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Spinal Muscular Atrophy (SMA) - Pipeline Review, H1 2015

Spinal Muscular Atrophy (SMA) - Pipeline Review, H1 2015

Summary

Global Markets Direct’s, ‘Spinal Muscular Atrophy (SMA) - Pipeline Review, H1 2015’, provides an overview of the Spinal Muscular Atrophy (SMA)’s therapeutic pipeline.

This report provides comprehensive information on the therapeutic development for Spinal Muscular Atrophy (SMA), 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 Spinal Muscular Atrophy (SMA) 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 Spinal Muscular Atrophy (SMA)
  • 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 Spinal Muscular Atrophy (SMA) and enlists all their major and minor projects
  • The report summarizes all the dormant and discontinued pipeline projects
  • A review of the Spinal Muscular Atrophy (SMA) 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 Spinal Muscular Atrophy (SMA) 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 Spinal Muscular Atrophy (SMA)
  • Plan mergers and acquisitions effectively by identifying key players of the most promising pipeline
  • Devise corrective measures for pipeline projects by understanding Spinal Muscular Atrophy (SMA) 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
Spinal Muscular Atrophy (SMA) Overview
Therapeutics Development
Pipeline Products for Spinal Muscular Atrophy (SMA) - Overview
Pipeline Products for Spinal Muscular Atrophy (SMA) - Comparative Analysis
Spinal Muscular Atrophy (SMA) - Therapeutics under Development by Companies
Spinal Muscular Atrophy (SMA) - Therapeutics under Investigation by Universities/Institutes
Spinal Muscular Atrophy (SMA) - Pipeline Products Glance
Late Stage Products
Clinical Stage Products
Early Stage Products
Unknown Stage Products
Spinal Muscular Atrophy (SMA) - Products under Development by Companies
Spinal Muscular Atrophy (SMA) - Products under Investigation by Universities/Institutes
Spinal Muscular Atrophy (SMA) - Companies Involved in Therapeutics Development
Bioblast Pharma Ltd.
Cellular Biomedicine Group, Inc.
Cytokinetics, Inc.
F. Hoffmann-La Roche Ltd.
Genethon
Genzyme Corporation
GMP-Orphan SAS
Isis Pharmaceuticals, Inc.
Longevity Biotech, Inc
Neurodyn Inc.
Neurotune AG
Nexgenix Pharmaceuticals, LLC
Novartis AG
OrphageniX, Inc.
Paratek Pharmaceuticals, Inc.
Pfizer Inc.
PTC Therapeutics, Inc.
Retrophin Inc.
Sarepta Therapeutics, Inc.
Trophos SA
Vybion, Inc.
Zambon Company S.p.A.
Spinal Muscular Atrophy (SMA) - 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
ALB-111 - Drug Profile
Product Description
Mechanism of Action
R&D Progress
Antisense Oligonucleotide for Spinal Muscular Atrophy and Myotonic Dystrophy - Drug Profile
Product Description
Mechanism of Action
R&D Progress
Antisense Oligonucleotide to Inhibit SMN2 gene for SMA - Drug Profile
Product Description
Mechanism of Action
R&D Progress
Antisense Oligonucleotides for Spinal Muscular Atrophy - Drug Profile
Product Description
Mechanism of Action
R&D Progress
ataluren - Drug Profile
Product Description
Mechanism of Action
R&D Progress
BBrm-01 - Drug Profile
Product Description
Mechanism of Action
R&D Progress
DNA Oligonucleotides for Spinal Muscular Atrophy - Drug Profile
Product Description
Mechanism of Action
R&D Progress
Gene Therapy to Activate SMN1 for Spinal Muscular Atrophy - Drug Profile
Product Description
Mechanism of Action
R&D Progress
Gene Therapy to Activate SMN1 for Spinal Muscular Atrophy - Drug Profile
Product Description
Mechanism of Action
R&D Progress
INT-41 - Drug Profile
Product Description
Mechanism of Action
R&D Progress
LMI-070 - Drug Profile
Product Description
Mechanism of Action
R&D Progress
ND-602 - Drug Profile
Product Description
Mechanism of Action
R&D Progress
NT-1654 - Drug Profile
Product Description
Mechanism of Action
R&D Progress
nusinersen - Drug Profile
Product Description
Mechanism of Action
R&D Progress
NXD-30001 - Drug Profile
Product Description
Mechanism of Action
R&D Progress
olesoxime - Drug Profile
Product Description
Mechanism of Action
R&D Progress
Oligonucleotide to Activate SMN2 for Spinal Muscular Atrophy - Drug Profile
Product Description
Mechanism of Action
R&D Progress
PF-06687859 - Drug Profile
Product Description
Mechanism of Action
R&D Progress
PMO-25 - Drug Profile
Product Description
Mechanism of Action
R&D Progress
PTKSMA-2 - Drug Profile
Product Description
Mechanism of Action
R&D Progress
RE-003 - Drug Profile
Product Description
Mechanism of Action
R&D Progress
RG-7800 - Drug Profile
Product Description
Mechanism of Action
R&D Progress
scAAV9.CB.SMN - Drug Profile
Product Description
Mechanism of Action
R&D Progress
Small Molecules for CNS Disorders - Drug Profile
Product Description
Mechanism of Action
R&D Progress
sodium phenylbutyrate - Drug Profile
Product Description
Mechanism of Action
R&D Progress
Stem Cell Therapy for Amyotrophic Lateral Sclerosis and Spinal Muscular Atrophy - Drug Profile
Product Description
Mechanism of Action
R&D Progress
Synthetic Peptides for Central Nervous System Disorders and Diabetic Neuropathy - Drug Profile
Product Description
Mechanism of Action
R&D Progress
tirasemtiv - Drug Profile
Product Description
Mechanism of Action
R&D Progress
ZP-037 - Drug Profile
Product Description
Mechanism of Action
R&D Progress
Spinal Muscular Atrophy (SMA) - Recent Pipeline Updates
Spinal Muscular Atrophy (SMA) - Dormant Projects
Spinal Muscular Atrophy (SMA) - Product Development Milestones
Featured News & Press Releases
Nov 25, 2014: Isis Pharmaceuticals Initiates Phase 3 Study of ISIS-SMN Rx in Children With Spinal Muscular Atrophy
Nov 17, 2014: Cure SMA Presents Isis Pharmaceuticals with SMA Breakthrough Award
Oct 10, 2014: Isis Pharmaceuticals Reports Data From ISIS-SMN Rx Phase 2 Studies in Infants and Children With Spinal Muscular Atrophy
Oct 02, 2014: Isis Pharmaceuticals to Host Webcast to Discuss ISIS-SMN Rx Results Presented at the World Muscle Society Congress
Oct 02, 2014: AveXis Receives Orphan Drug Status from the US Food and Drug Administration for chariSMA for the Treatment of Spinal Muscular Atrophy
Aug 15, 2014: Isis Pharmaceuticals Earns $2M from the Advancement of ISIS-SMN Rx in Children with Spinal Muscular Atrophy
Aug 01, 2014: Isis Pharmaceuticals Initiates Phase 3 Study of ISIS-SMN Rx in Infants with Spinal Muscular Atrophy
Jun 13, 2014: Cytokinetics Announces Data Relating To Tirasemtiv Presented At The 2014 Annual Spinal Muscular Atrophy Conference
Jun 06, 2014: Cytokinetics Announces Two Presentations Relating To Tirasemtiv To Be Presented At The 2014 Annual Spinal Muscular Atrophy Conference
May 22, 2014: Gene therapy extends survival in an animal model of spinal muscular atrophy
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer
List of Tables
Number of Products under Development for Spinal Muscular Atrophy (SMA), H1 2015
Number of Products under Development for Spinal Muscular Atrophy (SMA) - Comparative Analysis, H1 2015
Number of Products under Development by Companies, H1 2015
Number of Products under Development by Companies, H1 2015 (Contd..1)
Number of Products under Investigation by Universities/Institutes, H1 2015
Comparative Analysis by Late Stage Development, H1 2015
Comparative Analysis by Clinical Stage Development, H1 2015
Comparative Analysis by Early Stage Development, H1 2015
Comparative Analysis by Unknown Stage Development, H1 2015
Products under Development by Companies, H1 2015
Products under Development by Companies, H1 2015 (Contd..1)
Products under Investigation by Universities/Institutes, H1 2015
Spinal Muscular Atrophy (SMA) - Pipeline by Bioblast Pharma Ltd., H1 2015
Spinal Muscular Atrophy (SMA) - Pipeline by Cellular Biomedicine Group, Inc., H1 2015
Spinal Muscular Atrophy (SMA) - Pipeline by Cytokinetics, Inc., H1 2015
Spinal Muscular Atrophy (SMA) - Pipeline by F. Hoffmann-La Roche Ltd., H1 2015
Spinal Muscular Atrophy (SMA) - Pipeline by Genethon, H1 2015
Spinal Muscular Atrophy (SMA) - Pipeline by Genzyme Corporation, H1 2015
Spinal Muscular Atrophy (SMA) - Pipeline by GMP-Orphan SAS, H1 2015
Spinal Muscular Atrophy (SMA) - Pipeline by Isis Pharmaceuticals, Inc., H1 2015
Spinal Muscular Atrophy (SMA) - Pipeline by Longevity Biotech, Inc, H1 2015
Spinal Muscular Atrophy (SMA) - Pipeline by Neurodyn Inc., H1 2015
Spinal Muscular Atrophy (SMA) - Pipeline by Neurotune AG, H1 2015
Spinal Muscular Atrophy (SMA) - Pipeline by Nexgenix Pharmaceuticals, LLC, H1 2015
Spinal Muscular Atrophy (SMA) - Pipeline by Novartis AG, H1 2015
Spinal Muscular Atrophy (SMA) - Pipeline by OrphageniX, Inc., H1 2015
Spinal Muscular Atrophy (SMA) - Pipeline by Paratek Pharmaceuticals, Inc., H1 2015
Spinal Muscular Atrophy (SMA) - Pipeline by Pfizer Inc., H1 2015
Spinal Muscular Atrophy (SMA) - Pipeline by PTC Therapeutics, Inc., H1 2015
Spinal Muscular Atrophy (SMA) - Pipeline by Retrophin Inc., H1 2015
Spinal Muscular Atrophy (SMA) - Pipeline by Sarepta Therapeutics, Inc., H1 2015
Spinal Muscular Atrophy (SMA) - Pipeline by Trophos SA, H1 2015
Spinal Muscular Atrophy (SMA) - Pipeline by Vybion, Inc., H1 2015
Spinal Muscular Atrophy (SMA) - Pipeline by Zambon Company S.p.A., H1 2015
Assessment by Monotherapy Products, H1 2015
Number of Products by Stage and Target, H1 2015
Number of Products by Stage and Mechanism of Action, H1 2015
Number of Products by Stage and Route of Administration, H1 2015
Number of Products by Stage and Molecule Type, H1 2015
Spinal Muscular Atrophy (SMA) Therapeutics - Recent Pipeline Updates, H1 2015
Spinal Muscular Atrophy (SMA) - Dormant Projects, H1 2015
List of Figures
Number of Products under Development for Spinal Muscular Atrophy (SMA), H1 2015
Number of Products under Development for Spinal Muscular Atrophy (SMA) - Comparative Analysis, H1 2015
Number of Products under Development by Companies, H1 2015
Comparative Analysis by Clinical Stage Development, H1 2015
Comparative Analysis by Early Stage Products, H1 2015
Assessment by Monotherapy Products, H1 2015
Number of Products by Top 10 Targets, H1 2015
Number of Products by Stage and Top 10 Targets, H1 2015
Number of Products by Top 10 Mechanism of Actions, H1 2015
Number of Products by Stage and Top 10 Mechanism of Actions, H1 2015
Number of Products by Top 10 Routes of Administration, H1 2015
Number of Products by Stage and Top 10 Routes of Administration, H1 2015
Number of Products by Top 10 Molecule Types, H1 2015
Number of Products by Stage and Top 10 Molecule Types, H1 2015

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