Prion Disease Treatment and Diagnosing

NCEs for the treatment/diagnostics of Creutzfeldt-Jakob disease (CJD).

Advantages

  • Demonstration data has been obtained to improve the survival time of prion-infected mice
  • Companion diagnostics (CoDx) candidate probes have been obtained

Background and Technology

This research group has been exploring the search for small molecules that can be used as therapeutic drugs for prion disease [1]. Researchers have been selecting compounds from both in vitro and in vivo screening with known compound libraries (about 690,000 molecules). Among them, two compounds, named NPR-130 and NPR-162, that bind to normal prions (PrPC) and suppress the expression of abnormal prions (PrPSc) were found. The relevance of these compounds to prion disease has not been reported so far.
Researchers are also developing synthetic new effective small molecules for prion disease, based on structure-activity relationships from the skeleton and functional group information of these two compounds.
In addition, another group at the same university is now developing novel imaging probes for amyloidosis, and some of the probes show significant signals in PrPSc positive regions.

[1] Ishibashi, D., et al. Brain 2019 Apr 1;142(4):1035-1050. doi: 10.1093/brain/awz016.

Data

The two known compounds (NPR-130, NPR-162) were confirmed to be effective in improving the survival time of mice infected with prions (unpublished data).

  • Similar experiments are in progress for the novel chemical entities.
  • Diagnosing probes show significant signals in PrPSc positive regions.

Patents

PCT/JP2019/036887 (Treatment drugs), published in Japanese as WO2020/059841.
PCT/JP2019/008168 (Diagnostics imaging probes), published in Japanese as WO2019/168170.

Researchers

  • Treatment drug: Daisuke Ishibashi (Associate Professor, Nagasaki University, Japan), et al.
  • Imaging probe: Takeshi Fuchigami (Associate Professor, Nagasaki University, Japan), et al.

Expectations

Tech Manage Corp. is now looking for a pharmaceutical/biotech company to collaborate with the researchers and develop this technology further under licensing of the patent(s) described above.

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