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

Global Markets Direct’s, ‘Alpha L-Iduronidase (IDUA or EC 3.2.1.76) – Pipeline Review, 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 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. Alpha L-Iduronidase (IDUA or EC 3.2.1.76)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. The report analyses the pipeline products from therapy areas Genetic Disorders under development targeting Alpha L-Iduronidase (IDUA or EC 3.2.1.76)

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

Companies mentioned

AngioChem Inc

ArmaGen Inc

Bioasis Technologies Inc

Immusoft Corp

RegenxBio Inc

Sangamo Therapeutics Inc

Table of Contents

Table of Contents

List of Figures

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 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

Appendix

Methodology

Coverage

Secondary Research

Primary Research

Expert Panel Validation

Contact Us

Disclaimer

List of Tables

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

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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