Mucopolysaccharidosis I - Pipeline Review, H1 2017
Mucopolysaccharidosis I - Pipeline Review, H1 2017
SUMMARY
Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Mucopolysaccharidosis I - Pipeline Review, H1 2017, provides an overview of the Mucopolysaccharidosis I (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 - Pipeline Review, H1 2017, provides comprehensive information on the therapeutics under development for Mucopolysaccharidosis I (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 (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, Preclinical and Discovery stages are 5, 1, 6 and 3 respectively. Similarly, the Universities portfolio in Preclinical and Discovery stages comprises 2 and 1 molecules, respectively.
Mucopolysaccharidosis I (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
SUMMARY
Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Mucopolysaccharidosis I - Pipeline Review, H1 2017, provides an overview of the Mucopolysaccharidosis I (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 - Pipeline Review, H1 2017, provides comprehensive information on the therapeutics under development for Mucopolysaccharidosis I (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 (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, Preclinical and Discovery stages are 5, 1, 6 and 3 respectively. Similarly, the Universities portfolio in Preclinical and Discovery stages comprises 2 and 1 molecules, respectively.
Mucopolysaccharidosis I (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 (Metabolic Disorders).
- The pipeline guide reviews pipeline therapeutics for Mucopolysaccharidosis I (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 (Metabolic Disorders) therapeutics and enlists all their major and minor projects.
- The pipeline guide evaluates Mucopolysaccharidosis I (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 (Metabolic Disorders)
- 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 (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 (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.
Introduction
Global Markets Direct Report Coverage
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Overview
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Therapeutics Development
Pipeline Overview
Pipeline by Companies
Pipeline by Universities/Institutes
Products under Development by Companies
Products under Development by Universities/Institutes
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Therapeutics Assessment
Assessment by Target
Assessment by Mechanism of Action
Assessment by Route of Administration
Assessment by Molecule Type
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Companies Involved in Therapeutics Development
AngioChem Inc
ArmaGen Inc
Bioasis Technologies Inc
CRISPR Therapeutics
Eloxx Pharmaceuticals Ltd
Immusoft Corp
Inventiva
OPKO Health Inc
PTC Therapeutics Inc
RegenxBio Inc
Sangamo Therapeutics Inc
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Drug Profiles
AGT-181 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
ataluren - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Cell Therapy for Mucopolysaccharidosis I - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Cell Therapy to Activate Alpha L-Iduronidase for Hurler Syndrome - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
ELX-02 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
JOT-102 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
MGTA-456 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
odiparcil - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Oligonucleotide for Mucopolysaccharidosis I - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
pentosan polysulfate sodium - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Recombinant Alpha-L-Iduronidase Replacement for Hurler Syndrome - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Recombinant Alpha-L-Iduronidase Replacement for Mucopolysaccharidosis I - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Recombinant Alpha-L-Iduronidase Replacement for Mucopolysaccharidosis I - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
RGX-111 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
SB-318 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Stem Cell Therapy for Hurler Sydrome - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Stem Cell Therapy for Type1 Mucopolysaccharidosis - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
X-372 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Dormant Projects
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Discontinued Products
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Product Development Milestones
Featured News & Press Releases
Feb 16, 2017: ArmaGen Reports Preliminary Evidence of Cognitive Improvement in Children with Hurler Syndrome (MPS I) Treated with AGT-181
Feb 07, 2017: ArmaGen Announces Oral Presentation of Preliminary Results from its Phase 2 Clinical Trial of AGT-181 in Patients with MPS 1 to be Presented at WORLDSymposium 2017
Nov 07, 2016: Eloxx Pharmaceuticals Announces Orphan Drug Designation in the U.S. and Europe for ELX-02 in Mucopolysacchardisis Type 1 (MPS 1)
Sep 13, 2016: REGENXBIO Publishes Data from Ongoing Preclinical Studies of NAV Gene Therapy RGX-111
Jul 05, 2016: REGENXBIO Provides Update On Gene Therapy Development Program RGX-111
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
Dec 30, 2015: FDA Grants Rare Pediatric Disease Designation to REGENXBIO RGX-111 Gene Therapy for the Treatment of Mucopolysaccharidosis Type I (MPS I)
Nov 05, 2015: ArmaGen Receives Rare Pediatric Disease Designation from FDA for AGT-181 for the Potential Treatment of Hurler Syndrome
Oct 01, 2015: FDA Grants Orphan Drug Designation to REGENXBIO's RGX-111 Gene Therapy for the Treatment of Mucopolysaccharidosis Type I
Sep 02, 2015: ArmaGen Announces First Patient Dosed in Phase 1/2a Clinical Trial of AGT-181 for the Treatment of Hurler Syndrome
Apr 08, 2015: ArmaGen Announces FDA Acceptance of IND Application for AGT-181 for the Treatment of Hurler Syndrome
Dec 19, 2014: Translarna Granted Orphan Drug Designation in the U.S. and Europe for the Treatment of Mucopolysaccharidosis I
Feb 05, 2014: Mouse Study Shows Gene Therapy May Be Possible Cure for Hurler Syndrome
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer
Global Markets Direct Report Coverage
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Overview
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Therapeutics Development
Pipeline Overview
Pipeline by Companies
Pipeline by Universities/Institutes
Products under Development by Companies
Products under Development by Universities/Institutes
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Therapeutics Assessment
Assessment by Target
Assessment by Mechanism of Action
Assessment by Route of Administration
Assessment by Molecule Type
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Companies Involved in Therapeutics Development
AngioChem Inc
ArmaGen Inc
Bioasis Technologies Inc
CRISPR Therapeutics
Eloxx Pharmaceuticals Ltd
Immusoft Corp
Inventiva
OPKO Health Inc
PTC Therapeutics Inc
RegenxBio Inc
Sangamo Therapeutics Inc
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Drug Profiles
AGT-181 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
ataluren - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Cell Therapy for Mucopolysaccharidosis I - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Cell Therapy to Activate Alpha L-Iduronidase for Hurler Syndrome - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
ELX-02 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
JOT-102 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
MGTA-456 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
odiparcil - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Oligonucleotide for Mucopolysaccharidosis I - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
pentosan polysulfate sodium - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Recombinant Alpha-L-Iduronidase Replacement for Hurler Syndrome - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Recombinant Alpha-L-Iduronidase Replacement for Mucopolysaccharidosis I - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Recombinant Alpha-L-Iduronidase Replacement for Mucopolysaccharidosis I - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
RGX-111 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
SB-318 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Stem Cell Therapy for Hurler Sydrome - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Stem Cell Therapy for Type1 Mucopolysaccharidosis - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
X-372 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Dormant Projects
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Discontinued Products
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Product Development Milestones
Featured News & Press Releases
Feb 16, 2017: ArmaGen Reports Preliminary Evidence of Cognitive Improvement in Children with Hurler Syndrome (MPS I) Treated with AGT-181
Feb 07, 2017: ArmaGen Announces Oral Presentation of Preliminary Results from its Phase 2 Clinical Trial of AGT-181 in Patients with MPS 1 to be Presented at WORLDSymposium 2017
Nov 07, 2016: Eloxx Pharmaceuticals Announces Orphan Drug Designation in the U.S. and Europe for ELX-02 in Mucopolysacchardisis Type 1 (MPS 1)
Sep 13, 2016: REGENXBIO Publishes Data from Ongoing Preclinical Studies of NAV Gene Therapy RGX-111
Jul 05, 2016: REGENXBIO Provides Update On Gene Therapy Development Program RGX-111
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
Dec 30, 2015: FDA Grants Rare Pediatric Disease Designation to REGENXBIO RGX-111 Gene Therapy for the Treatment of Mucopolysaccharidosis Type I (MPS I)
Nov 05, 2015: ArmaGen Receives Rare Pediatric Disease Designation from FDA for AGT-181 for the Potential Treatment of Hurler Syndrome
Oct 01, 2015: FDA Grants Orphan Drug Designation to REGENXBIO's RGX-111 Gene Therapy for the Treatment of Mucopolysaccharidosis Type I
Sep 02, 2015: ArmaGen Announces First Patient Dosed in Phase 1/2a Clinical Trial of AGT-181 for the Treatment of Hurler Syndrome
Apr 08, 2015: ArmaGen Announces FDA Acceptance of IND Application for AGT-181 for the Treatment of Hurler Syndrome
Dec 19, 2014: Translarna Granted Orphan Drug Designation in the U.S. and Europe for the Treatment of Mucopolysaccharidosis I
Feb 05, 2014: Mouse Study Shows Gene Therapy May Be Possible Cure for Hurler Syndrome
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer
LIST OF TABLES
Number of Products under Development for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ), H1 2017
Number of Products under Development by Companies, H1 2017
Number of Products under Development by Universities/Institutes, H1 2017
Products under Development by Companies, H1 2017
Products under Development by Universities/Institutes, H1 2017
Number of Products by Stage and Target, H1 2017
Number of Products by Stage and Mechanism of Action, H1 2017
Number of Products by Stage and Route of Administration, H1 2017
Number of Products by Stage and Molecule Type, H1 2017
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by AngioChem Inc, H1 2017
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by ArmaGen Inc, H1 2017
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by Bioasis Technologies Inc, H1 2017
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by CRISPR Therapeutics, H1 2017
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by Eloxx Pharmaceuticals Ltd, H1 2017
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by Immusoft Corp, H1 2017
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by Inventiva, H1 2017
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by OPKO Health Inc, H1 2017
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by PTC Therapeutics Inc, H1 2017
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by RegenxBio Inc, H1 2017
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by Sangamo Therapeutics Inc, H1 2017
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Dormant Projects, H1 2017
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Discontinued Products, H1 2017
Number of Products under Development for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ), H1 2017
Number of Products under Development by Companies, H1 2017
Number of Products under Development by Universities/Institutes, H1 2017
Products under Development by Companies, H1 2017
Products under Development by Universities/Institutes, H1 2017
Number of Products by Stage and Target, H1 2017
Number of Products by Stage and Mechanism of Action, H1 2017
Number of Products by Stage and Route of Administration, H1 2017
Number of Products by Stage and Molecule Type, H1 2017
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by AngioChem Inc, H1 2017
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by ArmaGen Inc, H1 2017
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by Bioasis Technologies Inc, H1 2017
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by CRISPR Therapeutics, H1 2017
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by Eloxx Pharmaceuticals Ltd, H1 2017
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by Immusoft Corp, H1 2017
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by Inventiva, H1 2017
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by OPKO Health Inc, H1 2017
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by PTC Therapeutics Inc, H1 2017
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by RegenxBio Inc, H1 2017
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by Sangamo Therapeutics Inc, H1 2017
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Dormant Projects, H1 2017
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Discontinued Products, H1 2017
LIST OF FIGURES
Number of Products under Development for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ), H1 2017
Number of Products under Development by Companies, H1 2017
Number of Products under Development by Universities/Institutes, H1 2017
Number of Products by Targets, H1 2017
Number of Products by Stage and Targets, H1 2017
Number of Products by Mechanism of Actions, H1 2017
Number of Products by Stage and Mechanism of Actions, H1 2017
Number of Products by Routes of Administration, H1 2017
Number of Products by Stage and Routes of Administration, H1 2017
Number of Products by Molecule Types, H1 2017
Number of Products by Stage and Molecule Types, H1 2017
COMPANIES MENTIONED
AngioChem Inc
ArmaGen Inc
Bioasis Technologies Inc
CRISPR Therapeutics
Eloxx Pharmaceuticals Ltd
Immusoft Corp
Inventiva
OPKO Health Inc
PTC Therapeutics Inc
RegenxBio Inc
Sangamo Therapeutics Inc
Number of Products under Development for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ), H1 2017
Number of Products under Development by Companies, H1 2017
Number of Products under Development by Universities/Institutes, H1 2017
Number of Products by Targets, H1 2017
Number of Products by Stage and Targets, H1 2017
Number of Products by Mechanism of Actions, H1 2017
Number of Products by Stage and Mechanism of Actions, H1 2017
Number of Products by Routes of Administration, H1 2017
Number of Products by Stage and Routes of Administration, H1 2017
Number of Products by Molecule Types, H1 2017
Number of Products by Stage and Molecule Types, H1 2017
COMPANIES MENTIONED
AngioChem Inc
ArmaGen Inc
Bioasis Technologies Inc
CRISPR Therapeutics
Eloxx Pharmaceuticals Ltd
Immusoft Corp
Inventiva
OPKO Health Inc
PTC Therapeutics Inc
RegenxBio Inc
Sangamo Therapeutics Inc
