Iduronate 2 Sulfatase (Alpha L Iduronate Sulfate Sulfatase or Idursulfase or IDS or EC 3.1.6.13) - Pipeline Review, H2 2017

Iduronate 2 Sulfatase (Alpha L Iduronate Sulfate Sulfatase or Idursulfase or IDS or EC 3.1.6.13) - Pipeline Review, H2 2017

Summary

Iduronate 2 Sulfatase (Alpha L Iduronate Sulfate Sulfatase or Idursulfase or IDS or EC 3.1.6.13) pipeline Target constitutes close to 11 molecules. Out of which approximately 10 molecules are developed by companies and remaining by the universities/institutes. The latest report Iduronate 2 Sulfatase - Pipeline Review, H2 2017, outlays comprehensive information on the Iduronate 2 Sulfatase (Alpha L Iduronate Sulfate Sulfatase or Idursulfase or IDS or EC 3.1.6.13) targeted therapeutics, complete with analysis by indications, stage of development, mechanism of action (MoA), route of administration (RoA) and molecule type.

Iduronate 2 Sulfatase (Alpha L Iduronate Sulfate Sulfatase or Idursulfase or IDS or EC 3.1.6.13) - Iduronate 2-sulfatase (IDS) is a sulfatase enzyme associated with Hunter syndrome. Iduronate 2-sulfatase is required for the lysosomal degradation of heparan sulfate and dermatan sulfate. Mutations in this X-chromosome gene that result in enzymatic deficiency lead to Hunter syndrome. The molecules developed by companies in Phase III, Phase II, Preclinical and Discovery stages are 1, 4, 4 and 1 respectively. Similarly, the universities portfolio in Preclinical stages comprises 1 molecules, respectively. Report covers products from therapy areas Genetic Disorders and Central Nervous System which include indications Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) and Cognitive Impairment.

Furthermore, this report also reviews key players involved in Iduronate 2 Sulfatase (Alpha L Iduronate Sulfate Sulfatase or Idursulfase or IDS or EC 3.1.6.13) targeted therapeutics development with respective active and dormant or discontinued projects. Driven by data and information sourced from 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.

Note: Certain content / sections in the pipeline guide may be removed or altered based on the availability and relevance of data.

Scope

- The report provides a snapshot of the global therapeutic landscape for Iduronate 2 Sulfatase (Alpha L Iduronate Sulfate Sulfatase or Idursulfase or IDS or EC 3.1.6.13)
- The report reviews Iduronate 2 Sulfatase (Alpha L Iduronate Sulfate Sulfatase or Idursulfase or IDS or EC 3.1.6.13) 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 Iduronate 2 Sulfatase (Alpha L Iduronate Sulfate Sulfatase or Idursulfase or IDS or EC 3.1.6.13) targeted therapeutics and enlists all their major and minor projects
- The report assesses Iduronate 2 Sulfatase (Alpha L Iduronate Sulfate Sulfatase or Idursulfase or IDS or EC 3.1.6.13) 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 Iduronate 2 Sulfatase (Alpha L Iduronate Sulfate Sulfatase or Idursulfase or IDS or EC 3.1.6.13) 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 Iduronate 2 Sulfatase (Alpha L Iduronate Sulfate Sulfatase or Idursulfase or IDS or EC 3.1.6.13)
- 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 Iduronate 2 Sulfatase (Alpha L Iduronate Sulfate Sulfatase or Idursulfase or IDS or EC 3.1.6.13) 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

Introduction
Global Markets Direct Report Coverage
Iduronate 2 Sulfatase (Alpha L Iduronate Sulfate Sulfatase or Idursulfase or IDS or EC 3.1.6.13) - Overview
Iduronate 2 Sulfatase (Alpha L Iduronate Sulfate Sulfatase or Idursulfase or IDS or EC 3.1.6.13) - Therapeutics Development
Products under Development by Stage of Development
Products under Development by Therapy Area
Products under Development by Indication
Products under Development by Companies
Products under Development by Universities/Institutes
Iduronate 2 Sulfatase (Alpha L Iduronate Sulfate Sulfatase or Idursulfase or IDS or EC 3.1.6.13) - Therapeutics Assessment
Assessment by Mechanism of Action
Assessment by Route of Administration
Assessment by Molecule Type
Iduronate 2 Sulfatase (Alpha L Iduronate Sulfate Sulfatase or Idursulfase or IDS or EC 3.1.6.13) - Companies Involved in Therapeutics Development
AngioChem Inc
ArmaGen Inc
Denali Therapeutics Inc
Green Cross Corp
JCR Pharmaceuticals Co Ltd
Laboratorios Del Dr Esteve SA
RegenxBio Inc
Sangamo Therapeutics Inc
Shire Plc
Iduronate 2 Sulfatase (Alpha L Iduronate Sulfate Sulfatase or Idursulfase or IDS or EC 3.1.6.13) - Drug Profiles
AGT-182 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
EGT-301 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy to Activate Iduronate 2 Sulfatase for Mucopolysaccharidosis II - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Iduronate 2 Sulfatase Replacement for Mucopolysaccharidosis II - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
idursulfase - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
idursulfase beta - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
JR-032 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
JR-141 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Recombinant Iduronate 2-Sulfatase Replacement for Mucopolysaccharidosis II - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
RGX-121 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
SB-913 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Iduronate 2 Sulfatase (Alpha L Iduronate Sulfate Sulfatase or Idursulfase or IDS or EC 3.1.6.13) - Dormant Products
Iduronate 2 Sulfatase (Alpha L Iduronate Sulfate Sulfatase or Idursulfase or IDS or EC 3.1.6.13) - Product Development Milestones
Featured News & Press Releases
Nov 15, 2017: Sangamo Announces Treatment of First Patient in Landmark Phase 1/2 Clinical Trial Evaluating In Vivo Genome Editing for MPS II
Jul 13, 2017: Sangamo Receives Fast Track Designation From The FDA For SB-913
May 04, 2017: Sangamo Therapeutics Announces Rare Pediatric Disease Designation for SB-913 In Vivo Genome Editing Treatment for MPS II
Mar 31, 2017: JCR Announces First Patient Dosed in Phase 1/2 Clinical Trial of JR-141 in Hunter Syndrome
Mar 01, 2017: Sangamo Therapeutics Receives Orphan Drug Designation from the FDA for SB-913 Genome Editing Treatment for MPS II
Feb 17, 2017: JCR Announces Data Presentation on the Efficacy of BBB-Penetrating IDS on CNS in MPS II Mouse Model at the 13th Annual WORLDSymposium 2017
Feb 08, 2017: Sangamo Therapeutics Announces Data on SB-913 at The 13th Annual WORLDSymposium Meeting
Jan 19, 2017: JCR announces completion of PMDA review of clinical trial plan for JR-141
Oct 26, 2016: National Center for Child Health and Development Initiates Phase 1/2 Trial of Green Cross idursulfase-beta ICV for the Treatment of Hunter Syndrome with Neurocognitive Decline
Sep 13, 2016: REGENXBIO Publishes Data from Ongoing Preclinical Studies of NAV Gene Therapy RGX-121
Jul 14, 2016: ArmaGen Presents Data from First Cohort of Phase 1/2a of AGT-182 for Treatment of Hunter Syndrome
Jul 05, 2016: REGENXBIO Provides Update On Gene Therapy Development Program RGX-121
Jun 20, 2016: Sangamo BioSciences Announces FDA Clearance of Investigational New Drug Application for ZFN-Mediated Genome Editing Treatment of MPS II
Apr 19, 2016: Preclinical Data from REGENXBIOs RGX-121 Gene Therapy Program to be Presented at the American Society of Gene & Cell Therapy 19th Annual Meeting
Feb 25, 2016: ESTEVE Provides Update On EGT-301 For Hunter Syndrome
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer

List Of Tables


Number of Products under Development by Stage of Development, H2 2017
Number of Products under Development by Therapy Areas, H2 2017
Number of Products under Development by Indication, H2 2017
Number of Products under Development by Companies, H2 2017
Products under Development by Companies, H2 2017
Number of Products under Investigation by Universities/Institutes, H2 2017
Products under Investigation by Universities/Institutes, H2 2017
Number of Products by Stage and Mechanism of Actions, H2 2017
Number of Products by Stage and Route of Administration, H2 2017
Number of Products by Stage and Molecule Type, H2 2017
Pipeline by AngioChem Inc, H2 2017
Pipeline by ArmaGen Inc, H2 2017
Pipeline by Denali Therapeutics Inc, H2 2017
Pipeline by Green Cross Corp, H2 2017
Pipeline by JCR Pharmaceuticals Co Ltd, H2 2017
Pipeline by Laboratorios Del Dr Esteve SA, H2 2017
Pipeline by RegenxBio Inc, H2 2017
Pipeline by Sangamo Therapeutics Inc, H2 2017
Pipeline by Shire Plc, H2 2017
Dormant Projects, H2 2017

List Of Figures


Number of Products under Development by Stage of Development, H2 2017
Number of Products under Development by Therapy Areas, H2 2017
Number of Products under Development by Indications, H2 2017
Number of Products by Mechanism of Actions, H2 2017
Number of Products by Stage and Mechanism of Actions, H2 2017
Number of Products by Routes of Administration, H2 2017
Number of Products by Stage and Routes of Administration, H2 2017
Number of Products by Molecule Types, H2 2017
Number of Products by Stage and Molecule Types, H2 2017

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