(Albany, USA) 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.
- On April 2024, Astellas Gene Therapies announced results of a Study to Evaluate Seroprevalence of Antibodies to AAV8 and Assessment of Biomarkers in Patients With Late-Onset Pompe Disease.
- On April 2024, Amicus Therapeutics announced results of an Open-Label, Fixed-Sequence, Ascending-Dose, First-in-Human Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of Intravenous Infusions of ATB200 Co-Administered With Oral AT2221 in Adult Subjects With Pompe Disease.
- On March 2024, Genzyme, a Sanofi Company announced results of a Single Arm, Prospective, Open-label, Multi-center Study to Evaluate Efficacy and Safety in Chinese Patients With Late Onset Pompe Disease With Alglucosidase Alfa Treatment.
- On November 2023, Spark Therapeutics, Inc announced results of a Phase 1/2, Dose-escalation Study to Evaluate the Safety, Tolerability and Efficacy of a Single Intravenous Infusion of SPK-3006 in Adults With Late-onset Pompe Disease.
- On June 2023, Asklepios Biopharmaceutical, Inc announced results of a Phase 1 Study of the Safety of AAV2/8-LSPhGAA (ACTUS-101) in Late-onset Pompe Disease.
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
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
Further Pompe Disease product details are provided in the report. Download the Pompe Disease pipeline report to learn more about the emerging Pompe Disease therapies @ Pompe Disease Therapeutics Assessment
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
- Report Introduction
- Pompe Disease Disease Overview
- Pompe Disease Pipeline Outlook
- Comparative Analysis
- Pompe Disease Therapeutic Products in Clinical Stage
- Pompe Disease Late Stage Products (Phase III)
- Pompe Disease Mid Stage Products (Phase II)
- Pompe Disease Early Stage Products (Phase I)
- Pompe Disease Therapeutic Products in Non-clinical Stage
- Pompe Disease Preclinical and Discovery Stage Products
- Pompe Disease – DelveInsight’s Analytical Perspective
- In-depth Commercial Assessment
- Pompe Disease Collaboration Deals
- Pompe Disease Therapeutics Pipeline Analysis
- Inactive Pompe Disease Pipeline Products
- Pompe Disease Companies
- Pompe Disease Unmet Needs
- Pompe Disease Market Drivers and Barriers
- Pompe Disease- Future Perspectives and Conclusion
- Appendix
- Report Methodology
- Pompe Disease Consulting Services
- Disclaimer
- About DelveInsight
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