Composition for treating hemoglobinopathy and use thereof
By using a combination of nuclease variants and a BCL11A gene-targeting gene for gene editing, the lack of effective treatments for hemoglobinopathies in existing technologies has been addressed, resulting in a significant increase in fetal hemoglobin levels and providing a potential method for the treatment and prevention of hemoglobinopathies.
WO2026067861A1 Publication Date: 2026-04-02YOLTECH THERAPEUTICS CO LTD
3 Cites 0 Cited by
Patent Information
- Application Number
- PCT/CN2025/126070
- Authority / Receiving Office
- WO · WO
- Patent Type
- Applications
- Current Assignee / Owner
- Priority Date
- 2024-09-30
- Filing Date
- 2025-09-30
- Publication Date
- 2026-04-02
AI Technical Summary
Technical Problem
Currently, there is a lack of safe and effective treatments for hemoglobinopathies, particularly therapeutic strategies that use CRISPR-Cas technology to regulate the BCL11A gene to increase γ-globin expression.
Method used
A nuclease variant and a composition targeting the BCL11A gene are provided, comprising a nuclease, guide RNA, polynucleotide, vector, ribonucleoprotein, and lipid nanoparticles, for gene editing of the BCL11A gene to enhance γ-globin expression.
Benefits of technology
Gene editing significantly increased fetal hemoglobin levels in cells, providing a potential treatment option for hemoglobinopathies, with potential for both treatment and prevention.
✦ Generated by Eureka AI based on patent content.
Abstract
The present disclosure provides a composition for treating hemoglobinopathy (e.g. sickle cell anemia, hemophilia, β-thalassemia, etc.). The present composition comprises a nuclease for modifying the BCL11A gene and a CRISPR-Cas system comprising a guide RNA. Also provided is a method for treatment by administering, in a subject with a hemoglobinopathy-related disease, a system that targets the BCL11A gene or a nucleic acid that encodes such a system.
Need to check novelty before this filing date? Find Prior Art