Gene therapy approach for treating disorders associated with TNNT2
The gene therapy approach enhances TNNT2 protein incorporation in cardiac cells by combining a TNNT2 transgene with a nucleic acid to reduce endogenous TNNT2 expression, effectively addressing cardiomyopathies caused by TNNT2 mutations.
WO2025226841A1 Publication Date: 2025-10-30KATE THERAPEUTICS INC
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Patent Information
- Application Number
- PCT/US2025/026015
- Authority / Receiving Office
- WO · WO
- Patent Type
- Applications
- Current Assignee / Owner
- Priority Date
- 2024-04-24
- Filing Date
- 2025-04-23
- Publication Date
- 2025-10-30
AI Technical Summary
Technical Problem
Current treatments for cardiomyopathies, particularly those caused by TNNT2 gene mutations, are inadequate in effectively addressing the heart muscle dysfunction and associated risks such as heart failure and sudden death.
Method used
A gene therapy approach using an expression cassette that combines a TNNT2 transgene with a nucleic acid to reduce endogenous TNNT2 expression, enhancing TNNT2 protein incorporation in cardiac cells, potentially doubling the incorporation level.
Benefits of technology
This approach significantly increases TNNT2 protein incorporation in cardiac sarcomeres, offering a promising therapeutic strategy for cardiomyopathies by correcting the underlying genetic defects.
✦ Generated by Eureka AI based on patent content.
Abstract
The present invention provides expression cassettes and methods that reduce expression of an endogenous TNNT2 gene while providing a non-mutant TNNT2 transgene (tTNNT2), resulting in greatly increased TNNT2 transgene incorporation in the sarcomere than from delivery of the TNNT2 transgene alone.
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