Facioscapulohumeral Muscular Dystrophy Clinical Trial Pipeline Shows Potential with Active Contributions from 10+ Key Companies | DelveInsight

DelveInsight's "Facioscapulohumeral Muscular Dystrophy Pipeline Insight 2025" report provides comprehensive insights about 10+ companies and 12+ pipeline drugs in the Facioscapulohumeral Muscular Dystrophy pipeline landscape. It covers the Facioscapulohumeral Muscular Dystrophy pipeline drug profiles, including clinical and nonclinical stage products. It also covers the Facioscapulohumeral Muscular Dystrophy therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

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Key Takeaways from the Facioscapulohumeral Muscular Dystrophy Pipeline Report

* In November 2025, Epicrispr Biotechnologies Inc. announced a clinical trial is to learn how safe and tolerable EPI-321 is and whether there may be early signs it is working in male or female adult (18 to 75 years) participants with facioscapulohumeral muscular dystrophy (FSHD) Type 1 condition.
* In November 2025, Hoffmann-La Roche conducted a study is to evaluate the pharmacodynamics, safety, tolerability, pharmacokinetics, and efficacy of RO7204239, a humanized monoclonal antibody that binds to human latent myostatin, in ambulant adult participants with facioscapulohumeral muscular dystrophy (FSHD).
* DelveInsight's Facioscapulohumeral Muscular Dystrophy pipeline report depicts a robust space with 10+ active players working to develop 12+ pipeline therapies for Facioscapulohumeral Muscular Dystrophy treatment.
* The leading Facioscapulohumeral Muscular Dystrophy Companies such as Avidity Biosciences Inc., Hoffmann-La Roche, Arrowhead Pharmaceuticals, Epicrispr Biotechnologies Inc., Dyne Therapeutics Inc . and others.
* Promising Facioscapulohumeral Muscular Dystrophy Therapies such as EPI-321, ATYR1940, RO7204239, Losmapimod oral tablet, AOC 1020, MYO-029 and others.

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The Facioscapulohumeral Muscular Dystrophy Pipeline Report provides a disease overview, pipeline scenario, and therapeutic assessment of the key pipeline therapies in this domain. The Facioscapulohumeral Muscular Dystrophy Pipeline Report also highlights the unmet needs with respect to the Facioscapulohumeral Muscular Dystrophy.

Facioscapulohumeral Muscular Dystrophy Overview

Facioscapulohumeral Muscular Dystrophy (FSHD) is a genetic muscle disorder characterized by the progressive weakening and wasting of skeletal muscles, primarily affecting the muscles of the face (facio), shoulders (scapulo), and upper arms (humeral). It is one of the most common forms of muscular dystrophy and typically begins in adolescence or early adulthood, though onset can vary. FSHD is caused by genetic changes on chromosome 4 that lead to abnormal activation of the DUX4 gene, which damages muscle cells over time. Symptoms often start with difficulty smiling, whistling, or lifting the arms and can gradually extend to the trunk and legs.

Facioscapulohumeral Muscular Dystrophy Emerging Drugs Profile

* AOC-1020: Avidity Biosciences, Inc.

AOC 1020 is designed to treat the underlying cause of FSHD, which is caused by the abnormal expression of a gene called double homeobox 4 or DUX4. The abnormal expression of DUX4 protein leads to changes in gene expression in muscle cells that are associated with the life-long, progressive loss of muscle function in patients with FSHD. AOC 1020 aims to reduce the expression of DUX4 mRNA and DUX4 protein in muscles in patients with FSHD. AOC 1020 consists of a proprietary monoclonal antibody that binds to the transferrin receptor 1 (TfR1) conjugated with a siRNA targeting DUX4 mRNA. In preclinical studies, a single intravenous dose with the murine version of AOC 1020 prevented development of muscle weakness demonstrated by three functional assays - treadmill running, in vivo force and compound muscle action potential. Currently, the drug is in Phase III stage of its development for the treatment of Facioscapulohumeral Muscular Dystrophy.

* RO7204239: Hoffmann-La Roche

Anti-latent myostatin (gym 329, RG6237) is a Sequential Monoclonal Antibody Recycling Technology - Immunoglobulin (SMART-Ig) using recycling and sweeping antibody technologies that eliminates latent myostatin from plasma and tissues. Latent myostatin is an inactive form that is mainly secreted from muscle cells, and is activated by BMP-1 and other protein degrading enzymes. Activated myostatin inhibits muscle growth and hypertrophy, and by inhibiting latent myostatin, gym 329 is more targeted and expected to improve the various conditions associated with muscle atrophy and loss of muscular strength. Currently, the drug is in Phase II stage of its development for the treatment of Facioscapulohumeral Muscular Dystrophy.

* ARO-DUX4: Arrowhead Pharmaceuticals

ARO-DUX4 is an RNA interference (RNAi) conjugate designed to specifically target the gene that encodes human double homeobox 4 (DUX4) protein as a potential treatment for patients with facioscapulohumeral muscular dystrophy type 1 (FSHD1). FSHD1 is an autosomal dominant disease associated with the failure to maintain complete epigenetic suppression of DUX4 expression in differentiated skeletal muscle, leading to overexpression of DUX4, which is myotoxic and can lead to muscle degeneration. Published literature suggests that the silencing of aberrantly transcribed DUX4 mRNA using ARO-DUX4 may halt progression of, and possibly reverse, DUX4-induced muscle toxicities in patients with FSHD1, improving muscle strength and function. Currently, the drug is in Phase I/II stage of its development for the treatment of Facioscapulohumeral Muscular Dystrophy.

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The Facioscapulohumeral Muscular Dystrophy Pipeline Report Provides Insights into

* The report provides detailed insights about companies that are developing therapies for the treatment of Facioscapulohumeral Muscular 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 Facioscapulohumeral Muscular Dystrophy Treatment.
* Facioscapulohumeral Muscular Dystrophy Companies are involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
* Facioscapulohumeral Muscular 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 Facioscapulohumeral Muscular Dystrophy market

Facioscapulohumeral Muscular Dystrophy Companies

Avidity Biosciences Inc., Hoffmann-La Roche, Arrowhead Pharmaceuticals, Epicrispr Biotechnologies Inc., Dyne Therapeutics Inc . and others.

The Facioscapulohumeral Muscular 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
* Intravenous
* Subcutaneous
* Parenteral
* Topical

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

* Products have been categorized under various Molecule types such as
* Recombinant fusion proteins
* Small molecule
* Monoclonal antibody
* Peptide
* Polymer
* Gene therapy

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Scope of the Facioscapulohumeral Muscular Dystrophy Pipeline Report

* Coverage- Global
* Facioscapulohumeral Muscular Dystrophy Companies- Avidity Biosciences Inc., Hoffmann-La Roche, Arrowhead Pharmaceuticals, Epicrispr Biotechnologies Inc., Dyne Therapeutics Inc . and others.
* Facioscapulohumeral Muscular Dystrophy Therapies- EPI-321, ATYR1940, RO7204239, Losmapimod oral tablet, AOC 1020, MYO-029 and others
* Facioscapulohumeral Muscular Dystrophy Therapeutic Assessment by Product Type: Mono, Combination, Mono/Combination
* Facioscapulohumeral Muscular Dystrophy Therapeutic Assessment by Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III

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Table of Contents

* Introduction
* Executive Summary
* Facioscapulohumeral Muscular Dystrophy: Overview
* Pipeline Therapeutics
* Therapeutic Assessment
* Facioscapulohumeral Muscular Dystrophy- DelveInsight's Analytical Perspective
* Late Stage Products (Phase III)
* AOC-1020: Avidity Biosciences, Inc.
* Mid Stage Products (Phase II)
* RO7204239: Hoffmann-La Roche
* Early Stage Products (Phase I/II)
* ARO-DUX4: Arrowhead Pharmaceuticals
* Preclinical and Discovery Stage Products
* Inactive Products
* Facioscapulohumeral Muscular Dystrophy Key Companies
* Facioscapulohumeral Muscular Dystrophy Key Products
* Facioscapulohumeral Muscular Dystrophy- Unmet Needs
* Facioscapulohumeral Muscular Dystrophy- Market Drivers and Barriers
* Facioscapulohumeral Muscular Dystrophy- Future Perspectives and Conclusion
* Facioscapulohumeral Muscular Dystrophy Analyst Views
* Facioscapulohumeral Muscular Dystrophy Key Companies
* Appendix

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