Lysosomal Storage Disorder - Pipeline Review, H2 2015

Date: September 23, 2015
Pages: 78
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Publisher: Global Markets Direct
Report type: Strategic Report
Delivery: E-mail Delivery (PDF)
ID: LF202583D0CEN
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Lysosomal Storage Disorder - Pipeline Review, H2 2015

SUMMARY

Global Markets Direct’s, ‘Lysosomal Storage Disorder - Pipeline Review, H2 2015’, provides an overview of the Lysosomal Storage Disorder’s therapeutic pipeline.

This report provides comprehensive information on the therapeutic development for Lysosomal Storage Disorder, complete with comparative analysis at various stages, therapeutics assessment by drug target, mechanism of action (MoA), route of administration (RoA) and molecule type, along with latest updates, and featured news and press releases. It also reviews key players involved in the therapeutic development for Lysosomal Storage Disorder and special features on late-stage 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, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources, put together by Global Markets Direct’s team. Drug profiles/records featured in the report undergoes periodic updation following a stringent set of processes that ensures that all the profiles are updated with the latest set of information. Additionally, processes including live news & deals tracking, browser based alert-box and clinical trials registries tracking ensure that the most recent developments are captured on a real time basis.

The report enhances decision making capabilities and help to create effective counter strategies to gain competitive advantage. It strengthens R&D pipelines by identifying new targets and MOAs to produce first-in-class and best-in-class products.

Note: Certain sections in the report may be removed or altered based on the availability and relevance of data for the indicated disease.

SCOPE
  • The report provides a snapshot of the global therapeutic landscape of Lysosomal Storage Disorder
  • The report reviews key pipeline products under drug profile section which includes, product description, MoA and R&D brief, licensing and collaboration details & other developmental activities
  • The report reviews key players involved in the therapeutics development for Lysosomal Storage Disorder and enlists all their major and minor projects
  • The report summarizes all the dormant and discontinued pipeline projects
  • A review of the Lysosomal Storage Disorder products under development by companies and universities/research institutes based on information derived from company and industry-specific sources
  • Pipeline products coverage based on various stages of development ranging from pre-registration till discovery and undisclosed stages
  • A detailed assessment of monotherapy and combination therapy pipeline projects
  • Coverage of the Lysosomal Storage Disorder pipeline on the basis of target, MoA, route of administration and molecule type
  • Latest news and deals relating related to pipeline products
REASONS TO BUY
  • Provides strategically significant competitor information, analysis, and insights to formulate effective R&D development strategies
  • Identify emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage
  • Develop strategic initiatives by understanding the focus areas of leading companies
  • Identify and understand important and diverse types of therapeutics under development for Lysosomal Storage Disorder
  • Plan mergers and acquisitions effectively by identifying key players of the most promising pipeline
  • Devise corrective measures for pipeline projects by understanding Lysosomal Storage Disorder 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
Introduction
Global Markets Direct Report Coverage
Lysosomal Storage Disorder Overview
Therapeutics Development
Pipeline Products for Lysosomal Storage Disorder - Overview
Pipeline Products for Lysosomal Storage Disorder - Comparative Analysis
Lysosomal Storage Disorder - Therapeutics under Development by Companies
Lysosomal Storage Disorder - Therapeutics under Investigation by Universities/Institutes
Lysosomal Storage Disorder - Pipeline Products Glance
Clinical Stage Products
Early Stage Products
Unknown Stage Products
Lysosomal Storage Disorder - Products under Development by Companies
Lysosomal Storage Disorder - Products under Investigation by Universities/Institutes
Lysosomal Storage Disorder - Companies Involved in Therapeutics Development
2-BBB MEDICINES BV
Amicus Therapeutics, Inc.
AngioChem Inc.
Fate Therapeutics, Inc.
Minoryx Therapeutics s.l.
Nimbus Therapeutics, LLC
Nuo Therapeutics, Inc.
Orphazyme ApS
Oxyrane Belgium NV
Promosome, LLC
REGiMMUNE Corporation
Sangamo BioSciences, Inc.
Ultragenyx Pharmaceutical Inc.
Lysosomal Storage Disorder - Therapeutics Assessment
Assessment by Monotherapy Products
Assessment by Target
Assessment by Mechanism of Action
Assessment by Route of Administration
Assessment by Molecule Type
Drug Profiles
2B3-201 - DRUG PROFILE
Product Description
Mechanism of Action
R&D Progress
ALD-601 - Drug Profile
Product Description
Mechanism of Action
R&D Progress
Chaperone-ERT Combinations - Drug Profile
Product Description
Mechanism of Action
R&D Progress
Delta-tocopherol - Drug Profile
Product Description
Mechanism of Action
R&D Progress
FT-1050 - Drug Profile
Product Description
Mechanism of Action
R&D Progress
ML-SA1 - Drug Profile
Product Description
Mechanism of Action
R&D Progress
OR-02 - Drug Profile
Product Description
Mechanism of Action
R&D Progress
OR-03 - Drug Profile
Product Description
Mechanism of Action
R&D Progress
Recombinant Enzyme for Farber Disease and Cystic Fibrosis - Drug Profile
Product Description
Mechanism of Action
R&D Progress
Recombinant Enzyme for LSD-2 Disease - Drug Profile
Product Description
Mechanism of Action
R&D Progress
Recombinant Enzyme for LSD-3 Disease - Drug Profile
Product Description
Mechanism of Action
R&D Progress
Recombinant Enzyme for LSD-4 Disease - Drug Profile
Product Description
Mechanism of Action
R&D Progress
Recombinant Enzyme for LSD-5 Disease - Drug Profile
Product Description
Mechanism of Action
R&D Progress
Recombinant Enzyme for Lysosomal Storage Diseases and Metabolic Disorders - Drug Profile
Product Description
Mechanism of Action
R&D Progress
Recombinant Enzyme for Lysosomal Storage Disorder - Drug Profile
Product Description
Mechanism of Action
R&D Progress
Recombinant Enzyme to Target LRP1 for Lysosomal Storage Disorder - Drug Profile
Product Description
Mechanism of Action
R&D Progress
RGI-5000 - Drug Profile
Product Description
Mechanism of Action
R&D Progress
SBLSD-3 - Drug Profile
Product Description
Mechanism of Action
R&D Progress
SBLSD-4 - Drug Profile
Product Description
Mechanism of Action
R&D Progress
Small Molecule to Activate Beta-Galactosidase for GM1 Gangliosidosis and Gaucher’s disease - Drug Profile
Product Description
Mechanism of Action
R&D Progress
Small Molecules for GM1-gangliosidosis and Morquio B - Drug Profile
Product Description
Mechanism of Action
R&D Progress
Small Molecules for Lysosomal Storage Disorders - Drug Profile
Product Description
Mechanism of Action
R&D Progress
Stem Cell Therapy for Lysosomal Storage Disorder - Drug Profile
Product Description
Mechanism of Action
R&D Progress
UX-004 - Drug Profile
Product Description
Mechanism of Action
R&D Progress
Lysosomal Storage Disorder - Recent Pipeline Updates
Lysosomal Storage Disorder - Dormant Projects
Lysosomal Storage Disorder - Product Development Milestones
Featured News & Press Releases
Jul 29, 2014: Fate Therapeutics Announces FDA Clearance of IND for Clinical Development of PROHEMA in Inherited Metabolic Disorders
Feb 26, 2014: Fate Therapeutics Announces Observed Effects of Pharmacologic Modulation on T Cell Compartment From Its Phase 1b Study of PROHEMA
Oct 21, 2013: For first time, drug developed based on zebrafish studies passes Phase I clinical trial
Aug 15, 2012: Orphazyme Receives European Patent Covering Use Of HSP70 For Treatment Of Lysosomal Storage Diseases
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer

LIST OF TABLES

Number of Products under Development for Lysosomal Storage Disorder, H2 2015
Number of Products under Development for Lysosomal Storage Disorder - Comparative Analysis, H2 2015
Number of Products under Development by Companies, H2 2015
Number of Products under Investigation by Universities/Institutes, H2 2015
Comparative Analysis by Clinical Stage Development, H2 2015
Comparative Analysis by Early Stage Development, H2 2015
Comparative Analysis by Unknown Stage Development, H2 2015
Products under Development by Companies, H2 2015
Products under Development by Companies, H2 2015 (Contd..1)
Products under Investigation by Universities/Institutes, H2 2015
Lysosomal Storage Disorder - Pipeline by 2-BBB Medicines BV, H2 2015
Lysosomal Storage Disorder - Pipeline by Amicus Therapeutics, Inc., H2 2015
Lysosomal Storage Disorder - Pipeline by AngioChem Inc., H2 2015
Lysosomal Storage Disorder - Pipeline by Fate Therapeutics, Inc., H2 2015
Lysosomal Storage Disorder - Pipeline by Minoryx Therapeutics s.l., H2 2015
Lysosomal Storage Disorder - Pipeline by Nimbus Therapeutics, LLC, H2 2015
Lysosomal Storage Disorder - Pipeline by Nuo Therapeutics, Inc., H2 2015
Lysosomal Storage Disorder - Pipeline by Orphazyme ApS, H2 2015
Lysosomal Storage Disorder - Pipeline by Oxyrane Belgium NV, H2 2015
Lysosomal Storage Disorder - Pipeline by Promosome, LLC, H2 2015
Lysosomal Storage Disorder - Pipeline by REGiMMUNE Corporation, H2 2015
Lysosomal Storage Disorder - Pipeline by Sangamo BioSciences, Inc., H2 2015
Lysosomal Storage Disorder - Pipeline by Ultragenyx Pharmaceutical Inc., H2 2015
Assessment by Monotherapy Products, H2 2015
Number of Products by Stage and Target, H2 2015
Number of Products by Stage and Mechanism of Action, H2 2015
Number of Products by Stage and Route of Administration, H2 2015
Number of Products by Stage and Molecule Type, H2 2015
Lysosomal Storage Disorder Therapeutics - Recent Pipeline Updates, H2 2015
Lysosomal Storage Disorder - Dormant Projects, H2 2015

LIST OF FIGURES

Number of Products under Development for Lysosomal Storage Disorder, H2 2015
Number of Products under Development for Lysosomal Storage Disorder - Comparative Analysis, H2 2015
Number of Products under Development by Companies, H2 2015
Number of Products under Investigation by Universities/Institutes, H2 2015
Comparative Analysis by Early Stage Products, H2 2015
Assessment by Monotherapy Products, H2 2015
Number of Products by Top 10 Targets, H2 2015
Number of Products by Stage and Top 10 Targets, H2 2015
Number of Products by Top 10 Mechanism of Actions, H2 2015
Number of Products by Stage and Top 10 Mechanism of Actions, H2 2015
Number of Products by Top 10 Routes of Administration, H2 2015
Number of Products by Stage and Top 10 Routes of Administration, H2 2015
Number of Products by Top 10 Molecule Types, H2 2015
Number of Products by Stage and Top 10 Molecule Types, H2 2015

COMPANIES MENTIONED

2-BBB Medicines BV
Amicus Therapeutics, Inc.
AngioChem Inc.
Fate Therapeutics, Inc.
Minoryx Therapeutics s.l.
Nimbus Therapeutics, LLC
Nuo Therapeutics, Inc.
Orphazyme ApS
Oxyrane Belgium NV
Promosome, LLC
REGiMMUNE Corporation
Sangamo BioSciences, Inc.
Ultragenyx Pharmaceutical Inc.
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