Pompe Disease Pipeline Update 2024: FDA Approvals, Therapeutic Advancements, and Market Dynamics, MOA, ROA by DelveInsight | M6P Therapeutics, Entrada Therapeutics, Maze Therapeutics, Astellas

“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: 

• 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 – Asklepios Biopharmaceutical, GeneCradle Inc, Astellas Gene Therapies, Amicus Therapeutics, M6P Therapeutics, Entrada Therapeutics, Maze Therapeutics, Astellas Gene Therapies, Spark Therapeutics, and others, are developing therapies for the Pompe Disease treatment 

• Pompe Disease Emerging therapies such as – ACTUS-101, GC301, zocaglusagene nuzaparvovec, AT2221, 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 May 2024, Shionogi & Co., Ltd. (Osaka, Japan; CEO: Isao Teshirogi, Ph.D.) and Maze Therapeutics, Inc. (South San Francisco, California, USA; CEO: Jason Coloma, Ph.D.) have announced the completion of an exclusive global licensing agreement for MZE001. MZE001 is an investigational oral glycogen synthase 1 (GYS1) inhibitor designed to address Pompe disease by reducing the buildup of disease-causing glycogen.

• In November 2023, Aro Biotherapeutics, a clinical-stage biotech company, has secured $41.5 million in a Series B funding round to further develop its lead candidate, ABX1100, for the treatment of Pompe disease. The round was led by Cowen Healthcare Investments, with participation from Series A investors including Northpond Ventures, Johnson & Johnson Innovation – JJDC, BVF Partners, and HealthCap.

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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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.

Emerging Pompe Disease Drugs Under Different Phases of Clinical Development Include:

• ACTUS-101: Asklepios Biopharmaceutical

• GC301: GeneCradle Inc

• zocaglusagene nuzaparvovec: Astellas Gene Therapies

• AT2221: Amicus Therapeutics

• 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

Download Sample PDF Report to know more about Pompe Disease drugs and therapies

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.

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 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: Asklepios Biopharmaceutical, GeneCradle Inc, Astellas Gene Therapies, Amicus Therapeutics, M6P Therapeutics, Entrada Therapeutics, Maze Therapeutics, Astellas Gene Therapies, Spark Therapeutics, and others

• Key Pompe Disease Therapies: ACTUS-101, GC301, zocaglusagene nuzaparvovec, AT2221, 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 

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Table of Contents

*The Table of Contents (TOC) is not exhaustive; the final content may vary. Refer to the sample report for the complete table of contents.

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. It also offers Healthcare Consulting Services, which benefits in market analysis to accelerate business growth and overcome challenges with a practical approach.

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