Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) – Pipeline Review, H2 2017

Global Markets Direct’s, ‘Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) – Pipeline Review, H2 2017’, provides an overview of the Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) pipeline landscape.

The report provides comprehensive information on the therapeutics under development for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ), complete with analysis by stage of development, drug target, 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 therapeutic development for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) 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.

Scope

The report provides a snapshot of the global therapeutic landscape of Mucopolysaccharidosis I (MPS I) (Hurler Syndrome )

The report reviews pipeline therapeutics for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) 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 Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) therapeutics and enlists all their major and minor projects

The report assesses Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) therapeutics based on drug target, 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 related to pipeline therapeutics for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome )

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 important and diverse types of therapeutics under development for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome )

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 Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) 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

Modify the therapeutic portfolio by identifying discontinued projects and understanding the factors that drove them from pipeline

Companies mentioned

AngioChem Inc

ArmaGen Inc

CRISPR Therapeutics

Immusoft Corp

JCR Pharmaceuticals Co Ltd

OPKO Health Inc

PTC Therapeutics Inc

RegenxBio Inc

Sangamo Therapeutics Inc

Sevion Therapeutics Inc (Inactive)

Tamid Bio Inc

Table of Contents

Table of Contents

List of Tables

List of Figures

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

Immusoft Corp

JCR Pharmaceuticals Co Ltd

OPKO Health Inc

PTC Therapeutics Inc

RegenxBio Inc

Sangamo Therapeutics Inc

Sevion Therapeutics Inc (Inactive)

Tamid Bio 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

ataluren 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

ELX-02 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

JOT-102 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

JR-171 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 for Hurler Sydrome Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Stem Cell Therapy for Type1 Mucopolysaccharidosis 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

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

Nov 07, 2016: Eloxx Pharmaceuticals Announces Orphan Drug Designation in the U.S. and Europe for ELX-02 in Mucopolysacchardisis Type 1 (MPS 1)

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

Appendix

Methodology

Coverage

Secondary Research

Primary Research

Expert Panel Validation

Contact Us

Disclaimer

List of Tables

List of Tables

Number of Products under Development for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ), H2 2017

Number of Products under Development by Companies, H2 2017

Number of Products under Development by Universities/Institutes, H2 2017

Products under Development by Companies, H2 2017

Products under Development by Universities/Institutes, H2 2017

Number of Products by Stage and Target, H2 2017

Number of Products by Stage and Mechanism of Action, H2 2017

Number of Products by Stage and Route of Administration, H2 2017

Number of Products by Stage and Molecule Type, H2 2017

Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) Pipeline by AngioChem Inc, H2 2017

Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) Pipeline by ArmaGen Inc, H2 2017

Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) Pipeline by CRISPR Therapeutics, H2 2017

Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) Pipeline by Immusoft Corp, H2 2017

Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) Pipeline by JCR Pharmaceuticals Co Ltd, H2 2017

Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) Pipeline by OPKO Health Inc, H2 2017

Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) Pipeline by PTC Therapeutics Inc, H2 2017

Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) Pipeline by RegenxBio Inc, H2 2017

Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) Pipeline by Sangamo Therapeutics Inc, H2 2017

Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) Pipeline by Sevion Therapeutics Inc (Inactive), H2 2017

Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) Pipeline by Tamid Bio Inc, H2 2017

Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) Dormant Projects, H2 2017

Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) Discontinued Products, H2 2017

List of Figures

List of Figures

Number of Products under Development for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ), H2 2017

Number of Products under Development by Companies, H2 2017

Number of Products under Development by Universities/Institutes, H2 2017

Number of Products by Stage and Targets, 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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