Muscular Dystrophy Drugs in Development by Stages, Target, MoA, RoA, Molecule Type and Key Players, 2022 Update

October 2022 | 218 pages | ID: MCE64D166542EN
Global Markets Direct

Abstracts

Muscular Dystrophy Drugs in Development by Stages, Target, MoA, RoA, Molecule Type and Key Players, 2022 Update

SUMMARY

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Muscular Dystrophy - Drugs In Development, 2022, provides an overview of the Muscular Dystrophy (Musculoskeletal Disorders) pipeline landscape.

Muscular dystrophy is a group of diseases in which muscle fibers are unusually susceptible to damage. These damaged muscles become progressively weaker. Symptoms usually appear before age 6 and may appear as early as infancy. They may include fatigue, learning difficulties, intellectual disability, muscle weakness and progressive difficulty walking.

REPORT HIGHLIGHTS

Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Muscular Dystrophy - Drugs In Development, 2022, provides comprehensive information on the therapeutics under development for Muscular Dystrophy (Musculoskeletal Disorders), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The guide covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases.

The Muscular Dystrophy (Musculoskeletal Disorders) pipeline guide also reviews of key players involved in therapeutic development for Muscular Dystrophy and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies/Universities/Institutes, the molecules developed by Companies in Phase III, Phase II, Phase I, IND/CTA Filed, Preclinical and Discovery stages are 4, 10, 9, 1, 52 and 29 respectively. Similarly, the Universities portfolio in Phase II, Preclinical and Discovery stages comprises 2, 6 and 8 molecules, respectively.

Muscular Dystrophy (Musculoskeletal Disorders) pipeline guide helps in identifying and tracking emerging players in the market and their portfolios, enhances decision making capabilities and helps to create effective counter strategies to gain competitive advantage. The guide is built using data and information sourced from Global Markets Direct’s proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.

Note: Certain content/sections in the pipeline guide may be removed or altered based on the availability and relevance of data.

SCOPE

• The pipeline guide provides a snapshot of the global therapeutic landscape of Muscular Dystrophy (Musculoskeletal Disorders).
• The pipeline guide reviews pipeline therapeutics for Muscular Dystrophy (Musculoskeletal Disorders) by companies and universities/research institutes based on information derived from company and industry-specific sources.
• The pipeline guide covers pipeline products based on several stages of development ranging from pre-registration till discovery and undisclosed stages.
• The pipeline guide features descriptive drug profiles for the pipeline products which comprise, product description, descriptive licensing and collaboration details, R&D brief, MoA & other developmental activities.
• The pipeline guide reviews key companies involved in Muscular Dystrophy (Musculoskeletal Disorders) therapeutics and enlists all their major and minor projects.
• The pipeline guide evaluates Muscular Dystrophy (Musculoskeletal Disorders) therapeutics based on mechanism of action (MoA), drug target, route of administration (RoA) and molecule type.
• The pipeline guide encapsulates all the dormant and discontinued pipeline projects.
• The pipeline guide reviews latest news related to pipeline therapeutics for Muscular Dystrophy (Musculoskeletal Disorders)

REASONS TO BUY

• Procure strategically important competitor information, analysis, and insights to formulate effective R&D strategies.
• Recognize emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage.
• Find and recognize significant and varied types of therapeutics under development for Muscular Dystrophy (Musculoskeletal Disorders).
• Classify potential new clients or partners in the target demographic.
• Develop tactical initiatives by understanding the focus areas of leading companies.
• Plan mergers and acquisitions meritoriously by identifying key players and it’s most promising pipeline therapeutics.
• Formulate corrective measures for pipeline projects by understanding Muscular Dystrophy (Musculoskeletal Disorders) pipeline depth and focus of Indication therapeutics.
• Develop and design in-licensing and out-licensing strategies by identifying prospective partners with the most attractive projects to enhance and expand business potential and scope.
• Adjust the therapeutic portfolio by recognizing discontinued projects and understand from the know-how what drove them from pipeline.

Contents

Introduction
Global Markets Direct Report Coverage
Muscular Dystrophy - Overview
Muscular Dystrophy - Therapeutics Development
Pipeline Overview
Pipeline by Companies
Pipeline by Universities/Institutes
Products under Development by Companies
Products under Development by Universities/Institutes
Muscular Dystrophy - Therapeutics Assessment
Assessment by Target
Assessment by Mechanism of Action
Assessment by Route of Administration
Assessment by Molecule Type
Muscular Dystrophy - Companies Involved in Therapeutics Development
Muscular Dystrophy - Drug Profiles
Muscular Dystrophy - Dormant Projects
Muscular Dystrophy - Discontinued Products
Muscular Dystrophy - Product Development Milestones
Featured News & Press Releases
Oct 14, 2022: Avidity Biosciences announces upcoming presentation on AOC-1001 at 27th International Hybrid Annual Congress of World Muscle Society
Oct 14, 2022: Avidity Biosciences announces upcoming presentation on AOC-1001 at 27th International Hybrid Annual Congress of World Muscle Society
Sep 28, 2022: FDA places partial hold on Avidity’s Phase I/II myotonic dystrophy trial
Sep 27, 2022: Atamyo doses first subject in Phase I/II muscular dystrophy therapy trial
Aug 04, 2022: Entrada Therapeutics announces collaboration with the Myotonic Dystrophy Clinical Research Network to study the natural history of myotonic dystrophy type
Aug 02, 2022: Avidity Biosciences enrolls patients in the MARINA open-label extension study
Jul 13, 2022: Fulcrum phase 3 FSHD clinical trial using AMRA Medical's Whole-body MRI Measurements as a Key Secondary Endpoint
Jul 12, 2022: Dyne Therapeutics announces clearance of clinical trial application for DYNE-101 for the treatment of myotonic dystrophy type
Jul 05, 2022: Fulcrum Therapeutics enrolls first patient in pivotal global Phase 3 clinical trial of Losmapimod for facioscapulohumeral muscular dystrophy (FSHD)
Jun 20, 2022: New muscle disease indication for ATL1102 - Limb girdle muscular dystrophy R2
May 17, 2022: NeuBase presents new preclinical data at ASGCT 2022 for its DM1 program demonstrating wide tissue distribution and supporting a differentiated whole-body treatment solution
May 16, 2022: Dyne Therapeutics presents new in vivo data from DYNE-101 at ASGCT Annual Meeting demonstrating low monthly dosing leads to robust DMPK RNA knockdown
May 16, 2022: Atamyo Therapeutics announces significant for ATA-100 to treat Limb-Girdle Muscular Dystrophy 2I/R9
May 11, 2022: Entrada Therapeutics presents new data supporting its growing pipeline of Endosomal Escape Vehicle (EEV™) therapeutics, including ENTR-701 at TIDES USA 2022
May 02, 2022: NeuBase Therapeutics announces presentations at the American Society of Gene & Cell Therapy (ASGCT) 2022 Annual Meeting
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer

List Of Tables

LIST OF TABLES

Number of Products under Development for Muscular Dystrophy, 2022
Number of Products under Development by Companies, 2022
Number of Products under Development by Universities/Institutes, 2022
Products under Development by Companies, 2022
Products under Development by Universities/Institutes, 2022
Number of Products by Stage and Target, 2022
Number of Products by Stage and Mechanism of Action, 2022
Number of Products by Stage and Route of Administration, 2022
Number of Products by Stage and Molecule Type, 2022
Muscular Dystrophy - Dormant Projects, 2022
Muscular Dystrophy - Dormant Projects, 2022 (Contd..1)
Muscular Dystrophy - Dormant Projects, 2022 (Contd..2)
Muscular Dystrophy - Dormant Projects, 2022 (Contd..3)
Muscular Dystrophy - Discontinued Products, 2022

List Of Figures

LIST OF FIGURES

Number of Products under Development for Muscular Dystrophy, 2022
Number of Products under Development by Companies, 2022
Number of Products under Development by Universities/Institutes, 2022
Number of Products by Top 10 Targets, 2022
Number of Products by Stage and Top 10 Targets, 2022
Number of Products by Top 10 Mechanism of Actions, 2022
Number of Products by Stage and Top 10 Mechanism of Actions, 2022
Number of Products by Top 10 Routes of Administration, 2022
Number of Products by Stage and Top 10 Routes of Administration, 2022
Number of Products by Top 10 Molecule Types, 2022
Number of Products by Stage and Top 10 Molecule Types, 2022