Methods for assessing the risk of gene therapy for muscular dystrophy
RU2026107311APending Publication Date: 2026-07-01SAREPTA THERAPEUTICS INC +1
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Patent Information
- Authority / Receiving Office
- RU · RU
- Patent Type
- Applications
- Current Assignee / Owner
- SAREPTA THERAPEUTICS INC
- Filing Date
- 2024-09-16
- Publication Date
- 2026-07-01
AI Technical Summary
Technical Problem
There is a need for precise assessment of risks associated with administering gene therapy for Duchenne muscular dystrophy to avoid serious adverse effects and erroneous exclusion of subjects from treatment.
Method used
Methods involving genotyping the DMD gene and analyzing the HLA type of a subject before administering the gene therapy, such as delandistrogene moxeparvovec, to assess the risk of immune responses and ensure suitability for treatment.
Benefits of technology
Enhances the accuracy of risk assessment, allowing safe administration of gene therapy to subjects who would otherwise be incorrectly excluded, thereby reducing adverse effects and improving treatment efficacy.
✦ Generated by Eureka AI based on patent content.
Abstract
The disclosure provides for methods of assessing the risks (e.g., associated with a gene therapy for the treatment of DMD (e.g., delandistrogene moxeparvovec) comprising genotyping the DMD gene and analyzing the HLA type of a subject in need of the gene therapy (e.g., delandistrogene moxeparvovec).
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