonclinical | Dec 2027 |
| CCCh-360-TPP & more | Ultra-rare mitochondrial diseases | Development research | Oct 2030 |
| CCCh-560-TPP | Advanced cancer | Development research | Oct 2029 |
| CCC-021-TPP | Metastatic lung cancer | Basic research | — |
| CCC-018-TPP | Diabetes, hearing loss | Basic research | — |
| CCCh-560-TPP | Age-related and lifestyle diseases | Basic research | — |
| Company | A-ju Science Co., Ltd.（永寿サイエンス株式会社） |
|---|---|
| Founded | November 1, 2024 |
| President & CEO | Yasuo Nagase |
| Director & Chief Scientific Officer | Hiroki Nagase, M.D., Ph.D. (Visiting Professor, Juntendo University) |
| Headquarters | Motomachi Wellness Park West Bldg. 3F #302, 1-1-19 Hongo, Bunkyo-ku, Tokyo 113-0033, Japan |
| Business | Development of therapeutics for mitochondrial disease (designated intractable disease) and related disorders (refractory cancer, diabetes, etc.) |
| Supported by | AMED (Practical Research Project for Rare/Intractable Diseases); Tokyo Metropolitan Government Blockbuster Creation Program; Gemseki Investment Co., Ltd. |
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