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.
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
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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
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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