Alpha L-Iduronidase (IDUA or EC 3.2.1.76) - Pipeline Review, H2 2016

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Alpha L-Iduronidase (IDUA or EC 3.2.1.76) - Pipeline Review, H2 2016 Summary Global Markets Directs, Alpha L-Iduronidase (IDUA or EC 3.2.1.76) - Pipeline Review, H2 2016?, provides in depth analysis on Alpha L-Iduronidase (IDUA or EC 3.2.1.76) targeted pipeline therapeutics. The report provides comprehensive information on the Alpha L-Iduronidase (IDUA or EC 3.2.1.76), targeted therapeutics, complete with analysis by indications, stage of development, mechanism of action (MoA), route of administration (RoA) and molecule type. The report also covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases. Additionally, the report provides an overview of key players involved in Alpha L-Iduronidase (IDUA or EC 3.2.1.76) targeted therapeutics development and features dormant and discontinued projects. Global Markets Directs report features investigational drugs from across globe covering over 20 therapy areas and nearly 3,000 indications. The report is built using data and information sourced from Global Markets Directs 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. Drug profiles featured in the report undergoes periodic review following a stringent set of processes to ensure that all the profiles are updated with the latest set of information. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis. The report helps in identifying and tracking emerging players in the market and their portfolios, enhances decision making capabilities and helps to create effective counter strategies to gain competitive advantage. Note: *Certain sections in the report may be removed or altered based on the availability and relevance of data. *Updated report will be delivered in 48 hours of order confirmation. Scope - The report provides a snapshot of the global therapeutic landscape for Alpha L-Iduronidase (IDUA or EC 3.2.1.76) - The report reviews Alpha L-Iduronidase (IDUA or EC 3.2.1.76) 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 Alpha L-Iduronidase (IDUA or EC 3.2.1.76) targeted therapeutics and enlists all their major and minor projects - The report assesses Alpha L-Iduronidase (IDUA or EC 3.2.1.76) 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 Alpha L-Iduronidase (IDUA or EC 3.2.1.76) 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 Alpha L-Iduronidase (IDUA or EC 3.2.1.76) - 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 its most promising pipeline therapeutics - Devise corrective measures for pipeline projects by understanding Alpha L-Iduronidase (IDUA or EC 3.2.1.76) 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 Table of Contents 2 List of Tables 4 List of Figures 4 Introduction 5 Global Markets Direct Report Coverage 5 Alpha L-Iduronidase (IDUA or EC 3.2.1.76) Overview 6 Therapeutics Development 7 Alpha L-Iduronidase (IDUA or EC 3.2.1.76) - Products under Development by Stage of Development 7 Alpha L-Iduronidase (IDUA or EC 3.2.1.76) - Products under Development by Therapy Area 8 Alpha L-Iduronidase (IDUA or EC 3.2.1.76) - Products under Development by Indication 9 Alpha L-Iduronidase (IDUA or EC 3.2.1.76) - Pipeline Products Glance 10 Late Stage Products 10 Early Stage Products 11 Alpha L-Iduronidase (IDUA or EC 3.2.1.76) - Products under Development by Companies 12 Alpha L-Iduronidase (IDUA or EC 3.2.1.76) - Products under Development by Universities/Institutes 14 Alpha L-Iduronidase (IDUA or EC 3.2.1.76) - Therapeutics Assessment 16 Assessment by Monotherapy/Combination Products 16 Assessment by Mechanism of Action 17 Assessment by Route of Administration 19 Assessment by Molecule Type 21 Alpha L-Iduronidase (IDUA or EC 3.2.1.76) - Companies Involved in Therapeutics Development 23 AngioChem Inc. 23 ArmaGen Inc. 24 Bioasis Technologies Inc. 25 RegenxBio Inc. 26 Sangamo BioSciences, Inc. 27 Alpha L-Iduronidase (IDUA or EC 3.2.1.76) - Drug Profiles 28 AGT-181 - Drug Profile 28 Product Description 28 Mechanism Of Action 28 R&D Progress 28 Cell Therapy for Mucopolysaccharidosis I - Drug Profile 30 Product Description 30 Mechanism Of Action 30 R&D Progress 30 Cell Therapy to Activate Alpha L-Iduronidase for Hurler Syndrome - Drug Profile 31 Product Description 31 Mechanism Of Action 31 R&D Progress 31 Recombinant Alpha-L-Iduronidase Replacement for Hurler Syndrome - Drug Profile 32 Product Description 32 Mechanism Of Action 32 R&D Progress 32 Recombinant Alpha-L-Iduronidase Replacement for Mucopolysaccharidosis I - Drug Profile 33 Product Description 33 Mechanism Of Action 33 R&D Progress 33 Recombinant Alpha-L-Iduronidase Replacement for Mucopolysaccharidosis I - Drug Profile 34 Product Description 34 Mechanism Of Action 34 R&D Progress 34 RGX-111 - Drug Profile 35 Product Description 35 Mechanism Of Action 35 R&D Progress 35 SB-318 - Drug Profile 37 Product Description 37 Mechanism Of Action 37 R&D Progress 37 X-372 - Drug Profile 39 Product Description 39 Mechanism Of Action 39 R&D Progress 39 Alpha L-Iduronidase (IDUA or EC 3.2.1.76) - Dormant Projects 40 Alpha L-Iduronidase (IDUA or EC 3.2.1.76) - Featured News & Press Releases 41 Jul 05, 2016: REGENXBIO Provides Update On Gene Therapy Development Program RGX-111 41 May 09, 2016: Sangamo BioSciences Presents Recent Developments From Research And ZFP Therapeutic Programs In Multiple Presentations At Annual Meeting of the American Society of Gene and Cell Therapy 41 Mar 31, 2016: ArmaGen Announces Initiation of Phase 2 Proof-of-Concept Clinical Trial in Brazil to Study AGT-181 for the Treatment of Hurler Syndrome 43 Feb 08, 2016: Sangamo BioSciences Announces FDA Clearance Of Investigational New Drug Application For ZFN-Mediated Genome Editing Treatment Of MPS I 44 Dec 30, 2015: FDA Grants Rare Pediatric Disease Designation to REGENXBIO RGX-111 Gene Therapy for the Treatment of Mucopolysaccharidosis Type I (MPS I) 45 Nov 05, 2015: ArmaGen Receives Rare Pediatric Disease Designation from FDA for AGT-181 for the Potential Treatment of Hurler Syndrome 45 Oct 01, 2015: FDA Grants Orphan Drug Designation to REGENXBIO's RGX-111 Gene Therapy for the Treatment of Mucopolysaccharidosis Type I 46 Sep 02, 2015: ArmaGen Announces First Patient Dosed in Phase 1/2a Clinical Trial of AGT-181 for the Treatment of Hurler Syndrome 46 Apr 08, 2015: ArmaGen Announces FDA Acceptance of IND Application for AGT-181 for the Treatment of Hurler Syndrome 47 Mar 10, 2015: PacificGMP and ArmaGen Announce Successful Manufacture of ArmaGen's investigational enzyme replacement therapy, AGT-181, for Phase 1 Clinical Trial 48 Feb 05, 2014: Mouse Study Shows Gene Therapy May Be Possible Cure for Hurler Syndrome 48 Sep 18, 2009: ArmaGen Completes Primate Safety Study For AGT-181 49 Appendix 50 Methodology 50 Coverage 50 Secondary Research 50 Primary Research 50 Expert Panel Validation 50 Contact Us 50 Disclaimer 51

List of Tables Number of Products under Development for, H2 2016 7 Number of Products under Development by Therapy Area, H2 2016 8 Number of Products under Development by Indication, H2 2016 9 Comparative Analysis by Late Stage Development, H2 2016 10 Comparative Analysis by Early Stage Products, H2 2016 11 Number of Products under Development by Companies, H2 2016 12 Products under Development by Companies, H2 2016 13 Number of Products under Investigation by Universities/Institutes, H2 2016 14 Products under Investigation by Universities/Institutes, H2 2016 15 Assessment by Monotherapy/Combination Products, H2 2016 16 Number of Products by Stage and Mechanism of Action, H2 2016 18 Number of Products by Stage and Route of Administration, H2 2016 20 Number of Products by Stage and Molecule Type, H2 2016 22 Pipeline by AngioChem Inc., H2 2016 23 Pipeline by ArmaGen Inc., H2 2016 24 Pipeline by Bioasis Technologies Inc., H2 2016 25 Pipeline by RegenxBio Inc., H2 2016 26 Pipeline by Sangamo BioSciences, Inc., H2 2016 27 Dormant Projects, H2 2016 40


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