(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
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