Pompe Disease Pipeline Drugs Analysis Report (2024 Updates): FDA Approvals, Clinical Trials, Therapies, Mechanism of Action, Route of Administration by DelveInsight | M6P Therapeutics, Entrada Therapeutics, Maze Therapeutics, Astellas Gene Therapies, Askl

Las Vega (Nevada), United States //- As per DelveInsight's assessment, globally, Pompe Disease pipeline constitutes 15+ key companies continuously working towards developing 20+ Pompe Disease treatment therapies, analysis of Clinical Trials, Therapies, Mechanism of Action, Route of Administration, and Developments analyzes DelveInsight.

"Pompe Disease Pipeline Insight, 2024" report by DelveInsight outlines comprehensive insights into the present clinical development scenario and growth prospects across the Pompe Disease Market.

The Pompe Disease Pipeline report embraces in-depth commercial and clinical assessment of the pipeline products from the pre-clinical developmental phase to the marketed phase. The report also covers a detailed description of the drug, including the mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, collaborations, mergers acquisition, funding, designations, and other product-related details.

Some of the key takeaways from the Pompe Disease Pipeline Report: https://www.delveinsight.com/sample-request/pompe-disease-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=gpr
• Companies across the globe are diligently working toward developing novel Pompe Disease treatment therapies with a considerable amount of success over the years. Pompe Disease Key players such as - M6P Therapeutics, Entrada Therapeutics, Maze Therapeutics, Astellas Gene Therapies, Asklepios Biopharmaceutical, Spark Therapeutics, Amicus Therapeutics, and others, are developing therapies for the Pompe Disease treatment
• Pompe Disease Emerging therapies such as - M 021, GYS1 Knockdown Oligonucleotide, MZE 001, AT845, AAV2/8LSPhGAA, SPK-3006, Cipaglucosidase alfa, and others are expected to have a significant impact on the Pompe Disease market in the coming years.
• In February 2020, Amicus Therapeutics initiated an open-label study of the safety, pharmacokinetics, efficacy, pharmacodynamics, and immunogenicity of ATB200/AT2221 in pediatric subjects aged0 to < 18 years with Pompe Disease.
• In May 2024, Shionogi & Co., Ltd. (headquartered in Osaka, Japan, and led by CEO Isao Teshirogi, Ph.D.) and Maze Therapeutics, Inc. (headquartered in South San Francisco, California, USA, and led by CEO Jason Coloma, Ph.D.) have announced the completion of an exclusive global license agreement for MZE001. This investigational oral glycogen synthase 1 (GYS1) inhibitor is designed to combat Pompe disease by reducing the harmful accumulation of glycogen.
• In October 2023, Aro Biotherapeutics has initiated the Phase I clinical trial for ABX1100, aimed at treating Pompe disease, by enrolling its first participant. The trial, which is taking place in Canada, features a double-blind, placebo-controlled, single ascending dose design to study ABX1100 in healthy volunteers. The primary objectives are to assess the drug's tolerability and safety, along with its pharmacokinetics and various pharmacodynamic biomarkers to confirm target engagement.

Pompe Disease Overview
Pompe disease belongs to the 'lysosomal storage disorders' (LSDs) group of diseases. Within the cells, lysosomes are tiny compartments where all sorts of substances are recycled. By the action of digestive enzymes, the compounds are degraded. There are currently more than 50 different LSDs that are considered to be caused by the deficiency of one of these enzymes.

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Emerging Pompe Disease Drugs Under Different Phases of Clinical Development Include:
• M 021: M6P Therapeutics
• GYS1 Knockdown Oligonucleotide: Entrada Therapeutics
• MZE 001: Maze Therapeutics
• AT845: Astellas Gene Therapies
• AAV2/8LSPhGAA: Asklepios Biopharmaceutical
• SPK-3006: Spark Therapeutics
• Cipaglucosidase alfa: Amicus Therapeutics

Route of Administration
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
• Parenteral
• Intravenous
• Subcutaneous
• Topical

Molecule Type
Products have been categorized under various Molecule types, such as
• Monoclonal Antibody
• Peptides
• Polymer
• Small molecule
• Gene therapy

Pompe Disease Pipeline Therapeutics Assessment
• Pompe Disease Assessment by Product Type
• Pompe Disease By Stage and Product Type
• Pompe Disease Assessment by Route of Administration
• Pompe Disease By Stage and Route of Administration
• Pompe Disease Assessment by Molecule Type
• Pompe Disease by Stage and Molecule Type

DelveInsight's Pompe Disease Report covers around 20+ products under different phases of clinical development like
• Late-stage products (Phase III)
• Mid-stage products (Phase II)
• Early-stage product (Phase I)
• Pre-clinical and Discovery stage candidates
• Discontinued & Inactive candidates
• Route of Administration

Some of the key companies in the Pompe Disease Therapeutics Market include:
Key companies developing therapies for Pompe Disease are - Spark Therapeutics, Amicus Therapeutics, Asklepios Biopharmaceutical, Audentes Therapeutics, Rocket Pharmaceuticals, Avidity Biosciences, AVROBIO, Maze Therapeutics, Denali Therapeutics, and others.

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
https://www.delveinsight.com/sample-request/pompe-disease-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=gpr

Pompe Disease Pipeline Analysis:
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 key 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.
The report is built using data and information traced from the researcher's proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations, and featured press releases from company/university websites and industry-specific third-party sources, etc.

Download Sample PDF Report to know more about Pompe Disease drugs and therapies
https://www.delveinsight.com/sample-request/pompe-disease-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=gpr

Pompe Disease Pipeline Market Drivers
• Improving Healthcare Infrastructure
• Demand for New and Effective Drugs
• Increasing research and development in the gene therapy and enzyme replacement therapy areas

Pompe Disease Pipeline Market Barriers
• Lack Of Cost-Effective Treatment

Scope of Pompe Disease Pipeline Drug Insight
• Coverage: Global
• Key Pompe Disease Companies: M6P Therapeutics, Entrada Therapeutics, Maze Therapeutics, Astellas Gene Therapies, Asklepios Biopharmaceutical, Spark Therapeutics, Amicus Therapeutics, and others
• Key Pompe Disease Therapies: M 021, GYS1 Knockdown Oligonucleotide, MZE 001, AT845, AAV2/8LSPhGAA, SPK-3006, Cipaglucosidase alfa, and others
• Pompe Disease Therapeutic Assessment: Pompe Disease current marketed and Pompe Disease emerging therapies
• Pompe Disease Market Dynamics: Pompe Disease market drivers and Pompe Disease market barriers

Request for Sample PDF Report for Pompe Disease Pipeline Assessment and clinical trials
https://www.delveinsight.com/sample-request/pompe-disease-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=gpr

Table of Contents
1 Pompe Disease Report Introduction
2 Pompe Disease Executive Summary
3 Pompe Disease Overview
4 Pompe Disease- Analytical Perspective In-depth Commercial Assessment
5 Pompe Disease Pipeline Therapeutics
6 Pompe Disease Late Stage Products (Phase II/III)
7 Pompe Disease Mid Stage Products (Phase II)
8 Pompe Disease Early Stage Products (Phase I)
9 Pompe Disease Preclinical Stage Products
10 Pompe Disease Therapeutics Assessment
11 Pompe Disease Inactive Products
12 Company-University Collaborations (Licensing/Partnering) Analysis
13 Pompe Disease Key Companies
14 Pompe Disease Key Products
15 Pompe Disease Unmet Needs
16 Pompe Disease Market Drivers and Barriers
17 Pompe Disease Future Perspectives and Conclusion
18 Pompe Disease Analyst Views
19 Appendix
20 About DelveInsight
*The Table of Contents (TOC) is not exhaustive; the final content may vary. Refer to the sample report for the complete table of contents.

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