“Charcot Marie Tooth Disease Pipeline Insight, 2025“ report by DelveInsight outlines comprehensive insights into the present clinical development scenario and growth prospects across the Charcot Marie Tooth Disease Market.

The Charcot Marie Tooth Disease 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.

Some of the key takeaways from the Charcot Marie Tooth Disease Pipeline Report:

• Companies across the globe are diligently working toward developing novel Charcot Marie Tooth Disease treatment therapies with a considerable amount of success over the years.

• Charcot Marie Tooth Disease companies working in the treatment market are DTx Pharma, Augustine therapeutics, Addex Therapeutics, InFlectis BioScience, Chong Kun Dang pharmaceutical, ENCell, HELIXMITH, Pharnext SA, MedDay Pharmaceuticals, and others, are developing therapies for the Charcot Marie Tooth Disease treatment

• Emerging Charcot Marie Tooth Disease therapies in the different phases of clinical trials are- DTx-1252, HDAC6i, Research programme: GABAB PAM, IFB-088, CKD-510, EN001, Engensis, PXT3003, MD1003, and others are expected to have a significant impact on the Charcot Marie Tooth Disease market in the coming years.

• In June 2025, Vanda Pharmaceuticals Inc. (Nasdaq: VNDA) announced that the first patient has been dosed in its first-in-human clinical trial evaluating VCA-894A, an antisense oligonucleotide (ASO) therapy, for a rare IGHMBP2 gene variant causing Charcot-Marie-Tooth disease Type 2S (CMT2S). The patient, diagnosed at age five, has this rare inherited neuromuscular disorder, which progressively causes muscle weakness and loss of motor function. CMT2S affects fewer than 1 in 1,000,000 people globally, with its severity and symptoms varying based on the diverse genetic variants linked to CMT disease.

• In May 2025, Augustine Therapeutics NV (“Augustine”), a biotechnology company developing novel therapies for neuromuscular, neurodegenerative, and cardio-metabolic diseases by inhibiting the cytosolic Histone DeACetylase 6 (HDAC6) enzyme, announced that the first patient has been dosed in its Phase I clinical trial of lead candidate AGT-100216, the first peripherally restricted, selective HDAC6 inhibitor.

• In March 2025, Actio Biosciences, a clinical-stage biotechnology company applying its one-to-many approach in genetics and precision medicine to develop therapies targeting shared underlying biology in rare and common diseases, announced that the first participant has been dosed in a Phase 1 healthy volunteer trial of its lead candidate, ABS-0871. This TRPV4 inhibitor is being developed for the treatment of TRPV4-positive Charcot-Marie-Tooth disease subtype 2C (CMT2C), a severe peripheral nerve disorder.

• In May 2025, Applied Therapeutics, Inc. (Nasdaq: APLT), a clinical-stage biopharmaceutical company focused on developing transformative therapies for rare diseases, presented full 12-month clinical results along with new topline data at 18 and 24 months from the INSPIRE Phase 2/3 trial of govorestat (AT-007) for treating Sorbitol Dehydrogenase (SORD) Deficiency, a subtype of Charcot-Marie-Tooth (CMT) disease. The data were shared in an oral presentation at the Peripheral Nerve Society (PNS) 2025 Annual Meeting, held May 17-20, 2025, in Edinburgh, Scotland.

• In March 2025, ENCell, a prominent biopharmaceutical company specializing in cell and gene therapy CDMO services and innovative drug development, announced that its investigational drug EN001 has received Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA) for treating Charcot-Marie-Tooth disease (CMT). CMT is an inherited neuromuscular disorder that leads to progressive muscle weakness and deformities in the hands and feet, and in severe cases, may result in vision and hearing impairments.

• In January 2025, Biotechnology company NMD Pharma announced that the US Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to its Charcot-Marie-Tooth (CMT) disease therapy, NMD670. Previously, NMD670 had received ODD from the FDA for generalized myasthenia gravis in September 2022. NMD670 is a small molecule inhibitor targeting the skeletal muscle-specific chloride ion channel CIC-1. In November 2024, NMD Pharma initiated the Phase 2 SYNAPSE-CMT trial, evaluating a twice-daily oral dose of NMD670 over 21 days in 80 adult patients with genetically confirmed CMT1 or CMT2 subtypes. CMT is a hereditary neuropathy affecting an estimated 136,000 individuals in the US and three million globally, causing muscle weakness, fatigue, and sensory deficits.

Charcot Marie Tooth Disease Overview

Charcot-Marie-Tooth disease, also known as hereditary motor and sensory neuropathy (HMSN), is a group of inherited neurological disorders that affect the peripheral nerves. These nerves are responsible for transmitting signals between the brain and spinal cord to the muscles, skin, and sensory organs, controlling movements and sensation in the arms and legs.

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Emerging Charcot Marie Tooth Disease Drugs Under Different Phases of Clinical Development Include:

• DTx-1252: DTx Pharma

• HDAC6i: Augustine therapeutics

• Research programme: GABAB PAM: Addex Therapeutics

• IFB-088: InFlectis BioScience

• CKD-510: Chong Kun Dang pharmaceutical

• EN001: ENCell

• Engensis: HELIXMITH

• PXT3003: Pharnext SA

• MD1003: MedDay Pharmaceuticals

Charcot Marie Tooth Disease Route of Administration

Charcot Marie Tooth Disease pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs, such as

• Intra-articular

• Intraocular

• Intrathecal

• Intravenous

• Ophthalmic

• Oral

• Parenteral

• Subcutaneous

• Topical

• Transdermal

Charcot Marie Tooth Disease Molecule Type

Charcot Marie Tooth Disease Products have been categorized under various Molecule types, such as

• Oligonucleotide

• Peptide

• Small molecule

Charcot Marie Tooth Disease Pipeline Therapeutics Assessment

• Charcot Marie Tooth Disease Assessment by Product Type

• Charcot Marie Tooth Disease By Stage and Product Type

• Charcot Marie Tooth Disease Assessment by Route of Administration

• Charcot Marie Tooth Disease By Stage and Route of Administration

• Charcot Marie Tooth Disease Assessment by Molecule Type

• Charcot Marie Tooth Disease by Stage and Molecule Type

DelveInsight’s Charcot Marie Tooth Disease Report covers around 15+ 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 Charcot Marie Tooth Disease product details are provided in the report. Download the Charcot Marie Tooth Disease pipeline report to learn more about the emerging Charcot Marie Tooth Disease therapies

Some of the key companies in the Charcot Marie Tooth Disease Therapeutics Market include:

Key companies developing therapies for Charcot Marie Tooth Disease are – Pharnext SA, MedDay Pharmaceuticals, HELIXMITH, ENCell, Chong Kun Dang Pharmaceutical, InFlectis BioScience, Addex Therapeutics, Augustine Therapeutics, DTx Pharma, and others.

Charcot Marie Tooth Disease Pipeline Analysis:

The Charcot Marie Tooth Disease pipeline report provides insights into

• The report provides detailed insights about companies that are developing therapies for the treatment of Charcot Marie Tooth Disease 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 Charcot Marie Tooth Disease Treatment.

• Charcot Marie Tooth Disease key companies are involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.

• Charcot Marie Tooth Disease 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 Charcot Marie Tooth Disease 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.

Download Sample PDF Report to know more about Charcot Marie Tooth Disease drugs and therapies

Charcot Marie Tooth Disease Pipeline Market Drivers

• Increase in prevalence of Charcot Marie Tooth Disease, increase in Research and development Activities are some of the important factors that are fueling the Charcot Marie Tooth Disease Market.

Charcot Marie Tooth Disease Pipeline Market Barriers

• However, lack of FDA approved treatment, difficulty in diagnosis of the disease and other factors are creating obstacles in the Charcot Marie Tooth Disease Market growth.

Scope of Charcot Marie Tooth Disease Pipeline Drug Insight

• Coverage: Global

• Key Charcot Marie Tooth Disease Companies: DTx Pharma, Augustine therapeutics, Addex Therapeutics, InFlectis BioScience, Chong Kun Dang pharmaceutical, ENCell, HELIXMITH, Pharnext SA, MedDay Pharmaceuticals, and others

• Key Charcot Marie Tooth Disease Therapies: DTx-1252, HDAC6i, Research programme: GABAB PAM, IFB-088, CKD-510, EN001, Engensis, PXT3003, MD1003, and others

• Charcot Marie Tooth Disease Therapeutic Assessment: Charcot Marie Tooth Disease current marketed and Charcot Marie Tooth Disease emerging therapies

• Charcot Marie Tooth Disease Market Dynamics: Charcot Marie Tooth Disease market drivers and Charcot Marie Tooth Disease market barriers

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Table of Contents

1. Charcot Marie Tooth Disease Report Introduction

2. Charcot Marie Tooth Disease Executive Summary

3. Charcot Marie Tooth Disease Overview

4. Charcot Marie Tooth Disease- Analytical Perspective In-depth Commercial Assessment

5. Charcot Marie Tooth Disease Pipeline Therapeutics

6. Charcot Marie Tooth Disease Late Stage Products (Phase II/III)

7. Charcot Marie Tooth Disease Mid Stage Products (Phase II)

8. Charcot Marie Tooth Disease Early Stage Products (Phase I)

9. Charcot Marie Tooth Disease Preclinical Stage Products

10. Charcot Marie Tooth Disease Therapeutics Assessment

11. Charcot Marie Tooth Disease Inactive Products

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

13. Charcot Marie Tooth Disease Key Companies

14. Charcot Marie Tooth Disease Key Products

15. Charcot Marie Tooth Disease Unmet Needs

16 . Charcot Marie Tooth Disease Market Drivers and Barriers

17. Charcot Marie Tooth Disease Future Perspectives and Conclusion

18. Charcot Marie Tooth Disease 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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