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Published: Dec, 2016 | Pages:
79 | Publisher: Global Markets Direct
Industry: Pharmaceuticals & Healthcare | Report Format: Electronic (PDF)
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline Review, H2 2016 Summary Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline Review, H2 2016, provides an overview of the Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) (Metabolic Disorders) pipeline landscape. MPS I (Mucopolysaccharidosis I) is an inherited lysosomal storage disorder caused by a deficiency of alpha-L-iduronidase, a lysosomal enzyme normally required for the breakdown of certain complex carbohydrates known as glycosaminoglycans (GAGs). Symptoms include abnormal bones in the spine, claw hand, cloudy corneas, deafness and heart valve problems. Treatment includes bone marrow transplantation, enzyme therapy and gene therapy. Report Highlights Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline Review, H2 2016, provides comprehensive information on the therapeutics under development for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) (Metabolic Disorders), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The guide covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases. The Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) (Metabolic Disorders) pipeline guide also reviews of key players involved in therapeutic development for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Phase II, Phase I, IND/CTA Filed, Preclinical and Discovery stages are 4, 2, 1, 5 and 3 respectively. Similarly, the Universities portfolio in Preclinical and Discovery stages comprises 2 and 1 molecules, respectively. Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) (Metabolic Disorders) pipeline guide 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. The guide is built using data and information sourced from Global Markets Direct's 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. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis. Note: Certain content / sections in the pipeline guide may be removed or altered based on the availability and relevance of data. Scope - The pipeline guide provides a snapshot of the global therapeutic landscape of Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) (Metabolic Disorders). - The pipeline guide reviews pipeline therapeutics for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) (Metabolic Disorders) by companies and universities/research institutes based on information derived from company and industry-specific sources. - The pipeline guide covers pipeline products based on several stages of development ranging from pre-registration till discovery and undisclosed stages. - The pipeline guide features descriptive drug profiles for the pipeline products which comprise, product description, descriptive licensing and collaboration details, R&D brief, MoA & other developmental activities. - The pipeline guide reviews key companies involved in Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) (Metabolic Disorders) therapeutics and enlists all their major and minor projects. - The pipeline guide evaluates Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) (Metabolic Disorders) therapeutics based on mechanism of action (MoA), drug target, route of administration (RoA) and molecule type. - The pipeline guide encapsulates all the dormant and discontinued pipeline projects. - The pipeline guide reviews latest news related to pipeline therapeutics for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) (Metabolic Disorders) Reasons To Buy - Procure strategically important competitor information, analysis, and insights to formulate effective R&D strategies. - Recognize emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage. - Find and recognize significant and varied types of therapeutics under development for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) (Metabolic Disorders). - Classify potential new clients or partners in the target demographic. - Develop tactical initiatives by understanding the focus areas of leading companies. - Plan mergers and acquisitions meritoriously by identifying key players and it's most promising pipeline therapeutics. - Formulate corrective measures for pipeline projects by understanding Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) (Metabolic Disorders) pipeline depth and focus of Indication therapeutics. - 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. - Adjust the therapeutic portfolio by recognizing discontinued projects and understand from the know-how what drove them from pipeline.
Table of Contents
Table of Contents 2 List of Tables 5 List of Figures 6 Introduction 7 Global Markets Direct Report Coverage 7 Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) Overview 8 Therapeutics Development 9 Pipeline Products for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Overview 9 Pipeline Products for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Comparative Analysis 10 Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Therapeutics under Development by Companies 11 Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Therapeutics under Investigation by Universities/Institutes 12 Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline Products Glance 13 Clinical Stage Products 13 Early Stage Products 14 Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Products under Development by Companies 15 Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Products under Investigation by Universities/Institutes 16 Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Companies Involved in Therapeutics Development 17 AngioChem Inc 17 ArmaGen Inc 18 Bioasis Technologies Inc 19 CRISPR Therapeutics 20 Eloxx Pharmaceuticals Ltd 21 Inventiva 22 Novartis AG 23 OPKO Health Inc 24 PTC Therapeutics Inc 25 RegenxBio Inc 26 Sangamo BioSciences Inc 27 Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Therapeutics Assessment 28 Assessment by Monotherapy Products 28 Assessment by Target 29 Assessment by Mechanism of Action 31 Assessment by Route of Administration 33 Assessment by Molecule Type 35 Drug Profiles 37 AGT-181 - Drug Profile 37 Product Description 37 Mechanism Of Action 37 R&D Progress 37 ataluren - Drug Profile 39 Product Description 39 Mechanism Of Action 39 R&D Progress 39 Cell Therapy for Mucopolysaccharidosis I - Drug Profile 48 Product Description 48 Mechanism Of Action 48 R&D Progress 48 Cell Therapy to Activate Alpha L-Iduronidase for Hurler Syndrome - Drug Profile 49 Product Description 49 Mechanism Of Action 49 R&D Progress 49 ELX-02 - Drug Profile 50 Product Description 50 Mechanism Of Action 50 R&D Progress 50 HSC-835 - Drug Profile 51 Product Description 51 Mechanism Of Action 51 R&D Progress 51 JOT-102 - Drug Profile 52 Product Description 52 Mechanism Of Action 52 R&D Progress 52 odiparcil - Drug Profile 53 Product Description 53 Mechanism Of Action 53 R&D Progress 53 Oligonucleotide for Mucopolysaccharidosis I - Drug Profile 54 Product Description 54 Mechanism Of Action 54 R&D Progress 54 pentosan polysulfate sodium - Drug Profile 55 Product Description 55 Mechanism Of Action 55 R&D Progress 55 Recombinant Alpha-L-Iduronidase Replacement for Hurler Syndrome - Drug Profile 56 Product Description 56 Mechanism Of Action 56 R&D Progress 56 Recombinant Alpha-L-Iduronidase Replacement for Mucopolysaccharidosis I - Drug Profile 57 Product Description 57 Mechanism Of Action 57 R&D Progress 57 Recombinant Alpha-L-Iduronidase Replacement for Mucopolysaccharidosis I - Drug Profile 58 Product Description 58 Mechanism Of Action 58 R&D Progress 58 RGX-111 - Drug Profile 59 Product Description 59 Mechanism Of Action 59 R&D Progress 59 SB-318 - Drug Profile 61 Product Description 61 Mechanism Of Action 61 R&D Progress 61 Stem Cell Therapy for Hurler Sydrome - Drug Profile 63 Product Description 63 Mechanism Of Action 63 R&D Progress 63 Stem Cell Therapy for Type1 Mucopolysaccharidosis - Drug Profile 64 Product Description 64 Mechanism Of Action 64 R&D Progress 64 X-372 - Drug Profile 65 Product Description 65 Mechanism Of Action 65 R&D Progress 65 Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Dormant Projects 66 Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Discontinued Products 67 Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Product Development Milestones 68 Featured News & Press Releases 68 Nov 07, 2016: Eloxx Pharmaceuticals Announces Orphan Drug Designation in the U.S. and Europe for ELX-02 in Mucopolysacchardisis Type 1 (MPS 1) 68 Sep 13, 2016: REGENXBIO Publishes Data from Ongoing Preclinical Studies of NAV Gene Therapy RGX-111 68 Jul 05, 2016: REGENXBIO Provides Update On Gene Therapy Development Program RGX-111 69 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 69 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 71 Feb 08, 2016: Sangamo BioSciences Announces FDA Clearance Of Investigational New Drug Application For ZFN-Mediated Genome Editing Treatment Of MPS I 72 Dec 30, 2015: FDA Grants Rare Pediatric Disease Designation to REGENXBIO RGX-111 Gene Therapy for the Treatment of Mucopolysaccharidosis Type I (MPS I) 73 Nov 05, 2015: ArmaGen Receives Rare Pediatric Disease Designation from FDA for AGT-181 for the Potential Treatment of Hurler Syndrome 73 Oct 01, 2015: FDA Grants Orphan Drug Designation to REGENXBIO's RGX-111 Gene Therapy for the Treatment of Mucopolysaccharidosis Type I 73 Sep 02, 2015: ArmaGen Announces First Patient Dosed in Phase 1/2a Clinical Trial of AGT-181 for the Treatment of Hurler Syndrome 74 Apr 08, 2015: ArmaGen Announces FDA Acceptance of IND Application for AGT-181 for the Treatment of Hurler Syndrome 75 Dec 19, 2014: Translarna Granted Orphan Drug Designation in the U.S. and Europe for the Treatment of Mucopolysaccharidosis I 75 Feb 05, 2014: Mouse Study Shows Gene Therapy May Be Possible Cure for Hurler Syndrome 76 Appendix 78 Methodology 78 Coverage 78 Secondary Research 78 Primary Research 78 Expert Panel Validation 78 Contact Us 78 Disclaimer 79
List of Tables
Number of Products under Development for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ), H2 2016 9 Number of Products under Development for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Comparative Analysis, H2 2016 10 Number of Products under Development by Companies, H2 2016 11 Number of Products under Investigation by Universities/Institutes, H2 2016 12 Comparative Analysis by Clinical Stage Development, H2 2016 13 Comparative Analysis by Early Stage Development, H2 2016 14 Products under Development by Companies, H2 2016 15 Products under Investigation by Universities/Institutes, H2 2016 16 Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by AngioChem Inc, H2 2016 17 Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by ArmaGen Inc, H2 2016 18 Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by Bioasis Technologies Inc, H2 2016 19 Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by CRISPR Therapeutics, H2 2016 20 Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by Eloxx Pharmaceuticals Ltd, H2 2016 21 Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by Inventiva, H2 2016 22 Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by Novartis AG, H2 2016 23 Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by OPKO Health Inc, H2 2016 24 Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by PTC Therapeutics Inc, H2 2016 25 Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by RegenxBio Inc, H2 2016 26 Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by Sangamo BioSciences Inc, H2 2016 27 Assessment by Monotherapy Products, H2 2016 28 Number of Products by Stage and Target, H2 2016 30 Number of Products by Stage and Mechanism of Action, H2 2016 32 Number of Products by Stage and Route of Administration, H2 2016 34 Number of Products by Stage and Molecule Type, H2 2016 36 Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Dormant Projects, H2 2016 66 Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Discontinued Products, H2 2016 67
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