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Published: May, 2017 | Pages:
60 | Publisher: Global Markets Direct
Industry: Pharmaceuticals & Healthcare | Report Format: Electronic (PDF)
Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) - Pipeline Review, H1 2017 Summary Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) pipeline Target constitutes close to 15 molecules. Out of which approximately 12 molecules are developed by companies and remaining by the universities/institutes. The latest report Lysosomal Alpha Glucosidase - Pipeline Review, H1 2017, 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. 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 molecules developed by companies in Phase III, Phase II, IND/CTA Filed, Preclinical and Discovery stages are 1, 1, 1, 7 and 2 respectively. Similarly, the universities portfolio in Phase II and Preclinical stages comprises 1 and 2 molecules, respectively. Report covers products from therapy areas Metabolic Disorders which include indications Pompe Disease. Furthermore, this report 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. Driven by data and information sourced from proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources. 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
List of Tables List of Figures Introduction Global Markets Direct 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 Audentes Therapeutics Inc Etubics Corp Genzyme Corp greenovation Biotech GmbH Huons Co Ltd JCR Pharmaceuticals Co Ltd Oxyrane Belgium NV Pharming Group NV Sarepta Therapeutics Inc Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) - Drug Profiles Antisense Oligonucleotide to Activate Lysosomal Alpha-Glucosidase for Pompe Disease - Drug Profile Product Description Mechanism Of Action R&D Progress AT-982 - Drug Profile Product Description Mechanism Of Action R&D Progress ATB-200 + miglustat - 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 Acid Alpha-Glucosidase for Pompe Disease - Drug Profile Product Description Mechanism Of Action R&D Progress GZ-402666 - Drug Profile Product Description Mechanism Of Action R&D Progress HU-023 - Drug Profile Product Description Mechanism Of Action R&D Progress JR-162 - Drug Profile Product Description Mechanism Of Action R&D Progress MOSS-GAA - 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 VAL-1221 - 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 May 15, 2017: Amicus Therapeutics Announces Positive Functional Data from Initial Patients in Pompe Phase 1/2 Study Mar 03, 2017: Valerion Therapeutics Demonstrates a New Mechanism for Treating Pompe Disease Feb 15, 2017: Amicus Therapeutics Presents Important New Scientific Findings and Preclinical Data for Pompe Program at WORLDSymposium 2017 Dec 08, 2016: Amicus Therapeutics Announces Positive Preliminary Data from Phase 1/2 Study of Novel Treatment Paradigm for Pompe Disease Nov 17, 2016: Pivotal Phase 3 Trial of NeoGAA Investigational Second-Generation Therapy for Pompe Disease to Begin in the UK Nov 04, 2016: Sanofi Genzyme Begins Pivotal Phase 3 Trial of NeoGAA Investigational Second-Generation Therapy for Pompe Disease Mar 03, 2016: Sanofi Genzyme Presents Results from Phase 1/2 Study of Investigational Second-Generation Therapy for Pompe Disease Mar 01, 2016: Amicus Therapeutics Highlights Preclinical Pompe Data at WORLDSymposium 2016 Feb 10, 2016: Amicus Therapeutics Announces Oral and Poster Presentations on Chaperone AT2221 at 12th Annual WORLDSymposium 2016 Jan 11, 2016: Amicus Therapeutics Provides Update On Novel ERT for Pompe Disease (ATB200 + Chaperone) Dec 22, 2015: Amicus Therapeutics Commences Phase 1/2 Study of Novel ERT for Treatment of Pompe Disease Sep 03, 2015: Valerion Therapeutics Announces Positive Results Of Non-Clinical Research Study at SSIEM Annual Symposium May 11, 2015: Audentes Therapeutics To Present New Data On AT-002 at American Society of Gene and Cell Therapy Annual Meeting Feb 10, 2015: Amicus Therapeutics Presents Preclinical Data on Next-Generation Pompe ERT at WORLD Symposium 2015 Appendix Methodology Coverage Secondary Research Primary Research Expert Panel Validation Contact Us Disclaimer
List of Tables
Number of Products under Development by Stage of Development, H1 2017 Number of Products under Development by Therapy Areas, H1 2017 Number of Products under Development by Indication, H1 2017 Number of Products under Development by Companies, H1 2017 Products under Development by Companies, H1 2017 Number of Products under Investigation by Universities/Institutes, H1 2017 Products under Investigation by Universities/Institutes, H1 2017 Number of Products by Stage and Mechanism of Actions, H1 2017 Number of Products by Stage and Route of Administration, H1 2017 Number of Products by Stage and Molecule Type, H1 2017 Pipeline by Amicus Therapeutics Inc, H1 2017 Pipeline by Audentes Therapeutics Inc, H1 2017 Pipeline by Etubics Corp, H1 2017 Pipeline by Genzyme Corp, H1 2017 Pipeline by greenovation Biotech GmbH, H1 2017 Pipeline by Huons Co Ltd, H1 2017 Pipeline by JCR Pharmaceuticals Co Ltd, H1 2017 Pipeline by Oxyrane Belgium NV, H1 2017 Pipeline by Pharming Group NV, H1 2017 Pipeline by Sarepta Therapeutics Inc, H1 2017 Dormant Projects, H1 2017 Discontinued Products, H1 2017
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