PAPD5 inhibitors and methods of use thereof

The PAPD5 inhibitor modulates TERC and telomerase activities, and solves the problem of difficult treatment of diseases caused by short telomeres in the prior art, and achieves the recovery of telomeres length and the improvement of related diseases.

CN119798159APending Publication Date: 2025-04-11CHILDRENS MEDICAL CENT CORP
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Patent Information

Application Number
CN202411839234.5
Authority / Receiving Office
CN · China
Patent Type
Applications(China)
Current Assignee / Owner
Priority Date
2019-12-23
Filing Date
2020-04-23
Publication Date
2025-04-11

AI Technical Summary

Technical Problem

The prior art is difficult to effectively treat diseases caused by short telomeres, such as aplastic anemia, pulmonary fibrosis, cirrhosis and bone marrow failure, and there is a lack of effective medical treatments.

Method used

Provides PAPD5 inhibitors to restore TERC and telomerase function in cells of PARN mutant patients by regulating TERC levels and telomerase activity.

Benefits of technology

By using PAPD5 inhibitors, TERC levels and telomerase activity can be restored, thereby prolonging telomere length and improving the clinical manifestations of related diseases.

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Abstract

Provided herein are compounds that are PAPD5 inhibitors and are useful in the treatment of a variety of conditions, such as cancer, telomere disease, and aging-related disorders, as well as other degenerative disorders.
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