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Pompe Disease Pipeline: Industry Analysis, Drugs, Pipeline, Treatment and Key Companies by DelveInsight

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Pompe Disease Pipeline: Industry Analysis, Drugs, Pipeline, Treatment and Key Companies by DelveInsight

October 08
22:10 2021
Pompe Disease Pipeline: Industry Analysis, Drugs, Pipeline, Treatment and Key Companies by DelveInsight
Pompe Disease Pipeline

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.

 

Request for Sample [email protected] https://www.delveinsight.com/sample-request/pompe-disease-pipeline-insight

 

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.

 

Pompe Disease Pipeline Drugs

Drug

Company

Phase

MoA

RoA

Cipaglucosidase alfa

Amicus Therapeutics

Preregistration

Alpha-glucosidase replacements

Intravenous

SPK-3006

Spark Therapeutics

Phase I/II

Alpha-glucosidase expression stimulants

Intravenous

AT845

Audentes Therapeutics

Phase I/II

Gene transference

Intravenous

ACTUS 101

Asklepios Biopharmaceutical

Phase I/II

Gene transference

Intravenous

RP A501

Rocket Pharmaceuticals

Phase I

Gene transference

NA

AVR-RD-03

AVROBIO

Preclinical

Alpha-glucosidase stimulants

NA

AOC-Pompe disease

Avidity Biosciences

Discovery

Glycogen synthase kinase modulators

Parenteral

 

Request for Sample [email protected] https://www.delveinsight.com/sample-request/pompe-disease-pipeline-insight

 

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

 

Request for Sample [email protected] https://www.delveinsight.com/sample-request/pompe-disease-pipeline-insight

 

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.

 

Table of Contents

1

Report Introduction

2

Pompe Disease Disease Overview

3

Pompe Disease Pipeline Outlook

4

Comparative Analysis

5

Pompe Disease Therapeutic Products in Clinical Stage

6

Pompe Disease Late Stage Products (Phase III)

7

Pompe Disease Mid Stage Products (Phase II)

8

Pompe Disease Early Stage Products (Phase I)

9

Pompe Disease Therapeutic Products in Non-clinical Stage

10

Pompe Disease Preclinical and Discovery Stage Products

11

Pompe Disease – DelveInsight’s Analytical Perspective

12

In-depth Commercial Assessment

13

Pompe Disease Collaboration Deals

14

Pompe Disease Therapeutics Pipeline Analysis

15

Inactive Pompe Disease Pipeline Products

16

Pompe Disease Key Companies

17

Pompe Disease- Unmet Needs

18

Pompe Disease Market Drivers and Barriers

19

Pompe Disease- Future Perspectives and Conclusion

20

Appendix

21

Report Methodology

22

Consulting Services

23

Disclaimer

24

About DelveInsight

 

About Delveinsight: 

DelveInsight Business Research is a leading Market Research, and Business Consultant focused purely on Healthcare. It helps pharma companies by providing them with end-to-end services to solve their business problems.

Get hold of all the Pharma and healthcare market research reports on our market research subscription-based platform PharmDelve.

Media Contact
Company Name: DelveInsight Business Research LLP
Contact Person: Ankit Nigam
Email: Send Email
Phone: +19193216187
Address:304 S. Jones Blvd #2432
City: Albany
State: New York
Country: United States
Website: https://www.delveinsight.com/report-store/pompe-disease-pipeline-insight

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