Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline Review, H1 2018

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Date: 12-Jun-2018
No. of pages: 68
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Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline Review, H1 2018

Summary

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline Review, H1 2018, provides an overview of the Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) (Genetic Disorders) pipeline landscape.

Mucopolysaccharidosis type III (MPS III), also known as Sanfilippo syndrome, is an inherited metabolic disease caused by an absence or malfunctioning of certain enzymes needed to breakdown molecules called glycosaminoglycans (GAG). Symptoms include seizures, hyperactivity, liver and spleen enlargement, severe diarrhea or constipation and enlargement of tonsils and adenoids. Treatment includes enzyme replacement therapy (ERT).

Report Highlights

Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline Review, H1 2018, provides comprehensive information on the therapeutics under development for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) (Genetic Disorders), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The guide covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases.

The Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) (Genetic Disorders) pipeline guide also reviews of key players involved in therapeutic development for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Phase II, IND/CTA Filed, Preclinical and Discovery stages are 6, 1, 6 and 3 respectively.

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) (Genetic Disorders) pipeline guide 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. The guide 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. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.

Note: Certain content / sections in the pipeline guide may be removed or altered based on the availability and relevance of data.

Scope

- The pipeline guide provides a snapshot of the global therapeutic landscape of Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) (Genetic Disorders).

- The pipeline guide reviews pipeline therapeutics for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) (Genetic Disorders) by companies and universities/research institutes based on information derived from company and industry-specific sources.

- The pipeline guide covers pipeline products based on several stages of development ranging from pre-registration till discovery and undisclosed stages.

- The pipeline guide features descriptive drug profiles for the pipeline products which comprise, product description, descriptive licensing and collaboration details, R&D brief, MoA & other developmental activities.

- The pipeline guide reviews key companies involved in Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) (Genetic Disorders) therapeutics and enlists all their major and minor projects.

- The pipeline guide evaluates Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) (Genetic Disorders) therapeutics based on mechanism of action (MoA), drug target, route of administration (RoA) and molecule type.

- The pipeline guide encapsulates all the dormant and discontinued pipeline projects.

- The pipeline guide reviews latest news related to pipeline therapeutics for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) (Genetic Disorders)

Reasons to buy

- Procure strategically important competitor information, analysis, and insights to formulate effective R&D strategies.

- Recognize emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage.

- Find and recognize significant and varied types of therapeutics under development for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) (Genetic Disorders).

- Classify potential new clients or partners in the target demographic.

- Develop tactical initiatives by understanding the focus areas of leading companies.

- Plan mergers and acquisitions meritoriously by identifying key players and it's most promising pipeline therapeutics.

- Formulate corrective measures for pipeline projects by understanding Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) (Genetic Disorders) 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.

- Adjust the therapeutic portfolio by recognizing discontinued projects and understand from the know-how what drove them from pipeline.

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline Review, H1 2018

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
Number of Products under Development for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ), H1 2018
Number of Products under Development by Companies, H1 2018
Products under Development by Companies, H1 2018
Products under Development by Companies, H1 2018 (Contd..1), H1 2018
Number of Products by Stage and Target, H1 2018
Number of Products by Stage and Mechanism of Action, H1 2018
Number of Products by Stage and Route of Administration, H1 2018
Number of Products by Stage and Molecule Type, H1 2018
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by Abeona Therapeutics Inc, H1 2018
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by ArmaGen Inc, H1 2018
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by Axcentua Pharmaceuticals AB, H1 2018
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by BioMarin Pharmaceutical Inc, H1 2018
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by Laboratorios Del Dr Esteve SA, H1 2018
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by Lysogene SAS, H1 2018
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by Swedish Orphan Biovitrum AB, H1 2018
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Dormant Projects, H1 2018
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Discontinued Products, H1 2018

List of Figures
Number of Products under Development for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ), H1 2018
Number of Products under Development by Companies, H1 2018
Number of Products by Targets, H1 2018
Number of Products by Stage and Targets, H1 2018
Number of Products by Mechanism of Actions, H1 2018
Number of Products by Stage and Mechanism of Actions, H1 2018
Number of Products by Routes of Administration, H1 2018
Number of Products by Stage and Routes of Administration, H1 2018
Number of Products by Molecule Types, H1 2018
Number of Products by Stage and Molecule Types, H1 2018
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