Marktanalyse - Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline Review, H2 2016

Global Markets Direct
12.2016
79 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 I (MPS I) (Hurler Syndrome ) - Pipeline Review, H2 2016


Summary


Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) — Pipeline Review, H2 2016, provides an overview of the Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) (Metabolic Disorders) pipeline landscape.

MPS I (Mucopolysaccharidosis I) is an inherited lysosomal storage disorder caused by a deficiency of alpha-L-iduronidase, a lysosomal enzyme normally required for the breakdown of certain complex carbohydrates known as glycosaminoglycans (GAGs). Symptoms include abnormal bones in the spine, claw hand, cloudy corneas, deafness and heart valve problems. Treatment includes bone marrow transplantation, enzyme therapy and gene therapy.

Report Highlights

Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) — Pipeline Review, H2 2016, provides comprehensive information on the therapeutics under development for Mucopolysaccharidosis I (MPS I) (Hurler 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 I (MPS I) (Hurler Syndrome ) (Metabolic Disorders) pipeline guide also reviews of key players involved in therapeutic development for Mucopolysaccharidosis I (MPS I) (Hurler 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, Phase I, IND/CTA Filed, Preclinical and Discovery stages are 4, 2, 1, 5 and 3 respectively. Similarly, the Universities portfolio in Preclinical and Discovery stages comprises 2 and 1 molecules, respectively.

Mucopolysaccharidosis I (MPS I) (Hurler 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 I (MPS I) (Hurler Syndrome ) (Metabolic Disorders).
- The pipeline guide reviews pipeline therapeutics for Mucopolysaccharidosis I (MPS I) (Hurler 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 I (MPS I) (Hurler Syndrome ) (Metabolic Disorders) therapeutics and enlists all their major and minor projects.
- The pipeline guide evaluates Mucopolysaccharidosis I (MPS I) (Hurler 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 I (MPS I) (Hurler 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 I (MPS I) (Hurler 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 I (MPS I) (Hurler 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 I (MPS I) (Hurler Syndrome ) Overview 8

Therapeutics Development 9

Pipeline Products for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Overview 9

Pipeline Products for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Comparative Analysis 10

Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Therapeutics under Development by Companies 11

Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Therapeutics under Investigation by Universities/Institutes 12

Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline Products Glance 13

Clinical Stage Products 13

Early Stage Products 14

Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Products under Development by Companies 15

Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Products under Investigation by Universities/Institutes 16

Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Companies Involved in Therapeutics Development 17

AngioChem Inc 17

ArmaGen Inc 18

Bioasis Technologies Inc 19

CRISPR Therapeutics 20

Eloxx Pharmaceuticals Ltd 21

Inventiva 22

Novartis AG 23

OPKO Health Inc 24

PTC Therapeutics Inc 25

RegenxBio Inc 26

Sangamo BioSciences Inc 27

Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Therapeutics Assessment 28

Assessment by Monotherapy Products 28

Assessment by Target 29

Assessment by Mechanism of Action 31

Assessment by Route of Administration 33

Assessment by Molecule Type 35

Drug Profiles 37

AGT-181 - Drug Profile 37

Product Description 37

Mechanism Of Action 37

R&D Progress 37

ataluren - Drug Profile 39

Product Description 39

Mechanism Of Action 39

R&D Progress 39

Cell Therapy for Mucopolysaccharidosis I - Drug Profile 48

Product Description 48

Mechanism Of Action 48

R&D Progress 48

Cell Therapy to Activate Alpha L-Iduronidase for Hurler Syndrome - Drug Profile 49

Product Description 49

Mechanism Of Action 49

R&D Progress 49

ELX-02 - Drug Profile 50

Product Description 50

Mechanism Of Action 50

R&D Progress 50

HSC-835 - Drug Profile 51

Product Description 51

Mechanism Of Action 51

R&D Progress 51

JOT-102 - Drug Profile 52

Product Description 52

Mechanism Of Action 52

R&D Progress 52

odiparcil - Drug Profile 53

Product Description 53

Mechanism Of Action 53

R&D Progress 53

Oligonucleotide for Mucopolysaccharidosis I - Drug Profile 54

Product Description 54

Mechanism Of Action 54

R&D Progress 54

pentosan polysulfate sodium - Drug Profile 55

Product Description 55

Mechanism Of Action 55

R&D Progress 55

Recombinant Alpha-L-Iduronidase Replacement for Hurler Syndrome - Drug Profile 56

Product Description 56

Mechanism Of Action 56

R&D Progress 56

Recombinant Alpha-L-Iduronidase Replacement for Mucopolysaccharidosis I - Drug Profile 57

Product Description 57

Mechanism Of Action 57

R&D Progress 57

Recombinant Alpha-L-Iduronidase Replacement for Mucopolysaccharidosis I - Drug Profile 58

Product Description 58

Mechanism Of Action 58

R&D Progress 58

RGX-111 - Drug Profile 59

Product Description 59

Mechanism Of Action 59

R&D Progress 59

SB-318 - Drug Profile 61

Product Description 61

Mechanism Of Action 61

R&D Progress 61

Stem Cell Therapy for Hurler Sydrome - Drug Profile 63

Product Description 63

Mechanism Of Action 63

R&D Progress 63

Stem Cell Therapy for Type1 Mucopolysaccharidosis - Drug Profile 64

Product Description 64

Mechanism Of Action 64

R&D Progress 64

X-372 - Drug Profile 65

Product Description 65

Mechanism Of Action 65

R&D Progress 65

Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Dormant Projects 66

Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Discontinued Products 67

Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Product Development Milestones 68

Featured News & Press Releases 68

Nov 07, 2016: Eloxx Pharmaceuticals Announces Orphan Drug Designation in the U.S. and Europe for ELX-02 in Mucopolysacchardisis Type 1 (MPS 1) 68

Sep 13, 2016: REGENXBIO Publishes Data from Ongoing Preclinical Studies of NAV Gene Therapy RGX-111 68

Jul 05, 2016: REGENXBIO Provides Update On Gene Therapy Development Program RGX-111 69

May 09, 2016: Sangamo BioSciences Presents Recent Developments From Research And ZFP Therapeutic Programs In Multiple Presentations At Annual Meeting of the American Society of Gene and Cell Therapy 69

Mar 31, 2016: ArmaGen Announces Initiation of Phase 2 Proof-of-Concept Clinical Trial in Brazil to Study AGT-181 for the Treatment of Hurler Syndrome 71

Feb 08, 2016: Sangamo BioSciences Announces FDA Clearance Of Investigational New Drug Application For ZFN-Mediated Genome Editing Treatment Of MPS I 72

Dec 30, 2015: FDA Grants Rare Pediatric Disease Designation to REGENXBIO RGX-111 Gene Therapy for the Treatment of Mucopolysaccharidosis Type I (MPS I) 73

Nov 05, 2015: ArmaGen Receives Rare Pediatric Disease Designation from FDA for AGT-181 for the Potential Treatment of Hurler Syndrome 73

Oct 01, 2015: FDA Grants Orphan Drug Designation to REGENXBIO's RGX-111 Gene Therapy for the Treatment of Mucopolysaccharidosis Type I 73

Sep 02, 2015: ArmaGen Announces First Patient Dosed in Phase 1/2a Clinical Trial of AGT-181 for the Treatment of Hurler Syndrome 74

Apr 08, 2015: ArmaGen Announces FDA Acceptance of IND Application for AGT-181 for the Treatment of Hurler Syndrome 75

Dec 19, 2014: Translarna Granted Orphan Drug Designation in the U.S. and Europe for the Treatment of Mucopolysaccharidosis I 75

Feb 05, 2014: Mouse Study Shows Gene Therapy May Be Possible Cure for Hurler Syndrome 76

Appendix 78

Methodology 78

Coverage 78

Secondary Research 78

Primary Research 78

Expert Panel Validation 78

Contact Us 78

Disclaimer 79





List of Tables

Number of Products under Development for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ), H2 2016 9

Number of Products under Development for Mucopolysaccharidosis I (MPS I) (Hurler 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 I (MPS I) (Hurler Syndrome ) - Pipeline by AngioChem Inc, H2 2016 17

Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by ArmaGen Inc, H2 2016 18

Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by Bioasis Technologies Inc, H2 2016 19

Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by CRISPR Therapeutics, H2 2016 20

Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by Eloxx Pharmaceuticals Ltd, H2 2016 21

Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by Inventiva, H2 2016 22

Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by Novartis AG, H2 2016 23

Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by OPKO Health Inc, H2 2016 24

Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by PTC Therapeutics Inc, H2 2016 25

Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by RegenxBio Inc, H2 2016 26

Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by Sangamo BioSciences Inc, H2 2016 27

Assessment by Monotherapy Products, H2 2016 28

Number of Products by Stage and Target, H2 2016 30

Number of Products by Stage and Mechanism of Action, H2 2016 32

Number of Products by Stage and Route of Administration, H2 2016 34

Number of Products by Stage and Molecule Type, H2 2016 36

Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Dormant Projects, H2 2016 66

Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Discontinued Products, H2 2016 67





List of Figures

Number of Products under Development for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ), H2 2016 9

Number of Products under Development for Mucopolysaccharidosis I (MPS I) (Hurler 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 Products, H2 2016 14

Assessment by Monotherapy Products, H2 2016 28

Number of Products by Targets, H2 2016 29

Number of Products by Stage and Targets, H2 2016 29

Number of Products by Mechanism of Actions, H2 2016 31

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

Number of Products by Routes of Administration, H2 2016 33

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

Number of Products by Molecule Types, H2 2016 35

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

* 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