Mucopolysaccharidosis III - Pipeline Review, H1 2017
Mucopolysaccharidosis III - Pipeline Review, H1 2017
SUMMARY
Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Mucopolysaccharidosis III - Pipeline Review, H1 2017, provides an overview of the Mucopolysaccharidosis III (Metabolic 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 - Pipeline Review, H1 2017, provides comprehensive information on the therapeutics under development for Mucopolysaccharidosis III (Metabolic 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 (Metabolic 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. Similarly, the Universities portfolio in Preclinical stages comprises 1 molecules, respectively.
Mucopolysaccharidosis III (Metabolic 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
SUMMARY
Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Mucopolysaccharidosis III - Pipeline Review, H1 2017, provides an overview of the Mucopolysaccharidosis III (Metabolic 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 - Pipeline Review, H1 2017, provides comprehensive information on the therapeutics under development for Mucopolysaccharidosis III (Metabolic 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 (Metabolic 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. Similarly, the Universities portfolio in Preclinical stages comprises 1 molecules, respectively.
Mucopolysaccharidosis III (Metabolic 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 (Metabolic Disorders).
- The pipeline guide reviews pipeline therapeutics for Mucopolysaccharidosis III (Metabolic 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 (Metabolic Disorders) therapeutics and enlists all their major and minor projects.
- The pipeline guide evaluates Mucopolysaccharidosis III (Metabolic 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 (Metabolic Disorders)
- 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 (Metabolic 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 (Metabolic 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.
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
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
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
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
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
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
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
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
