Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) – Pipeline Review, H1 2017

Global Markets Direct’s, ‘Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) – Pipeline Review, H1 2017’, provides an overview of the Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) pipeline landscape.

The report provides comprehensive information on the therapeutics under development for Mucopolysaccharidosis III (MPS III) (Sanfilippo 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 III (MPS III) (Sanfilippo 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 III (MPS III) (Sanfilippo Syndrome )

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

The report assesses Mucopolysaccharidosis III (MPS III) (Sanfilippo 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 III (MPS III) (Sanfilippo 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 III (MPS III) (Sanfilippo 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 III (MPS III) (Sanfilippo 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

Abeona Therapeutics Inc

Alexion Pharmaceuticals Inc

ArmaGen Inc

Axcentua Pharmaceuticals AB

BioMarin Pharmaceutical Inc

Laboratorios Del Dr Esteve SA

Lysogene SAS

Swedish Orphan Biovitrum AB

Table of Contents

Table of Contents

List of Tables

List of Figures

Introduction

Global Markets Direct Report Coverage

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Overview

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Therapeutics Development

Pipeline Overview

Pipeline by Companies

Pipeline by Universities/Institutes

Products under Development by Companies

Products under Development by Universities/Institutes

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Therapeutics Assessment

Assessment by Target

Assessment by Mechanism of Action

Assessment by Route of Administration

Assessment by Molecule Type

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Companies Involved in Therapeutics Development

Abeona Therapeutics Inc

Alexion Pharmaceuticals Inc

ArmaGen Inc

Axcentua Pharmaceuticals AB

BioMarin Pharmaceutical Inc

Laboratorios Del Dr Esteve SA

Lysogene SAS

Swedish Orphan Biovitrum AB

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Drug Profiles

ABO-101 - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

ABO-102 - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

AGT-184 - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

AXP-10711 - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

BMN-250 - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

EGT-101 - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

EGT-201 - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

lesinidase alfa - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

LYSSAF-301 - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

LYSSAF-302 - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Proteins for Mucopolysaccharidosis Type IIIC - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Recombinant N-Acetylgalactosamine-6-Sulfatase Replacement for Mucopolysaccharidosis Type IIID - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Small Molecule for Sanfilippo Syndrome - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

SOBI-003 - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Stem Cell Therapy for Mucopolysaccharidosis Type IIIA - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Stem Cell Therapy for Sanfilippo Syndrome - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Stem Cell Therapy to Activate NAGLU for Mucopolysaccharidosis IIIB - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Dormant Projects

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Discontinued Products

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Product Development Milestones

Featured News & Press Releases

Jan 19, 2017: Abeona Therapeutics Receives Orphan Drug Designation in The European Union for ABO-101 Gene Therapy in Sanfilippo Syndrome Type B

Oct 19, 2016: European Commission Grants SOBI003 Orphan Designation for the Treatment of MPS IIIA

May 24, 2016: Abeona Therapeutics Announces FDA Allowance of Investigational New Drug for Phase 1/2 Clinical Study With ABO-101 Gene Therapy for Patients With Sanfilippo Syndrome Type B (MPS IIIB)

Feb 25, 2016: ESTEVE Provides Update On EGT-201 For Sanfilippo B Syndrome

Jan 11, 2016: Abeona Therapeutics Announces Initial European Regulatory Approval for Phase 1/2 Gene Therapy Clinical Study for Patients With Sanfilippo Syndrome

May 20, 2015: PlasmaTech Biopharmaceuticals Announces Orphan Drug and Rare Pediatric Disease Designations For ABX-101 From FDA

Apr 02, 2014: Orphan designation granted for MPSIII

Jul 25, 2013: New stem cell gene therapy gives hope to prevent inherited neurological disease

Apr 11, 2013: Axcentua granted patent on ten solid forms of genistein

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 III (MPS III) (Sanfilippo Syndrome ), H1 2017

Number of Products under Development by Companies, H1 2017

Number of Products under Development by Universities/Institutes, H1 2017

Products under Development by Companies, H1 2017

Products under Development by Universities/Institutes, H1 2017

Number of Products by Stage and Target, H1 2017

Number of Products by Stage and Mechanism of Action, H1 2017

Number of Products by Stage and Route of Administration, H1 2017

Number of Products by Stage and Molecule Type, H1 2017

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) – Pipeline by Abeona Therapeutics Inc, H1 2017

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) – Pipeline by Alexion Pharmaceuticals Inc, H1 2017

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) – Pipeline by ArmaGen Inc, H1 2017

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) – Pipeline by Axcentua Pharmaceuticals AB, H1 2017

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) – Pipeline by BioMarin Pharmaceutical Inc, H1 2017

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) – Pipeline by Laboratorios Del Dr Esteve SA, H1 2017

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) – Pipeline by Lysogene SAS, H1 2017

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) – Pipeline by Swedish Orphan Biovitrum AB, H1 2017

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) – Dormant Projects, H1 2017

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) – Discontinued Products, H1 2017

List of Figures

List of Figures

Number of Products under Development for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ), H1 2017

Number of Products under Development by Companies, H1 2017

Number of Products by Targets, H1 2017

Number of Products by Stage and Targets, 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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