Methods for treating muscular dystrophy

A Duchenne muscular dystrophy, poor technology, used in gene therapy, biochemical equipment and methods, pharmaceutical formulations, etc., to solve problems such as limited pharmacological options

Pending Publication Date: 2020-07-14
SAREPTA THERAPEUTICS INC
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  • Summary
  • Abstract
  • Description
  • Claims
  • Application Information

AI Technical Summary

Problems solved by technology

However, despite the success of these trials, the pharmacological options available for the treatment of DMD are limited

Method used

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  • Methods for treating muscular dystrophy
  • Methods for treating muscular dystrophy
  • Methods for treating muscular dystrophy

Examples

Experimental program
Comparison scheme
Effect test

example

[0195] All examples are derived from the following ongoing first-in-human clinical trials testing the safety and efficacy of SRP-4053. The results reported here were obtained at week 48 during part 2 of the study.

[0196] Phase I / II Study of SRP-4053 in DMD Patients

[0197] ClinicalTrials.gov Identifier: NCT02310906

[0198] This is the first human multiple-dose 2-part study evaluating the safety, tolerability, efficacy and pharmacokinetics of SRP-4053 in Duchenne muscular dystrophy (DMD) patients with deletions suitable for exon 53 skipping .

[0199] Study Type: Interventional

[0200] Study Design: Allocation: Randomized

[0201] Intervention Mode: Parallel Assignment

[0202] Blinding: Quadruple (Participant, Care Provider, Investigator, Outcome Assessor)

[0203] Primary Purpose: Healing

[0204] Official Title: 2-Part, Randomized, Double-Blind, Placebo-Controlled, Dose-Titration, Safety, Tolerability, and Pharmacokinetic Study (Part 1), followed by SRP-4053 in E...

example 1

[0257] Example 1: Biochemical Efficacy Assessment

[0258] Paired muscle biopsies of the biceps brachii at baseline and on treatment were obtained from 25 patients participating in a multisite first-in-human trial evaluating the safety, tolerability, and tolerability of 30 mg / kg SRP-4053 administered weekly by intravenous infusion and dystrophin production (ClinicalTrials.gov Identifier: NCT02310906). For each surgery, two pieces of muscle are removed: the A block and the B block. For all assays, A and B blocks were analyzed separately.

[0259] Muscle biopsies were tested by optimized methods to assess dystrophin protein quantity (Western blot, primary biological endpoint) and exon skipping (RT-PCR). Novel automated imaging analysis (MuscleMap TM )) Immunohistochemistry was used to assess dystrophin localization (mean fiber intensity).

[0260] For Western blot analysis: Panels A and B run on duplicate gels = 4 tests averaged

[0261] For RT-PCR analysis: Blocks A and B ...

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Abstract

The present disclosure provides, among other things, improved compositions and methods for treating muscular dystrophy. For example, the disclosure provides methods for treating Duchenne muscular dystrophy patients having a mutation in the DMD gene that is amenable to exon 53 skipping by administering an effective amount of golodirsen.

Description

technical field [0001] The present invention relates to improved methods of treating muscular dystrophy in patients. It also provides compositions suitable for promoting exon 53 skipping in the human dystrophin gene. Background technique [0002] In a variety of genetic diseases, the effects of mutations on the ultimate expression of genes can be modulated by the process of targeted exon skipping during the splicing process. In cases where normally functional proteins are terminated prematurely due to mutations in them, a means of restoring the production of some functional proteins by antisense technology has been shown to be possible by intervening during the splicing process and if the exon associated with the causative mutation can Specific deletions from a few genes can sometimes result in shortened protein products that have biological properties similar to the native protein or have sufficient biological activity to ameliorate diseases caused by exon-associated mutat...

Claims

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Application Information

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Patent Type & Authority Applications(China)
IPC IPC(8): A61K31/00C12N15/113
CPCC12N15/111C12N2310/11C12N2310/3233C12N2320/33A61P21/00A61K31/7105A61K31/5377A61K31/7125G01N33/68
Inventor E.M.凯
Owner SAREPTA THERAPEUTICS INC
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