Marktanalyse - Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline Review, H2 2016

Global Markets Direct
12.2016
71 Seiten

 
Typ:
Marktanalyse
Verfüg­barkeit:
verfügbar
Regionen/­Länder:
  • Europa
  • Asien / Pazifik
  • Mittlerer Osten / Afrika
  • Nordamerika / USA
  • Australien
  • Mittel- / Südamerika
Sprache:
Englisch

Bitte wählen Sie ein Lieferformat und klicken Sie unten auf einen Bestellbutton:

PDF-Datei per E-Mail, Single User Price, versandkostenfrei
PDF-Datei per E-Mail, Site License Price**, versandkostenfrei
PDF-Datei per E-Mail, Enterprisewide Price (Global Site License)***, versandkostenfrei


Hinweise: **The report can be shared by users within one office (one geographical location) ***Across Geographical locations (by the purchasing company only)

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline Review, H2 2016


Summary


Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) — Pipeline Review, H2 2016, provides an overview of the Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) (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 (MPS III) (Sanfilippo Syndrome ) — Pipeline Review, H2 2016, provides comprehensive information on the therapeutics under development for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) (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 (MPS III) (Sanfilippo Syndrome ) (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 7, 1, 7 and 3 respectively. Similarly, the Universities portfolio in Preclinical stages comprises 1 molecules, respectively.

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) (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 (MPS III) (Sanfilippo Syndrome ) (Metabolic Disorders).
- The pipeline guide reviews pipeline therapeutics for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) (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 (MPS III) (Sanfilippo Syndrome ) (Metabolic Disorders) therapeutics and enlists all their major and minor projects.
- The pipeline guide evaluates Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) (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 (MPS III) (Sanfilippo Syndrome ) (Metabolic 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 ) (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 (MPS III) (Sanfilippo Syndrome ) (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.
Table of Contents

Table of Contents 2

List of Tables 5

List of Figures 6

Introduction 7

Global Markets Direct Report Coverage 7

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) Overview 8

Therapeutics Development 9

Pipeline Products for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Overview 9

Pipeline Products for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Comparative Analysis 10

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Therapeutics under Development by Companies 11

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Therapeutics under Investigation by Universities/Institutes 12

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline Products Glance 13

Clinical Stage Products 13

Early Stage Products 14

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Products under Development by Companies 15

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Products under Investigation by Universities/Institutes 16

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Companies Involved in Therapeutics Development 17

Abeona Therapeutics Inc 17

Alexion Pharmaceuticals Inc 18

ArmaGen Inc 19

Axcentua Pharmaceuticals AB 20

BioMarin Pharmaceutical Inc 21

Dorphan SA 22

Laboratorios Del Dr Esteve SA 23

Lysogene SAS 24

Swedish Orphan Biovitrum AB 25

UniQure NV 26

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Therapeutics Assessment 27

Assessment by Monotherapy Products 27

Assessment by Target 28

Assessment by Mechanism of Action 30

Assessment by Route of Administration 32

Assessment by Molecule Type 34

Drug Profiles 36

ABO-101 - Drug Profile 36

Product Description 36

Mechanism Of Action 36

R&D Progress 36

ABO-102 - Drug Profile 38

Product Description 38

Mechanism Of Action 38

R&D Progress 38

AGT-184 - Drug Profile 41

Product Description 41

Mechanism Of Action 41

R&D Progress 41

AMT-110 - Drug Profile 42

Product Description 42

Mechanism Of Action 42

R&D Progress 42

AXP-10711 - Drug Profile 44

Product Description 44

Mechanism Of Action 44

R&D Progress 44

BMN-250 - Drug Profile 46

Product Description 46

Mechanism Of Action 46

R&D Progress 46

EGT-101 - Drug Profile 48

Product Description 48

Mechanism Of Action 48

R&D Progress 48

EGT-201 - Drug Profile 49

Product Description 49

Mechanism Of Action 49

R&D Progress 49

lesinidase alfa - Drug Profile 50

Product Description 50

Mechanism Of Action 50

R&D Progress 50

LYSSAF-301 - Drug Profile 53

Product Description 53

Mechanism Of Action 53

R&D Progress 53

LYSSAF-302 - Drug Profile 54

Product Description 54

Mechanism Of Action 54

R&D Progress 54

Proteins for Mucopolysaccharidosis Type IIIC - Drug Profile 55

Product Description 55

Mechanism Of Action 55

R&D Progress 55

Recombinant N-Acetylgalactosamine-6-Sulfatase Replacement for Mucopolysaccharidosis Type IIID - Drug Profile 56

Product Description 56

Mechanism Of Action 56

R&D Progress 56

Small Molecule for Sanfilippo Syndrome - Drug Profile 57

Product Description 57

Mechanism Of Action 57

R&D Progress 57

Small Molecule to Target Alpha-N-Acetylglucosaminidase for MPSIIIB - Drug Profile 58

Product Description 58

Mechanism Of Action 58

R&D Progress 58

SOBI-003 - Drug Profile 59

Product Description 59

Mechanism Of Action 59

R&D Progress 59

Stem Cell Therapy for Mucopolysaccharidosis Type IIIA - Drug Profile 60

Product Description 60

Mechanism Of Action 60

R&D Progress 60

Stem Cell Therapy for Sanfilippo Syndrome - Drug Profile 61

Product Description 61

Mechanism Of Action 61

R&D Progress 61

Stem Cell Therapy to Activate NAGLU for Mucopolysaccharidosis IIIB - Drug Profile 62

Product Description 62

Mechanism Of Action 62

R&D Progress 62

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Dormant Projects 63

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Discontinued Products 64

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Product Development Milestones 65

Featured News & Press Releases 65

Oct 19, 2016: European Commission Grants SOBI003 Orphan Designation for the Treatment of MPS IIIA 65

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) 65

Feb 25, 2016: ESTEVE Provides Update On EGT-201 For Sanfilippo B Syndrome 66

Jan 11, 2016: Abeona Therapeutics Announces Initial European Regulatory Approval for Phase 1/2 Gene Therapy Clinical Study for Patients With Sanfilippo Syndrome 67

May 20, 2015: PlasmaTech Biopharmaceuticals Announces Orphan Drug and Rare Pediatric Disease Designations For ABX-101 From FDA 68

Apr 02, 2014: Orphan designation granted for MPSIII 68

Jul 25, 2013: New stem cell gene therapy gives hope to prevent inherited neurological disease 68

Apr 11, 2013: Axcentua granted patent on ten solid forms of genistein 69

Appendix 70

Methodology 70

Coverage 70

Secondary Research 70

Primary Research 70

Expert Panel Validation 70

Contact Us 70

Disclaimer 71





List of Tables

Number of Products under Development for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ), H2 2016 9

Number of Products under Development for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Comparative Analysis, H2 2016 10

Number of Products under Development by Companies, H2 2016 11

Number of Products under Investigation by Universities/Institutes, H2 2016 12

Comparative Analysis by Clinical Stage Development, H2 2016 13

Comparative Analysis by Early Stage Development, H2 2016 14

Products under Development by Companies, H2 2016 15

Products under Investigation by Universities/Institutes, H2 2016 16

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by Abeona Therapeutics Inc, H2 2016 17

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by Alexion Pharmaceuticals Inc, H2 2016 18

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by ArmaGen Inc, H2 2016 19

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by Axcentua Pharmaceuticals AB, H2 2016 20

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by BioMarin Pharmaceutical Inc, H2 2016 21

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by Dorphan SA, H2 2016 22

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by Laboratorios Del Dr Esteve SA, H2 2016 23

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by Lysogene SAS, H2 2016 24

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by Swedish Orphan Biovitrum AB, H2 2016 25

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by UniQure NV, H2 2016 26

Assessment by Monotherapy Products, H2 2016 27

Number of Products by Stage and Target, H2 2016 29

Number of Products by Stage and Mechanism of Action, H2 2016 31

Number of Products by Stage and Route of Administration, H2 2016 33

Number of Products by Stage and Molecule Type, H2 2016 35

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Dormant Projects, H2 2016 63

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Discontinued Products, H2 2016 64





List of Figures

Number of Products under Development for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ), H2 2016 9

Number of Products under Development for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Comparative Analysis, H2 2016 10

Number of Products under Development by Companies, H2 2016 11

Comparative Analysis by Early Stage Products, H2 2016 14

Assessment by Monotherapy Products, H2 2016 27

Number of Products by Targets, H2 2016 28

Number of Products by Stage and Targets, H2 2016 28

Number of Products by Top 10 Mechanism of Actions, H2 2016 30

Number of Products by Stage and Top 10 Mechanism of Actions, H2 2016 30

Number of Products by Routes of Administration, H2 2016 32

Number of Products by Stage and Routes of Administration, H2 2016 32

Number of Products by Molecule Types, H2 2016 34

Number of Products by Stage and Molecule Types, H2 2016 34

* Alle Preise sind netto ausgewiesen. In Abhängigkeit von Ihrer Rechnungsanschrift ist hierauf noch USt. zu entrichten (Deutschland z.Z. 19%). Unser Angebot richtet sich ausschließlich an Unternehmen, Gewerbetreibende und Freiberufler.

 

Über marktforschung.de

Branchenwissen an zentraler Stelle bündeln und abrufbar machen – das ist das Hauptanliegen von marktforschung.de. Unser breites Informationsangebot rund um die Marktforschung richtet sich sowohl an Marktforschungsinstitute, Felddienstleister, Panelbetreiber und Herausgeber von Studien, Marktdaten sowie Marktanalysen als auch an deren Kunden aus Industrie, Handel und Dienstleistungsgewerbe.

facebook twitter google plus