Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) – Pipeline Review, H2 2019

Global Markets Direct’s, ‘Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) – Pipeline Review, H2 2019’, 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

Allievex Corp

Amicus Therapeutics Inc

ArmaGen Inc

Bioasis Technologies Inc

Esteve Pharmaceuticals SA

JCR Pharmaceuticals Co Ltd

Jupiter Orphan Therapeutics Inc

Lacerta Therapeutics Inc

Lysogene SAS

Orchard Therapeutics Plc

Phoenix Nest Inc

Seelos Therapeutics, Inc.

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

Products under Development by Companies

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

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

AGT-187 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

LYSSAF-301 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

LYSSAF-302 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

OTL-201 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

OTL-202 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

SOBI-003 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

Feb 07, 2018: Abeona Therapeutics Reports on Initial Safety and Biopotency Signals in MPS IIIB Gene Therapy Clinical Trial

Feb 07, 2018: BioMarin Presents Interim Data of Phase 1/2 Study of BMN 250 for Treatment of Sanfilippo B Syndrome (MPS IIIB) at WORLDSymposium 2018

Jan 23, 2018: FDA accepts investigational new drug application and grants Fast Track status for SOBI003 for the treatment of MPS IIIA

Dec 20, 2017: Abeona Therapeutics Enrolls First Patient in ABO-101 Phase 1/2 Clinical Trial for MPS IIIB

Sep 06, 2017: BioMarin Presents Interim Data of Phase 1/2 Study of BMN 250 for Treatment of Sanfilippo B Syndrome at 13th International Congress of Inborn Errors of Metabolism 2017

Sep 05, 2017: BioMarin To Present Data On ICV BMN 250 At 13th International Congress of Inborn Errors of Metabolism 2017

Jul 05, 2017: FDA grants SOBI003 Orphan Drug Designation for the treatment of MPS IIIA

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)

Apr 21, 2016: BioMarin Enrolls First Patient in Phase 1/2 Trial of NAGLU Fusion Protein BMN 250 for Treatment of MPS IIIB (Sanfilippo B Syndrome)

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

Dec 10, 2014: FDA Grants BioMarin Orphan Drug Designation for NAGLU Fusion Protein, BMN 250, for the Treatment of MPS IIIB (Sanfilippo Syndrome Type B)

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 ), H2 2019

Number of Products under Development by Companies, H2 2019

Products under Development by Companies, H2 2019

Products under Development by Companies, H2 2019 (Contd..1), H2 2019

Number of Products by Stage and Target, H2 2019

Number of Products by Stage and Mechanism of Action, H2 2019

Number of Products by Stage and Route of Administration, H2 2019

Number of Products by Stage and Molecule Type, H2 2019

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) – Pipeline by Abeona Therapeutics Inc, H2 2019

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) – Dormant Projects, H2 2019

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) – Discontinued Products, H2 2019

List of Figures

List of Figures

Number of Products under Development for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ), H2 2019

Number of Products under Development by Companies, H2 2019

Number of Products by Targets, H2 2019

Number of Products by Stage and Targets, H2 2019

Number of Products by Top 10 Mechanism of Actions, H2 2019

Number of Products by Stage and Top 10 Mechanism of Actions, H2 2019

Number of Products by Routes of Administration, H2 2019

Number of Products by Stage and Routes of Administration, H2 2019

Number of Products by Molecule Types, H2 2019

Number of Products by Stage and Molecule Types, H2 2019

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