Myotonic Dystrophy Pipeline Drugs Analysis Report, 2024 Updates: FDA Approvals, Clinical Trials, Therapies, MOA, ROA by DelveInsight | Dyne Therapeutics, Avidity Biosciences, Harmony Biosciences

Myotonic Dystrophy Pipeline Drugs Analysis Report, 2024 Updates: FDA Approvals, Clinical Trials, Therapies, MOA, ROA by DelveInsight | Dyne Therapeutics, Avidity Biosciences, Harmony Biosciences
(Las Vegas, Nevada, United States) As per DelveInsight’s assessment, globally, Myotonic Dystrophy pipeline constitutes 20+ key companies continuously working towards developing 22+ Myotonic Dystrophy treatment therapies, analysis of Clinical Trials, Therapies, Mechanism of Action, Route of Administration, and Developments analyzes DelveInsight.

The Myotonic Dystrophy Pipeline report embraces in-depth commercial and clinical assessment of the pipeline products from the pre-clinical developmental phase to the marketed phase. The report also covers a detailed description of the drug, including the mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, collaborations, mergers acquisition, funding, designations, and other product-related details.


Myotonic Dystrophy Pipeline Insight, 2024 report by DelveInsight outlines comprehensive insights into the present clinical development scenario and growth prospects across the Myotonic Dystrophy Market.


Some of the key takeaways from the Myotonic Dystrophy Pipeline Report:

  • Companies across the globe are diligently working toward developing novel Myotonic Dystrophy treatment therapies with a considerable amount of success over the years. 
  • Myotonic Dystrophy companies working in the treatment market are Design Therapeutics, Enzerna, Juvena Therapeutics, Dyne Therapeutics, Avidity Biosciences, Harmony Biosciences, AMO Pharma, Lupin, AMO Pharma, Dyne Therapeutics, Entrada Therapeutics, and others, are developing therapies for the Myotonic Dystrophy treatment 
  • Emerging Myotonic Dystrophy therapies in the different phases of clinical trials are- DM1 program, ENZ-001, JUV 161, DYNE-101, AOC 1001, Pitolisant, Tideglusib, Mexiletine, AMO-02, DYNE-101, ENTR-701, and others are expected to have a significant impact on the Myotonic Dystrophy market in the coming years.   
  • In August 2022, The US has designated ATX-01, the lead experimental product from Arthex Biotech, as an orphan drug. FDA, the Food and Drug Administration. Myotonic Dystrophy Type 1 (DM1), a terrible, crippling, and potentially fatal condition for which there is neither a known cure nor a drug treatment in development, is the target of ATX-01.
  • In July 2022, The New Zealand Medicines and Medical Devices Safety Authority approved Dyne Therapeutics, Inc.’s clinical trial application to begin its Phase I/II multiple ascending doses (MAD) clinical trials of DYNE-101 in patients with myotonic dystrophy type 1 (DM1), according to the company.
  • In May 2022, In response to developments in AMO Pharma’s potentially crucial REACH-CDM study, a double-blind, placebo-controlled, randomised clinical trial to evaluate the efficacy and safety of AMO-02 (tideglusib) for the treatment of congenital myotonic dystrophy (CDM1), the company AMO Pharma Limited announced that its current investors have increased their investments in the business.
  • In August 2021, AMO Pharma Limited started a study titled “A 52-Week, Open-Label Study to Evaluatethe Long-Term Safety and Efficacy of Tideglusib for the Treatment of Congenital DM1 (REACH CDM X)”. In this open-label follow-up study, children and adolescents with a diagnosis of congenital DM1 who successfully completed the AMO-02-MD-2-003 study will receive weight-adjusted 1000 mg tideglusib once daily for 52 weeks.


Myotonic Dystrophy Overview

The most prevalent form of muscular dystrophy that manifests in adulthood is myotonic dystrophy (DM), which is regarded as a subtype of myopathy. Myotonic dystrophy type I (DM1), also known as Steinert disease, and myotonic dystrophy type II (DM2), also known as proximal myotonic myopathy, which is a milder form of DMI, are the two main forms recognised based on clinical and molecular presentation.


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Emerging Myotonic Dystrophy Drugs Under Different Phases of Clinical Development Include:

  • DM1 program: Design Therapeutics
  • ENZ-001: Enzerna
  • JUV 161: Juvena Therapeutics
  • DYNE-101: Dyne Therapeutics
  • AOC 1001: Avidity Biosciences
  • Pitolisant: Harmony Biosciences
  • Tideglusib: AMO Pharma
  • Mexiletine: Lupin
  • AMO-02: AMO Pharma
  • DYNE-101: Dyne Therapeutics
  • ENTR-701: Entrada Therapeutics


Myotonic Dystrophy Route of Administration

Myotonic Dystrophy pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs, such as 

  • Oral
  • Parenteral
  • Intravenous
  • Subcutaneous
  • Topical


Myotonic Dystrophy Molecule Type

Myotonic Dystrophy Products have been categorized under various Molecule types, such as

  • Monoclonal Antibody
  • Peptides
  • Polymer
  • Small molecule
  • Gene therapy 


Myotonic Dystrophy Pipeline Therapeutics Assessment

  • Myotonic Dystrophy Assessment by Product Type
  • Myotonic Dystrophy By Stage and Product Type
  • Myotonic Dystrophy Assessment by Route of Administration
  • Myotonic Dystrophy By Stage and Route of Administration
  • Myotonic Dystrophy Assessment by Molecule Type
  • Myotonic Dystrophy by Stage and Molecule Type


DelveInsight’s Myotonic Dystrophy Report covers around 22+ products under different phases of clinical development like

  • Late-stage products (Phase III)
  • Mid-stage products (Phase II)
  • Early-stage product (Phase I)
  • Pre-clinical and Discovery stage candidates
  • Discontinued & Inactive candidates
  • Route of Administration


Further Myotonic Dystrophy product details are provided in the report. Download the Myotonic Dystrophy pipeline report to learn more about the emerging Myotonic Dystrophy therapies


Some of the key companies in the Myotonic Dystrophy Therapeutics Market include:

Key companies developing therapies for Myotonic Dystrophy are – Lupin, AMO Pharma, Harmony Biosciences, Avidity Biosciences, Dyne Therapeutics, Nexien BioPharma, Locana, Inc., Entrada Therapeutics, Arthex Biotech, NeuBase Therapeutics, NeuBase Therapeutics, Enzerna, Enzerna, Astellas Gene Therapies, Dyne Therapeutics, Pepgen Corporation, Sangamo Therapeutics, Syros Pharmaceuticals, and others.


Myotonic Dystrophy Pipeline Analysis:

The Myotonic Dystrophy pipeline report provides insights into 

  • The report provides detailed insights about companies that are developing therapies for the treatment of Myotonic Dystrophy with aggregate therapies developed by each company for the same.
  • It accesses the Different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for Myotonic Dystrophy Treatment.
  • Myotonic Dystrophy key companies are involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
  • Myotonic Dystrophy Drugs under development based on the stage of development, route of administration, target receptor, monotherapy or combination therapy, a different mechanism of action, and molecular type. 
  • Detailed analysis of collaborations (company-company collaborations and company-academia collaborations), licensing agreement and financing details for future advancement of the Myotonic Dystrophy market.

The report is built using data and information traced from the researcher’s proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations, and featured press releases from company/university websites and industry-specific third-party sources, etc.


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Myotonic Dystrophy Pipeline Market Drivers

  • Technological advancements in Myotonic Dystrophy genomic research, management of multisystem disorder associated with Myotonic Dystrophy are some of the important factors that are fueling the Myotonic Dystrophy Market.


Myotonic Dystrophy Pipeline Market Barriers

  • However, lack of Approved therapies, lack of understanding of pathophysiology and mechanisms of DNA instability and other factors are creating obstacles in the Myotonic Dystrophy Market growth.


Scope of Myotonic Dystrophy Pipeline Drug Insight    

  • Coverage: Global
  • Key Myotonic Dystrophy Companies: Design Therapeutics, Enzerna, Juvena Therapeutics, Dyne Therapeutics, Avidity Biosciences, Harmony Biosciences, AMO Pharma, Lupin, AMO Pharma, Dyne Therapeutics, Entrada Therapeutics, and others
  • Key Myotonic Dystrophy Therapies: DM1 program, ENZ-001, JUV 161, DYNE-101, AOC 1001, Pitolisant, Tideglusib, Mexiletine, AMO-02, DYNE-101, ENTR-701, and others
  • Myotonic Dystrophy Therapeutic Assessment: Myotonic Dystrophy current marketed and Myotonic Dystrophy emerging therapies
  • Myotonic Dystrophy Market Dynamics: Myotonic Dystrophy market drivers and Myotonic Dystrophy market barriers 


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Table of Contents 

1. Myotonic Dystrophy Report Introduction

2. Myotonic Dystrophy Executive Summary

3. Myotonic Dystrophy Overview

4. Myotonic Dystrophy- Analytical Perspective In-depth Commercial Assessment

5. Myotonic Dystrophy Pipeline Therapeutics

6. Myotonic Dystrophy Late Stage Products (Phase II/III)

7. Myotonic Dystrophy Mid Stage Products (Phase II)

8. Myotonic Dystrophy Early Stage Products (Phase I)

9. Myotonic Dystrophy Preclinical Stage Products

10. Myotonic Dystrophy Therapeutics Assessment

11. Myotonic Dystrophy Inactive Products

12. Company-University Collaborations (Licensing/Partnering) Analysis

13. Myotonic Dystrophy Key Companies

14. Myotonic Dystrophy Key Products

15. Myotonic Dystrophy Unmet Needs

16 . Myotonic Dystrophy Market Drivers and Barriers

17. Myotonic Dystrophy Future Perspectives and Conclusion

18. Myotonic Dystrophy Analyst Views

19. Appendix

20. About DelveInsight


About DelveInsight

DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing comprehensive end-to-end solutions to improve their performance. It also offers Healthcare Consulting Services, which benefits in market analysis to accelerate business growth and overcome challenges with a practical approach.

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