DelveInsight’s, “Pompe Disease Pipeline Insight 2025” report provides comprehensive insights about 15+ companies and 20+ pipeline drugs in Pompe Disease pipeline landscape. It covers the Pompe Disease pipeline drug profiles, including clinical and nonclinical stage products. It also covers the Pompe Disease pipeline 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 Pompe Disease Pipeline Report

• In March 2025, Astellas Gene Therapies announced a study (FORTIS) will evaluate the safety and efficacy of an investigational gene replacement therapy, AT845, in adult subjects with LOPD. Subjects will receive a single dose of AT845 delivered via intravenous (IV) infusion.
• In March 2025, Amicus Therapeutics conducted a study for subjects who completed the ATB200-03 study. The subjects will stay in this study until regulatory approval or marketing authorization and/or commercialization in the participating subject’s country.
• In March 2025, Sanofi announced a study of the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Avalglucosidase Alfa in Treatment naïve Pediatric Participants with Infantile-Onset Pompe Disease (IOPD).
• DelveInsight’s Pompe Disease pipeline report depicts a robust space with 15+ active players working to develop 20+ pipeline therapies for Pompe Disease treatment.
• The leading Pompe Disease Companies such as Asklepios Biopharmaceutical, GeneCradle Therapeutics, Astellas Gene Therapies, Maze Therapeutics, M6P Therapeutics, Entrada Therapeutics and others.
• Promising Pompe Disease Pipeline Therapies such as ALGLUCOSIDASE ALFA, ACTUS-101, ATB200, AT2221, BMN 701, Duvoglustat, Myozyme, Methotrexate, GC301 and others.

Stay informed about the cutting-edge advancements in Pompe Disease Treatments. Download for updates and be a part of the revolution in Genetic Disorders Care @ Pompe Disease Clinical Trials Assessment

Pompe Disease Emerging Drugs Profile

• GC301: GeneCradle Therapeutics

GC301 injection is an AAV gene therapy drug designed and developed by Genecradle Therapeutics for the treatment of Pompe disease. It adopts a one-time intravenous injection strategy for widespread systemic expression, aiming to directly compensate for the GAA gene deficiency in tissues such as the liver, heart, skeletal muscles, and central nervous system. In previous clinical trials conducted (IIT+IND), IOPD subjects successfully discontinued enzyme replacement therapy after gene therapy, with varying degrees of improvement in motor ability. Currently, the drug is in the Phase I/II stage of its clinical trial for the treatment of Pompe disease.

• MZE001: Maze Therapeutics

MZE001 is an oral glycogen synthase (GYS1) inhibitor that aims to address Pompe disease by limiting disease-causing glycogen buildup. GYS1 is an enzyme responsible for glycogen production. MZE001 is currently being evaluated as a potential oral treatment for patients with Pompe disease, as well as other glycogen storage disorders. Currently, the drug is in the Phase I stage of its clinical trial for the treatment of Pompe disease.

Pompe Disease Market Drivers

• Increased Incidence of Pompe Disease
• Increased Research and Clinical Activities

Pompe Disease Market Barriers

• Substantial costs associated with the treatment
• Late Pompe Disease Diagnosis

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Pompe Disease Companies

Asklepios Biopharmaceutical, GeneCradle Therapeutics, Astellas Gene Therapies, Maze Therapeutics, M6P Therapeutics, Entrada Therapeutics and others.

The Pompe Disease Pipeline Report provides insights into

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

Pompe 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

• Oral
• Intravenous
• Subcutaneous
• Parenteral
• Topical

Pompe Disease Products have been categorized under various Molecule types such as

• Recombinant fusion proteins
• Small molecule
• Monoclonal antibody
• Peptide
• Polymer
• Gene therapy

Discover the latest advancements in Pompe Disease Treatment by visiting our website. Stay informed about how we’re transforming the future of genetic disorders @ Pompe Disease Market Drivers and Barriers, and Future Perspectives

Scope of the Pompe Disease Pipeline Report

• Coverage- Global
• Pompe Disease Companies- Asklepios Biopharmaceutical, GeneCradle Therapeutics, Astellas Gene Therapies, Maze Therapeutics, M6P Therapeutics, Entrada Therapeutics and others.
• Pompe Disease Pipeline Therapies- ALGLUCOSIDASE ALFA, ACTUS-101, ATB200, AT2221, BMN 701, Duvoglustat, Myozyme, Methotrexate, GC301 and others.
• Pompe Disease Therapeutic Assessment by Product Type: Mono, Combination, Mono/Combination
• Pompe Disease Therapeutic Assessment by Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III

For a detailed overview of our latest research findings and future plans, read the full details of Pompe Disease Pipeline on our website @ Pompe Disease Emerging Drugs and Companies

Table of Contents

1. Introduction
2. Executive Summary
3. Pompe Disease: Overview
4. Pipeline Therapeutics
5. Therapeutic Assessment
6. Pompe Disease– DelveInsight’s Analytical Perspective
7. Late Stage Products (Phase III)
8. Drug name: Company name
9. Mid-Stage Products (Phase II)
10. Drug name: Company name
11. Early Stage Products (Phase I)
12. MZE001: Maze Therapeutics
13. Preclinical and Discovery Stage Products
14. ERT/Oligonucleotide: Entrada Therapeutics
15. Inactive Products
16. Pompe Disease Companies
17. Pompe Disease Products
18. Pompe Disease Unmet Needs
19. Pompe Disease Market Drivers and Barriers
20. Pompe Disease Future Perspectives and Conclusion
21. Pompe Disease Analyst Views
22. Pompe Disease Key Companies
23. Appendix

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