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:
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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.
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
AT845
Audentes Therapeutics
Gene transference
ACTUS 101
Asklepios Biopharmaceutical
RP A501
Rocket Pharmaceuticals
Phase I
NA
AVR-RD-03
AVROBIO
Preclinical
Alpha-glucosidase stimulants
AOC-Pompe disease
Avidity Biosciences
Discovery
Glycogen synthase kinase modulators
Parenteral
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
By Stage
By Molecule Type
By Route of Administration
By Mechanism of Action
By Targets
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
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