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Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline Review, H1 2018

Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline Review, H1 2018

Summary

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline Review, H1 2018, provides an overview of the Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) (Genetic 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, H1 2018, provides comprehensive information on the therapeutics under development for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) (Genetic 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 ) (Genetic 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, 1, 1, 6 and 2 respectively. Similarly, the Universities portfolio in Preclinical and Discovery stages comprises 1 and 1 molecules, respectively.

Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) (Genetic 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 ) (Genetic Disorders).
  • The pipeline guide reviews pipeline therapeutics for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) (Genetic 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 ) (Genetic Disorders) therapeutics and enlists all their major and minor projects.
  • The pipeline guide evaluates Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) (Genetic 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 ) (Genetic 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 ) (Genetic 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 ) (Genetic 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
CRISPR Therapeutics
Eloxx Pharmaceuticals Inc
Immusoft Corp
JCR Pharmaceuticals Co Ltd
OPKO Health 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
Antisense Oligonucleotide for Mucopolysaccharidosis I - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Cell Therapy to Activate IDUA for Mucopolysaccharidosis I - 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
laronidase - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
MGTA-456 - 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 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 to Activate Alpha L-Iduronidase for Type1 Mucopolysaccharidosis - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Stem Cell Therapy to Target Alpha L-Iduronidase for Hurler Sydrome - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Tamid-001 - 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
May 02, 2018: Immusoft Granted Orphan Drug Designation for Treatment of Rare Childhood Disease
Apr 05, 2018: Magenta Therapeutics Announces First Patient Transplanted with MGTA-456 in Phase 2 Study in Inherited Metabolic Disorders
Mar 14, 2018: Jupiter Orphan Therapeutics Announces Submission of Investigational New Drug Application (IND) For Treatment Of Mucopolysaccharidosis Type-I
Feb 08, 2018: ArmaGen’s AGT-181 Demonstrates Neurocognitive Benefit in Children with Severe MPS I
Feb 05, 2018: Jupiter Orphan Therapeutics in Process to Submit IND for MPSI and Other Indications
Dec 08, 2017: Sangamo Announces EMA Recommendation of Orphan Medicinal Product Designation for Investigational Genome Editing Treatment for Mucopolysaccharidosis Type I
Nov 30, 2017: ArmaGen's AGT-181 Granted Fast Track Designation for the Treatment of Hurler Syndrome (MPS I)
Oct 26, 2017: JCR to Initiate Development of JR-171, a New Drug Candidate for Hurler Syndrome Using J-Brain Cargo
Aug 08, 2017: REGENXBIO Announces IND Active for Phase I Trial of RGX-111 to Treat Mucopolysaccharidosis Type I
Jul 13, 2017: Sangamo Receives Fast Track Designation From The FDA For SB-318 In Vivo Genome Editing Product Candidate For The Treatment Of MPS I
Feb 27, 2017: Sangamo Therapeutics Receives Rare Pediatric Disease Designation From FDA For SB-318 In Vivo Genome Editing Therapeutic For MPS I
Feb 16, 2017: ArmaGen Reports Preliminary Evidence of Cognitive Improvement in Children with Hurler Syndrome (MPS I) Treated with AGT-181
Feb 08, 2017: Sangamo Therapeutics Announces Data on SB-318 at The 13th Annual WORLDSymposium Meeting
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
Jan 11, 2017: Sangamo Therapeutics Receives Orphan Drug Designation from the FDA for SB-318 Genome Editing Treatment for MPS I
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 2018
Number of Products under Development by Companies, H1 2018
Number of Products under Development by Universities/Institutes, H1 2018
Products under Development by Companies, H1 2018
Products under Development by Universities/Institutes, H1 2018
Number of Products by Stage and Target, H1 2018
Number of Products by Stage and Mechanism of Action, H1 2018
Number of Products by Stage and Route of Administration, H1 2018
Number of Products by Stage and Molecule Type, H1 2018
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by AngioChem Inc, H1 2018
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by ArmaGen Inc, H1 2018
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by CRISPR Therapeutics, H1 2018
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by Eloxx Pharmaceuticals Inc, H1 2018
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by Immusoft Corp, H1 2018
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by JCR Pharmaceuticals Co Ltd, H1 2018
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by OPKO Health Inc, H1 2018
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by RegenxBio Inc, H1 2018
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by Sangamo Therapeutics Inc, H1 2018
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Dormant Projects, H1 2018
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Discontinued Products, H1 2018
List of Figures
Number of Products under Development for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ), H1 2018
Number of Products under Development by Companies, H1 2018
Number of Products under Development by Universities/Institutes, H1 2018
Number of Products by Stage and Targets, H1 2018
Number of Products by Mechanism of Actions, H1 2018
Number of Products by Stage and Mechanism of Actions, H1 2018
Number of Products by Routes of Administration, H1 2018
Number of Products by Stage and Routes of Administration, H1 2018
Number of Products by Molecule Types, H1 2018
Number of Products by Stage and Molecule Types, H1 2018

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