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Alpha L-Iduronidase (IDUA or EC 3.2.1.76) - Pipeline Review, H1 2017

DESCRIPTION

TABLE OF CONTENTS

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DESCRIPTION

Alpha L-Iduronidase (IDUA or EC 3.2.1.76) - Pipeline Review, H1 2017

Summary

Alpha L-Iduronidase (IDUA or EC 3.2.1.76) pipeline Target constitutes close to 9 molecules. Out of which approximately 7 molecules are developed by companies and remaining by the universities/institutes. The latest report Alpha L-Iduronidase - Pipeline Review, H1 2017, outlays 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.

Alpha L-Iduronidase (IDUA or EC 3.2.1.76) - Alpha L-iduronidase is an enzyme encoded by IDUA gene. It hydrolyzes the terminal alpha-L-iduronic acid residues of two glycosaminoglycans, dermatan sulfate and heparan sulfate. Mutations in this gene that result in enzymatic deficiency lead to the autosomal recessive disease mucopolysaccharidosis type I (MPS I). The molecules developed by companies in Phase II, Preclinical and Discovery stages are 2, 3 and 2 respectively. Similarly, the universities portfolio in Preclinical and Discovery stages comprises 1 and 1 molecules, respectively. Report covers products from therapy areas Genetic Disorders which include indications Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ).

Furthermore, this report also reviewsalso reviews key players involved in Alpha L-Iduronidase (IDUA or EC 3.2.1.76) 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 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

TABLE OF CONTENTS

Introduction
Global Markets Direct Report Coverage
Alpha L-Iduronidase (IDUA or EC 3.2.1.76) - Overview
Alpha L-Iduronidase (IDUA or EC 3.2.1.76) - 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
Alpha L-Iduronidase (IDUA or EC 3.2.1.76) - Therapeutics Assessment
Assessment by Mechanism of Action
Assessment by Route of Administration
Assessment by Molecule Type
Alpha L-Iduronidase (IDUA or EC 3.2.1.76) - Companies Involved in Therapeutics Development
AngioChem Inc
ArmaGen Inc
Bioasis Technologies Inc
Immusoft Corp
RegenxBio Inc
Sangamo Therapeutics Inc
Alpha L-Iduronidase (IDUA or EC 3.2.1.76) - Drug Profiles
AGT-181 - 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
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
X-372 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Alpha L-Iduronidase (IDUA or EC 3.2.1.76) - Dormant Products
Alpha L-Iduronidase (IDUA or EC 3.2.1.76) - Product Development Milestones
Featured News & Press Releases
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
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
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
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
Feb 08, 2016: Sangamo BioSciences Announces FDA Clearance Of Investigational New Drug Application For ZFN-Mediated Genome Editing Treatment Of MPS I
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 REGENXBIOs 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
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, H1 2017
Number of Products under Development by Therapy Areas, H1 2017
Number of Products under Development by Indication, H1 2017
Number of Products under Development by Companies, H1 2017
Products under Development by Companies, H1 2017
Number of Products under Investigation by Universities/Institutes, H1 2017
Products under Investigation by Universities/Institutes, H1 2017
Number of Products by Stage and Mechanism of Actions, H1 2017
Number of Products by Stage and Route of Administration, H1 2017
Number of Products by Stage and Molecule Type, H1 2017
Pipeline by AngioChem Inc, H1 2017
Pipeline by ArmaGen Inc, H1 2017
Pipeline by Bioasis Technologies Inc, H1 2017
Pipeline by Immusoft Corp, H1 2017
Pipeline by RegenxBio Inc, H1 2017
Pipeline by Sangamo Therapeutics Inc, H1 2017
Dormant Projects, H1 2017
List of Figures
Number of Products under Development by Stage of Development, 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
Introduction
Global Markets Direct Report Coverage
Global Markets Direct’s, ‘Alpha L-Iduronidase (IDUA or EC 3.2.1.76) H1 2017‘, 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, features dormant and discontinued projects 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 with respective active and dormant or discontinued projects.
Alpha L-Iduronidase (IDUA or EC 3.2.1.76) pipeline Target constitutes close to 9 molecules. Out of which approximately 7 molecules are developed by companies and remaining by the universities/institutes. The molecules developed by companies in Phase II, Preclinical and Discovery stages are 2, 3 and 2 respectively. Similarly, the universities portfolio in Preclinical and Discovery stages comprises 1 and 1 molecules, respectively. Report covers products from therapy areas Genetic Disorders which include indications Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ).
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.
Alpha L-Idu

List Of Figures

Number of Products under Development by Stage of Development, 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

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