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Gene Therapy for Muscular Dystrophy - A Pipeline Analysis Report

  • Published: Jul 2018
  • Pages: 82
  • SKU: IRTNTR23466
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This pipeline analysis report provides detailed insights into the clinical trials landscape of the gene therapy for muscular dystrophy including molecules at pre-clinical, discovery stage, and other developmental stages. The report also offers comprehensive information about the therapeutic assessment of the pipeline molecules based on various segmentation's such as RoA and therapeutic modality.

Overview of the gene therapy pipeline for muscular dystrophy 

The degeneration or weakening of skeletal muscles is caused due to a muscular dystrophy, a group of muscle diseases. Individuals start losing muscle mass if they are suffering from muscular dystrophy. The most common reason for muscular dystrophy is the mutation of the protein dystrophin. Moreover, genetic mutations are also responsible for this disease as it interferes with the muscle protein production. According to this pipeline analysis report, the growing instances of the muscular dystrophy are expected to proliferate the gene therapy for muscular dystrophy in the next few years. There are over 30 various types of muscular dystrophies with a few being the most common types such as Duchenne muscular dystrophy, Becker muscular dystrophy, and limb-girdle muscular dystrophy.

As per our pipeline analysis report, most of the gene therapy molecules in the pipeline are in the pre-clinical stages. For instance, sponsors such as Genethon developed novel gene therapy and Myonexus therapeutics developed MYO-101, which are under the pre-clinical stage. The pipeline landscape in the pipeline analysis report mentions the percentage of gene therapy molecules that are under discovery, pre-clinical, phase I, and phase I/II.

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Pipeline analysis report on gene therapy for muscular dystrophy: Companies covered

This pipeline analysis report provides a detailed analysis of the companies that are involved in the development of gene therapy molecules for the treatment of muscular dystrophy. In addition to providing information on the various stages of molecules developed by companies for different indications, this pipeline analysis report also provides information about the gene therapy molecules discontinued by companies.

Some of the companies covered in this pipeline analysis report are:

  • Genethon
  • Pfizer
  • Solid Biosciences

Therapeutic assessment of the gene therapy pipeline for muscular dystrophy by RoA

  • Intravenous
  • Parenteral

The intravenous route of administration (RoA) involves the administration of drug substances through veins. It has been observed that the majority of total therapeutics are being developed for intravenous administration.

Therapeutic assessment of the gene therapy pipeline for muscular dystrophy by therapeutic modality

  • Gene
  • Oligonucleotides

According to this pipeline analysis report, the majority of molecules that are currently in the gene therapy pipeline for Muscular Dystrophy are being developed as a gene. A gene is a sequence of DNA and RNA that encodes the function. It is the physical and functional unit of heredity.

Key questions answered in the report include

  • What are the gene therapy molecules in the various development stages for muscular dystrophy?
  • What are the companies that are currently involved in the development of gene therapy molecules for muscular dystrophy?
  • Insight into discontinued/inactive molecules with appropriate reasoning?
  • What are the major regulatory authorities approving drugs in various regions?
  • Detailed profiling of each active molecule

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PART 01: EXECUTIVE SUMMARY

PART 02: SCOPE OF THE REPORT

PART 03: RESEARCH METHODOLOGY

PART 04: INTRODUCTION

PART 05: MAJOR REGULATORY AUTHORITIES

  • US
  • Europe
  • China

PART 06: PIPELINE LANDSCAPE

PART 07: COMPARATIVE ANALYSIS

  • Discovery stage molecules
  • Pre-clinical stage molecules
  • Inactive and discontinued molecules

PART 08: INDICATION ANALYSIS

PART 09: THERAPEUTIC ASSESSMENT (THERAPY BASED)

PART 10: THERAPEUTIC ASSESSMENT (ROA BASED)

PART 11: THERAPEUTIC ASSESSMENT BY TARGET

PART 12: KEY COMPANIES

  • Active companies: Category and parameters

PART 13: APPENDIX

  • List of abbreviations

Research Framework

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TechnavioDATA ANALYSIS

Data Synthesis

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  • Analysis of derived insights

Data Validation

  • Triangulation with data models
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  • Corroboration with industry experts
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