Pompe Disease Pipeline Report 2023: Treatment Drugs, Therapies, FDA Approvals and Companies by DelveInsight | Spark Therapeutics, Amicus Therapeutics, Asklepios Biopharmaceutical, Audentes Therapeutics, Rocket Pharmaceuticals, Avidity Biosciences, AVROBIO

(Albany, USA) DelveInsight's, "Pompe Disease Pipeline Insights, 2023," 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 17+ key players and 17+ 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.
Pompe Disease Overview
Pompe Disease is a rare genetic disease that is a result of a build-up of a complex sugar, glycogen in the cells of the body. It exhibits variable rates of progression and different ages of onset. The cause of the disease is a deficiency of an enzyme called acid alfa glucosidase (GAA), which breaks downs complex sugars in the body.
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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

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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

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