DelveInsight Estimates a Promising Picture of Pompe Disease Pipeline Landscape in the Coming Years as Key Pharma Companies Bolster Rare Disease Pipeline

The Pompe Disease Pipeline is evolving with novel mechanisms of action (MoAs) that will bring positive changes in the disease pipeline landscape.


Los Angeles, USA, Sept. 15, 2021 (GLOBE NEWSWIRE) -- DelveInsight Estimates a Promising Picture of Pompe Disease Pipeline Landscape in the Coming Years as Key Pharma Companies Bolster Rare Disease Pipeline 

The Pompe Disease Pipeline is evolving with novel mechanisms of action (MoAs) that will bring positive changes in the disease pipeline landscape.

DelveInsight’s Pompe Disease Pipeline Insights report offers comprehensive coverage of the emerging Pompe Disease therapeutics landscape in different stages of clinical development from pre-clinical till a late-end stage, along with dormant, inactive and abandoned drugs agents. 

The Pompe Disease Pipeline report provides a complete outlook of the clinical trials, colloborations happening in the domain, recent breakthroughs and growth prospects across the Pompe Disease domain.

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 among 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.

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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.

For more information on emerging drugs, visit Pompe Disease Pipeline Analysis 

Pompe Disease Pipeline Drugs 

DrugCompanyPhaseMoARoA
Cipaglucosidase alfa Amicus TherapeuticsPreregistrationAlpha-glucosidase replacementsIntravenous
SPK-3006Spark TherapeuticsPhase I/IIAlpha-glucosidase expression stimulantsIntravenous
AT845Audentes
Therapeutics
Phase I/IIGene transferenceIntravenous
ACTUS 101Asklepios BiopharmaceuticalPhase I/IIGene transferenceIntravenous
RP A501Rocket PharmaceuticalsPhase IGene transferenceNA
AVR-RD-03AVROBIOPreclinicalAlpha-glucosidase stimulantsNA
AOC-Pompe diseaseAvidity BiosciencesDiscoveryGlycogen synthase kinase modulatorsParenteral

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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

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 Players: Spark Therapeutics, Amicus Therapeutics, Asklepios Biopharmaceutical, Audentes Therapeutics, Rocket Pharmaceuticals, Abeona Therapeutics, AVROBIO, Maze Therapeutic, Denali Therapeutics, Immusoft, Avidity Biosciences, 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.

Reach out @ Pompe Disease Pipeline: Novel therapies and Emerging technologies 

Table of Contents 

1Report Introduction
2Pompe Disease Disease Overview
3Pompe Disease Pipeline Outlook
4 Comparative Analysis
5Pompe Disease Therapeutic Products in Clinical Stage
6Pompe Disease Late Stage Products (Phase III)
7Pompe Disease Mid Stage Products (Phase II)
8Pompe Disease Early Stage Products (Phase I)
9Pompe Disease Therapeutic Products in Non-clinical Stage
10Pompe Disease Preclinical and Discovery Stage Products
11Pompe Disease – DelveInsight’s Analytical Perspective
12In-depth Commercial Assessment
13Pompe Disease Collaboration Deals
14Pompe Disease Therapeutics Pipeline Analysis
15Inactive Pompe Disease Pipeline Products 
16Pompe Disease Key Companies
17Pompe Disease- Unmet Needs
18Pompe Disease Market Drivers and Barriers
19Pompe Disease- Future Perspectives and Conclusion
20Appendix
21 Report Methodology
22Consulting Services
23Disclaimer
24About DelveInsight

Visit to know more of what’s covered @ Pompe Disease Emerging Therapies, Treatments and Ongoing Clinical Trials 

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