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Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) - Drugs in Development, 2021

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    Drug Pipelines

  • March 2021
  • Region: Global
  • Global Markets Direct
  • ID: 5331194
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According to the recently published report 'Lysosomal Alpha Glucosidase - Drugs in Development, 2021'; Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) pipeline Target constitutes close to 19 molecules. Out of which approximately 16 molecules are developed by companies and remaining by the universities/institutes.

Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) - Lysosomal alpha-glucosidase is an enzyme encoded by the GAA gene. It is essential for the degradation of glygogen to glucose in lysosomes. Defects in this gene lead to glycogen storage disease II or Pompe disease.

The report 'Lysosomal Alpha Glucosidase - Drugs in Development, 2021' outlays comprehensive information on the Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) targeted therapeutics, complete with analysis by indications, stage of development, mechanism of action (MoA), route of administration (RoA) and molecule type; that are being developed by Companies / Universities.

It also reviews key players involved in Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) targeted therapeutics development with respective active and dormant or discontinued projects. Currently, The molecules developed by companies in Pre-Registration, Phase III, Phase II, Preclinical and Discovery stages are 1, 1, 3, 10 and 1 respectively. Similarly, the universities portfolio in Phase II, Phase I and Preclinical stages comprises 1, 1 and 1 molecules, respectively. Report covers products from therapy areas Metabolic Disorders which include indications Pompe Disease.

Note: Certain content/sections in the pipeline guide may be removed or altered based on the availability and relevance of data.

Scope


  • The report provides a snapshot of the global therapeutic landscape for Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20)
  • The report reviews Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) targeted therapeutics under development by companies and universities/research institutes based on information derived from company and industry-specific sources
  • The report covers pipeline products based on various stages of development ranging from pre-registration till discovery and undisclosed stages
  • The report features descriptive drug profiles for the pipeline products which includes, product description, descriptive MoA, R&D brief, licensing and collaboration details & other developmental activities
  • The report reviews key players involved in Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) targeted therapeutics and enlists all their major and minor projects
  • The report assesses Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) targeted therapeutics based on mechanism of action (MoA), route of administration (RoA) and molecule type
  • The report summarizes all the dormant and discontinued pipeline projects
  • The report reviews latest news and deals related to Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) targeted therapeutics

Reasons to Buy


  • Gain strategically significant competitor information, analysis, and insights to formulate effective R&D strategies
  • Identify emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage
  • Identify and understand the targeted therapy areas and indications for Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20)Identify the use of drugs for target identification and drug repurposing
  • Identify potential new clients or partners in the target demographic
  • Develop strategic initiatives by understanding the focus areas of leading companies
  • Plan mergers and acquisitions effectively by identifying key players and it’s most promising pipeline therapeutics
  • Devise corrective measures for pipeline projects by understanding Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) development landscape
  • Develop and design in-licensing and out-licensing strategies by identifying prospective partners with the most attractive projects to enhance and expand business potential and scope

Table of Contents

Introduction
  • Report Coverage

Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) - Overview
Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) - Therapeutics Development
  • Products under Development by Stage of Development
  • Products under Development by Therapy Area
  • Products under Development by Indication
  • Products under Development by Companies
  • Products under Development by Universities/Institutes

Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) - Therapeutics Assessment
  • Assessment by Mechanism of Action
  • Assessment by Route of Administration
  • Assessment by Molecule Type

Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) - Companies Involved in Therapeutics Development
  • Amicus Therapeutics Inc
  • Asklepios BioPharmaceutical Inc
  • Audentes Therapeutics Inc
  • AVROBIO Inc
  • Denali Therapeutics Inc
  • eleva GmbH
  • Etubics Corp
  • Genzyme Corp
  • JCR Pharmaceuticals Co Ltd
  • M6P Therapeutics
  • NanoMedSyn SAS
  • Oxyrane Belgium NV
  • Pharming Group NV
  • Spark Therapeutics Inc

Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) - Drug Profiles
(cipaglucosidase alfa + miglustat) - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress

ACTUS-101 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress

AT-845 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress

avalglucosidase alfa - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress

AVRRD-03 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress

Fusion Protein to Replace GAA for Pompe Disease - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress

Gene Therapy 1 to Activate Acid Alpha-Glucosidase for Pompe Disease - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress

Gene Therapy 2 to Activate Acid Alpha-Glucosidase for Pompe Disease - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress

Gene Therapy to Activate Acid Alpha-Glucosidase for Pompe Disease - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress

Gene Therapy to Activate GAA for Pompe Disease - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress

Gene Therapy to Activate GAA for Pompe Disease - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress

JR-162 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress

M-021 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress

M-023 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress

OXY-2810 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress

PGN-004 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress

Recombinant Alpha Glucosidase Replacement for Pompe Disease - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress

RPV-002 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress

SPK-3006 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) - Dormant Products
  • Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) - Discontinued Products

Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) - Product Development Milestones
  • Featured News & Press Releases
  • Mar 15, 2021: Amicus Therapeutics announces presentation and posters at the 2021 MDA Clinical & Scientific Conference
  • Feb 11, 2021: Amicus’ AT-GAA shows clinically meaningful & significant improvements in both musculoskeletal and respiratory measures in late-onset Pompe disease compared to standard of care in pivotal phase 3 PROPEL study
  • Feb 02, 2021: Data presented at WORLDSymposium reinforces robust rare disease pipeline and highlights additional clinical data for investigational avalglucosidase alfa in Pompe disease
  • Feb 01, 2021: Spark Therapeutics announces first participant dosed in phase 1/2 study of investigational gene therapy for late-onset pompe disease
  • Feb 01, 2021: AVROBIO announces clinical data presentation on AVRRD-03 at 17th Annual WORLDSymposium 2021
  • Jan 20, 2021: Sanofi announced that it has submitted a new drug application in Japan for avalglucosidase alfa, an investigational enzyme replacement therapy
  • Dec 01, 2020: Amicus Therapeutics initiates rolling Biologic License Application to the U.S. Food and Drug Administration for AT-GAA in late-onset Pompe disease
  • Nov 18, 2020: FDA grants priority review for avalglucosidase alfa, a potential new therapy for Pompe disease
  • Oct 02, 2020: EMA accepts regulatory submission for avalglucosidase alfa, a potentially new standard of care enzyme replacement therapy for Pompe disease
  • Jun 16, 2020: Sanofi’s investigational enzyme replacement therapy shows clinically meaningful improvement in critical manifestations of late-onset Pompe disease
  • Jun 08, 2020: Sanofi to present Phase 3 results of avalglucosidase alfa in patients with late-onset Pompe disease
  • May 14, 2020: AVROBIO presents new preclinical data on lentiviral gene therapy program for Pompe Disease at ASGCT 2020
  • May 05, 2020: Amicus Therapeutics announces presentations on its pompe disease gene therapy at the American Society of Gene & Cell Therapy 23rd Annual Meeting
  • May 05, 2020: AVROBIO to present preclinical data at upcoming American Society of Gene & Cell Therapy (ASGCT) annual meeting, May 12-15, 2020
  • Feb 07, 2020: Amicus Therapeutics announces presentation on its Pompe Disease gene therapy at 16th Annual WORLDSymposium 2020

Appendix
  • Methodology
  • Coverage
  • Secondary Research
  • Primary Research
  • Expert Panel Validation
  • Contact the Publisher
  • Disclaimer

List of Tables
  • Number of Products under Development by Stage of Development, 2021
  • Number of Products under Development by Therapy Areas, 2021
  • Number of Products under Development by Indication, 2021
  • Number of Products under Development by Companies, 2021
  • Products under Development by Companies, 2021
  • Products under Development by Companies, 2021 (Contd..1)
  • Number of Products under Investigation by Universities/Institutes, 2021
  • Products under Investigation by Universities/Institutes, 2021
  • Number of Products by Stage and Mechanism of Actions, 2021
  • Number of Products by Stage and Route of Administration, 2021
  • Number of Products by Stage and Molecule Type, 2021
  • Pipeline by Amicus Therapeutics Inc, 2021
  • Pipeline by Asklepios BioPharmaceutical Inc, 2021
  • Pipeline by Audentes Therapeutics Inc, 2021
  • Pipeline by AVROBIO Inc, 2021
  • Pipeline by Denali Therapeutics Inc, 2021
  • Pipeline by eleva GmbH, 2021
  • Pipeline by Etubics Corp, 2021
  • Pipeline by Genzyme Corp, 2021
  • Pipeline by JCR Pharmaceuticals Co Ltd, 2021
  • Pipeline by M6P Therapeutics, 2021
  • Pipeline by NanoMedSyn SAS, 2021
  • Pipeline by Oxyrane Belgium NV, 2021
  • Pipeline by Pharming Group NV, 2021
  • Pipeline by Spark Therapeutics Inc, 2021
  • Dormant Projects, 2021
  • Discontinued Products, 2021

List of Figures
  • Number of Products under Development by Stage of Development, 2021
  • Number of Products by Mechanism of Actions, 2021
  • Number of Products by Stage and Mechanism of Actions, 2021
  • Number of Products by Routes of Administration, 2021
  • Number of Products by Stage and Routes of Administration, 2021
  • Number of Products by Molecule Types, 2021
  • Number of Products by Stage and Molecule Types, 2021

Companies Mentioned (Partial List)

A selection of companies mentioned in this report includes, but is not limited to:

  • Amicus Therapeutics Inc
  • Asklepios BioPharmaceutical Inc
  • Audentes Therapeutics Inc
  • AVROBIO Inc
  • Denali Therapeutics Inc
  • eleva GmbH
  • Etubics Corp
  • Genzyme Corp
  • JCR Pharmaceuticals Co Ltd
  • M6P Therapeutics
  • NanoMedSyn SAS
  • Oxyrane Belgium NV
  • Pharming Group NV
  • Spark Therapeutics Inc