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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

Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline Review, H2 2016
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
RandD Progress 37
ataluren - Drug Profile 39
Product Description 39
Mechanism Of Action 39
RandD Progress 39
Cell Therapy for Mucopolysaccharidosis I - Drug Profile 48
Product Description 48
Mechanism Of Action 48
RandD Progress 48
Cell Therapy to Activate Alpha L-Iduronidase for Hurler Syndrome - Drug Profile 49
Product Description 49
Mechanism Of Action 49
RandD Progress 49
ELX-02 - Drug Profile 50
Product Description 50
Mechanism Of Action 50
RandD Progress 50
HSC-835 - Drug Profile 51
Product Description 51
Mechanism Of Action 51
RandD Progress 51
JOT-102 - Drug Profile 52
Product Description 52
Mechanism Of Action 52
RandD Progress 52
odiparcil - Drug Profile 53
Product Description 53
Mechanism Of Action 53
RandD Progress 53
Oligonucleotide for Mucopolysaccharidosis I - Drug Profile 54
Product Description 54
Mechanism Of Action 54
RandD Progress 54
pentosan polysulfate sodium - Drug Profile 55
Product Description 55
Mechanism Of Action 55
RandD Progress 55
Recombinant Alpha-L-Iduronidase Replacement for Hurler Syndrome - Drug Profile 56
Product Description 56
Mechanism Of Action 56
RandD Progress 56
Recombinant Alpha-L-Iduronidase Replacement for Mucopolysaccharidosis I - Drug Profile 57
Product Description 57
Mechanism Of Action 57
RandD Progress 57
Recombinant Alpha-L-Iduronidase Replacement for Mucopolysaccharidosis I - Drug Profile 58
Product Description 58
Mechanism Of Action 58
RandD Progress 58
RGX-111 - Drug Profile 59
Product Description 59
Mechanism Of Action 59
RandD Progress 59
SB-318 - Drug Profile 61
Product Description 61
Mechanism Of Action 61
RandD Progress 61
Stem Cell Therapy for Hurler Sydrome - Drug Profile 63
Product Description 63
Mechanism Of Action 63
RandD Progress 63
Stem Cell Therapy for Type1 Mucopolysaccharidosis - Drug Profile 64
Product Description 64
Mechanism Of Action 64
RandD Progress 64
X-372 - Drug Profile 65
Product Description 65
Mechanism Of Action 65
RandD 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 and 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

List of Figures
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 Products, H2 2016 14
Assessment by Monotherapy Products, H2 2016 28
Number of Products by Targets, H2 2016 29
Number of Products by Stage and Targets, H2 2016 29
Number of Products by Mechanism of Actions, H2 2016 31
Number of Products by Stage and Mechanism of Actions, H2 2016 31
Number of Products by Routes of Administration, H2 2016 33
Number of Products by Stage and Routes of Administration, H2 2016 33
Number of Products by Molecule Types, H2 2016 35
Number of Products by Stage and Molecule Types, H2 2016 35

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