Alpha L-Iduronidase (IDUA or EC 3.2.1.76) - Pipeline Review, H2 2016

Publisher Name :
Date: 31-Aug-2016
No. of pages: 51

Alpha L-Iduronidase (IDUA or EC 3.2.1.76) - Pipeline Review, H2 2016

Summary

Global Markets Direct's, ‘Alpha L-Iduronidase (IDUA or EC 3.2.1.76) - Pipeline Review, H2 2016', provides in depth analysis on Alpha L-Iduronidase (IDUA or EC 3.2.1.76) targeted pipeline therapeutics.

The report provides comprehensive information on the Alpha L-Iduronidase (IDUA or EC 3.2.1.76), targeted therapeutics, complete with analysis by indications, stage of development, mechanism of action (MoA), route of administration (RoA) and molecule type. The report also covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases. Additionally, the report provides an overview of key players involved in Alpha L-Iduronidase (IDUA or EC 3.2.1.76) targeted therapeutics development and features dormant and discontinued projects.

Global Markets Direct's report features investigational drugs from across globe covering over 20 therapy areas and nearly 3,000 indications. The report 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. Drug profiles featured in the report undergoes periodic review following a stringent set of processes to ensure that all the profiles are updated with the latest set of information. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.

The report 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.

Note:

*Certain sections in the report may be removed or altered based on the availability and relevance of data.

*Updated report will be delivered in 48 hours of order confirmation.

Scope

- The report provides a snapshot of the global therapeutic landscape for Alpha L-Iduronidase (IDUA or EC 3.2.1.76)

- The report reviews Alpha L-Iduronidase (IDUA or EC 3.2.1.76) 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 Alpha L-Iduronidase (IDUA or EC 3.2.1.76) targeted therapeutics and enlists all their major and minor projects

- The report assesses Alpha L-Iduronidase (IDUA or EC 3.2.1.76) 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 Alpha L-Iduronidase (IDUA or EC 3.2.1.76) 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 Alpha L-Iduronidase (IDUA or EC 3.2.1.76)

- 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 it's most promising pipeline therapeutics

- Devise corrective measures for pipeline projects by understanding Alpha L-Iduronidase (IDUA or EC 3.2.1.76) 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

Alpha L-Iduronidase (IDUA or EC 3.2.1.76) - Pipeline Review, H2 2016

Table of Contents
Table of Contents 2
List of Tables 4
List of Figures 4
Introduction 5
Global Markets Direct Report Coverage 5
Alpha L-Iduronidase (IDUA or EC 3.2.1.76) Overview 6
Therapeutics Development 7
Alpha L-Iduronidase (IDUA or EC 3.2.1.76) - Products under Development by Stage of Development 7
Alpha L-Iduronidase (IDUA or EC 3.2.1.76) - Products under Development by Therapy Area 8
Alpha L-Iduronidase (IDUA or EC 3.2.1.76) - Products under Development by Indication 9
Alpha L-Iduronidase (IDUA or EC 3.2.1.76) - Pipeline Products Glance 10
Late Stage Products 10
Early Stage Products 11
Alpha L-Iduronidase (IDUA or EC 3.2.1.76) - Products under Development by Companies 12
Alpha L-Iduronidase (IDUA or EC 3.2.1.76) - Products under Development by Universities/Institutes 14
Alpha L-Iduronidase (IDUA or EC 3.2.1.76) - Therapeutics Assessment 16
Assessment by Monotherapy/Combination Products 16
Assessment by Mechanism of Action 17
Assessment by Route of Administration 19
Assessment by Molecule Type 21
Alpha L-Iduronidase (IDUA or EC 3.2.1.76) - Companies Involved in Therapeutics Development 23
AngioChem Inc. 23
ArmaGen Inc. 24
Bioasis Technologies Inc. 25
RegenxBio Inc. 26
Sangamo BioSciences, Inc. 27
Alpha L-Iduronidase (IDUA or EC 3.2.1.76) - Drug Profiles 28
AGT-181 - Drug Profile 28
Product Description 28
Mechanism Of Action 28
R&D Progress 28
Cell Therapy for Mucopolysaccharidosis I - Drug Profile 30
Product Description 30
Mechanism Of Action 30
R&D Progress 30
Cell Therapy to Activate Alpha L-Iduronidase for Hurler Syndrome - Drug Profile 31
Product Description 31
Mechanism Of Action 31
R&D Progress 31
Recombinant Alpha-L-Iduronidase Replacement for Hurler Syndrome - Drug Profile 32
Product Description 32
Mechanism Of Action 32
R&D Progress 32
Recombinant Alpha-L-Iduronidase Replacement for Mucopolysaccharidosis I - Drug Profile 33
Product Description 33
Mechanism Of Action 33
R&D Progress 33
Recombinant Alpha-L-Iduronidase Replacement for Mucopolysaccharidosis I - Drug Profile 34
Product Description 34
Mechanism Of Action 34
R&D Progress 34
RGX-111 - Drug Profile 35
Product Description 35
Mechanism Of Action 35
R&D Progress 35
SB-318 - Drug Profile 37
Product Description 37
Mechanism Of Action 37
R&D Progress 37
X-372 - Drug Profile 39
Product Description 39
Mechanism Of Action 39
R&D Progress 39
Alpha L-Iduronidase (IDUA or EC 3.2.1.76) - Dormant Projects 40
Alpha L-Iduronidase (IDUA or EC 3.2.1.76) - Featured News & Press Releases 41
Jul 05, 2016: REGENXBIO Provides Update On Gene Therapy Development Program RGX-111 41
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 41
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 43
Feb 08, 2016: Sangamo BioSciences Announces FDA Clearance Of Investigational New Drug Application For ZFN-Mediated Genome Editing Treatment Of MPS I 44
Dec 30, 2015: FDA Grants Rare Pediatric Disease Designation to REGENXBIO RGX-111 Gene Therapy for the Treatment of Mucopolysaccharidosis Type I (MPS I) 45
Nov 05, 2015: ArmaGen Receives Rare Pediatric Disease Designation from FDA for AGT-181 for the Potential Treatment of Hurler Syndrome 45
Oct 01, 2015: FDA Grants Orphan Drug Designation to REGENXBIO's RGX-111 Gene Therapy for the Treatment of Mucopolysaccharidosis Type I 46
Sep 02, 2015: ArmaGen Announces First Patient Dosed in Phase 1/2a Clinical Trial of AGT-181 for the Treatment of Hurler Syndrome 46
Apr 08, 2015: ArmaGen Announces FDA Acceptance of IND Application for AGT-181 for the Treatment of Hurler Syndrome 47
Mar 10, 2015: PacificGMP and ArmaGen Announce Successful Manufacture of ArmaGen's investigational enzyme replacement therapy, AGT-181, for Phase 1 Clinical Trial 48
Feb 05, 2014: Mouse Study Shows Gene Therapy May Be Possible Cure for Hurler Syndrome 48
Sep 18, 2009: ArmaGen Completes Primate Safety Study For AGT-181 49
Appendix 50
Methodology 50
Coverage 50
Secondary Research 50
Primary Research 50
Expert Panel Validation 50
Contact Us 50
Disclaimer 51

List of Tables
Number of Products under Development for, H2 2016 7
Number of Products under Development by Therapy Area, H2 2016 8
Number of Products under Development by Indication, H2 2016 9
Comparative Analysis by Late Stage Development, H2 2016 10
Comparative Analysis by Early Stage Products, H2 2016 11
Number of Products under Development by Companies, H2 2016 12
Products under Development by Companies, H2 2016 13
Number of Products under Investigation by Universities/Institutes, H2 2016 14
Products under Investigation by Universities/Institutes, H2 2016 15
Assessment by Monotherapy/Combination Products, H2 2016 16
Number of Products by Stage and Mechanism of Action, H2 2016 18
Number of Products by Stage and Route of Administration, H2 2016 20
Number of Products by Stage and Molecule Type, H2 2016 22
Pipeline by AngioChem Inc., H2 2016 23
Pipeline by ArmaGen Inc., H2 2016 24
Pipeline by Bioasis Technologies Inc., H2 2016 25
Pipeline by RegenxBio Inc., H2 2016 26
Pipeline by Sangamo BioSciences, Inc., H2 2016 27
Dormant Projects, H2 2016 40

List of Figures
Number of Products under Development for, H2 2016 7
Comparative Analysis by Early Stage Products, H2 2016 11
Assessment by Monotherapy/Combination Products, H2 2016 16
Number of Products by Mechanism of Actions, H2 2016 17
Number of Products by Stage and Mechanism of Actions, H2 2016 17
Number of Products by Routes of Administration, H2 2016 19
Number of Products by Stage and Routes of Administration, H2 2016 19
Number of Products by Molecule Types, H2 2016 21
Number of Products by Stage and Molecule Type, H2 2016 21
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