Nicotinamide mononucleotide derivatives and their use in treatment and prevention of erythrocyte disorders

By using nicotinamide mononucleotide derivative compounds to increase the NAD+ level in the red blood cells of patients with sickle cell disease, the toxicity and inapplicability problems of existing treatments are solved, and safe and effective treatment and prevention of sickle cell disease is achieved.

CN120699073APending Publication Date: 2025-09-26NUVAMID SA
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Patent Information

Application Number
CN202510776750.6
Authority / Receiving Office
CN · China
Patent Type
Applications(China)
Current Assignee / Owner
Priority Date
2020-12-18
Filing Date
2021-12-17
Publication Date
2025-09-26

AI Technical Summary

Technical Problem

Existing treatments for sickle cell disease (SCD) are limited and suffer from toxicity and inapplicability issues, necessitating the development of safer and more effective therapeutic and preventive treatments.

Method used

Nicotinamide mononucleotide derivative compounds of formula (I) are used to improve the pathophysiology of sickle cell disease and reduce acute pain crises and other complications by increasing the NAD+ redox ratio in red blood cells.

Benefits of technology

It improves the tolerance and clinical effects of sickle cell disease patients, reduces the incidence of acute complications, and provides a safe and effective treatment and prevention method.

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Abstract

The present invention relates to nicotinamide mononucleotide derivatives of formula (I) for use in the treatment and / or prevention of blood disorders, in particular sickle cell disease. The invention also relates to a pharmaceutical composition comprising the compound of formula (I) for use in the treatment and / or prevention of erythrocyte disorders, in particular sickle cell disease.
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