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Pompe Disease Pipeline

DelveInsight’s, “Pompe Disease Pipeline Insights, 2024,” report provides comprehensive insights about 15+ companies and 20+ pipeline drugs in Pompe Disease pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the 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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Some of the key highlights from the Pompe Disease Pipeline report:

• The Pompe Disease Pipeline report offers a rich analysis of 15+ key players and 20+ key therapies.
• Pompe Disease pipeline comprises therapies in different stages of the clinical phase such as include Cipaglucosidase alfa, SPK-3006, ACTUS 101, AT845, RP A501, AOC-Pompe disease, AVR RD 03, GYS1 Program, ETV-GAA, and many others expected to enter the Pompe Disease market in the coming years.
• Key companies strengthening the Pompe Disease Pipeline are Spark Therapeutics, Amicus Therapeutics, Asklepios Biopharmaceutical, Audentes Therapeutics, Rocket Pharmaceuticals, Avidity Biosciences, AVROBIO, Maze Therapeutics, Denali Therapeutics, Novartis AG, Fresenius Kabi AG, Akorn Incorporated, Teva Pharmaceutical, Mylan N.V, Johnsons & Johnsons, F. Hoffman-La Roche Ltd., Trinity Biotech plc, and others.
• In May 2021, Amicus Therapeutics announced the completion of a successful Type B Pre-Biologics License Application (BLA) meeting with the US Food and Drug Administration (FDA) for AT-GAA (cipaglucosidase alfa co-administered with miglustat), for the treatment of Pompe disease.
• In March 2021, Maze Therapeutics revealed its first three lead therapeutic candidates in the company’s wholly owned pipeline. One of the candidates includes an oral therapy targeting GYS1 for the treatment of Pompe disease, enabled by Maze’s COMPASS platform. The company expects to start clinical trials in the first half of 2022.
• On August 6, 2021, the U.S. Food and Drug Administration (FDA) approved Nexviazyme for intravenous infusion to treat patients 1 year of age and older with late-onset Pompe disease.
• The FORTIS clinical trial (NCT04174105), evaluating the gene therapy candidate AT845, restarted after a hold placed by the U.S. Food and Drug Administration (FDA) was lifted. AT845 aims to deliver a healthy version of the GAA gene, which is mutated in Pompe disease, to muscle cells. This therapy leads to increased production of a functional acid alpha-glucosidase (GAA) enzyme.

Pompe Disease Overview

Pompe disease, also known as glycogen storage disease type II, is a rare genetic disorder characterized by the buildup of glycogen in cells, particularly within muscles. This accumulation impairs the function of various organs, predominantly affecting the muscles, heart, and liver. Pompe disease is caused by mutations in the GAA gene, leading to deficiency or dysfunction of the enzyme acid alpha-glucosidase (GAA), which is responsible for breaking down glycogen. Symptoms can vary widely in severity and may include muscle weakness, respiratory difficulties, enlarged heart (cardiomegaly), and liver problems. Early onset Pompe disease typically presents in infancy, while late-onset forms may manifest later in childhood or adulthood. Treatment options include enzyme replacement therapy (ERT) to supplement the deficient enzyme and manage symptoms, along with supportive care to address specific complications. Regular monitoring and management by healthcare professionals are essential for optimizing outcomes in individuals with Pompe disease.

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Pompe Disease Pipeline Drugs

• Cipaglucosidase alfa – Amicus Therapeutics
• SPK-3006 – Spark Therapeutics
• AT845 – Audentes Therapeutics
• ACTUS 101 – Asklepios Biopharmaceutical
• RP A501 – Rocket Pharmaceuticals
• AVR-RD-03 – AVROBIO
• AOC-Pompe disease – Avidity Biosciences

Pompe Disease Therapeutic Assessment

The Pompe Disease Pipeline report lays down complete insights into active Pompe Disease pipeline therapies segmented by Stage, Product Type, Route of Administration, Molecule Type, Target, and Mechanism of Action.

By Product Type

• Mono
• Combination

By Stage

• Discovery
• Pre-clinical
• IND
• Phase I
• Phase II
• Phase III
• Pre-registration

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By Molecule Type

• Gene therapies

Recombinant fusion proteins

• Small interfering RNA
• Cell therapies
• Monoclonal antibodies

By Route of Administration

• Intravenous
• Parenteral

By Mechanism of Action

• Alpha-glucosidase replacements
• Glycogen synthase kinase modulators
• RNA interference
• Gene transference

By Targets

• Alpha-glucosidase
• Gene replacement
• Glycogen synthase kinase

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Scope of the Pompe Disease Pipeline Report

• Coverage: Global
• Key Pompe Disease Companies: Spark Therapeutics, Amicus Therapeutics, Asklepios Biopharmaceutical, Audentes Therapeutics, Rocket Pharmaceuticals, Avidity Biosciences, AVROBIO, Maze Therapeutics, Denali Therapeutics, Novartis AG, Fresenius Kabi AG, Akorn Incorporated, Teva Pharmaceutical, Mylan N.V, Johnsons & Johnsons, F. Hoffman-La Roche Ltd., Trinity Biotech plc, and others.
• Key Pompe Disease Pipeline Therapies: Cipaglucosidase alfa, SPK-3006, ACTUS 101, AT845, RP A501, AIM vectors, AVR RD 03, GYS1 Program, ETV-GAA, Research programme: glycogen storage disease type II therapy, AOC-Pompe disease, and others.

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Table of Contents

1. Report Introduction
2. Pompe Disease Disease Overview
3. Pompe Disease Pipeline Outlook
4. Comparative Analysis
5. Pompe Disease Therapeutic Products in Clinical Stage
6. Pompe Disease Late Stage Products (Phase III)
7. Pompe Disease Mid Stage Products (Phase II)
8. Pompe Disease Early Stage Products (Phase I)
9. Pompe Disease Therapeutic Products in Non-clinical Stage
10. Pompe Disease Preclinical and Discovery Stage Products
11. Pompe Disease – DelveInsight’s Analytical Perspective
12. In-depth Commercial Assessment
13. Pompe Disease Collaboration Deals
14. Pompe Disease Therapeutics Pipeline Analysis
15. Inactive Pompe Disease Pipeline Products
16. Pompe Disease Companies
17. Pompe Disease Unmet Needs
18. Pompe Disease Market Drivers and Barriers
19. Pompe Disease- Future Perspectives and Conclusion
20. Appendix
21. Report Methodology
22. Pompe Disease Consulting Services
23. Disclaimer
24. 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

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