Methods of identifying patients likely to benefit from treatment with a telomerase inhibitor

IL279623A1Pending Publication Date: 2026-07-01GERON CORP
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Patent Information

Application Number
IL279623
Authority / Receiving Office
IL · IL
Patent Type
Applications
Current Assignee / Owner
Priority Date
2018-11-29
Filing Date
2019-07-29
Publication Date
2026-07-01
Estimated Expiration
2039-07-29

AI Technical Summary

Technical Problem

Current treatments for myelofibrosis, particularly those lacking mutations in JAK2, CALR, and MPL genes or having high-molecular risk mutations in ASXL1, EZH2, SRSF2, and IDH1/2, do not effectively address the disease progression and survival outcomes for patients, as they often lead to leukemic transformation and shortened overall survival.

Method used

Identifying patients with triple negative status or high-molecular risk based on genetic mutations using DNA samples from bone marrow or peripheral blood, and administering a telomerase inhibitor like imetelstat to target and inhibit telomerase activity, thereby potentially slowing disease progression.

Benefits of technology

The approach effectively identifies patients likely to benefit from telomerase inhibitor treatment, leading to improved spleen volume reduction, symptom response, and overall survival, particularly in patients with triple negative or high-molecular risk profiles, offering a new therapeutic option for myelofibrosis management.

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Abstract

This disclosure provides methods of identifying or selecting a patient most likely to benefit from treatment with a telomerase inhibitor, such as e.g. imetelstat, by testing a patient for: a lack of a mutation in each of JAK2, CALR, and MPL; and / or a high-molecular risk (HMR), based on the presence of a mutation in at least one of the following genes: ASXL1, EZH2, SRSF2, and IDH1 / 2. The patient may be suffering from myelofibrosis. The disclosure also provides methods of treating myelofibrosis, which include identifying such patients.
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