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Spinal Muscular Atrophy (SMA) - Pipeline Review, H1 2016

Spinal Muscular Atrophy (SMA) - Pipeline Review, H1 2016

Summary

Global Markets Direct’s, ‘Spinal Muscular Atrophy (SMA) - Pipeline Review, H1 2016’, provides an overview of the Spinal Muscular Atrophy (SMA) pipeline landscape.

The report provides comprehensive information on the therapeutics under development for Spinal Muscular Atrophy (SMA), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The report also covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases. Additionally, the report provides an overview of key players involved in therapeutic development for Spinal Muscular Atrophy (SMA) and features dormant 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, clinical trial registries, conferences, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources. Drug profiles featured in the report undergoes periodic review following a stringent set of processes to ensure that all the profiles are updated with the latest set of information. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.

The report 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.

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

Scope

  • The report provides a snapshot of the global therapeutic landscape of Spinal Muscular Atrophy (SMA)
  • The report reviews pipeline therapeutics for Spinal Muscular Atrophy (SMA) by companies and universities/research institutes based on information derived from company and industry-specific sources
  • The report covers pipeline products based on various stages of development ranging from pre-registration till discovery and undisclosed stages
  • The report features descriptive drug profiles for the pipeline products which includes, product description, descriptive MoA, R&D brief, licensing and collaboration details & other developmental activities
  • The report reviews key players involved Spinal Muscular Atrophy (SMA) therapeutics and enlists all their major and minor projects
  • The report assesses Spinal Muscular Atrophy (SMA) therapeutics based on drug target, mechanism of action (MoA), route of administration (RoA) and molecule type
  • The report summarizes all the dormant and discontinued pipeline projects
  • The report reviews latest news related to pipeline therapeutics for Spinal Muscular Atrophy (SMA)
Reasons to buy
  • Gain strategically significant competitor information, analysis, and insights to formulate effective R&D strategies
  • Identify emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage
  • Identify and understand important and diverse types of therapeutics under development for Spinal Muscular Atrophy (SMA)
  • Identify potential new clients or partners in the target demographic
  • Develop strategic initiatives by understanding the focus areas of leading companies
  • Plan mergers and acquisitions effectively by identifying key players and it’s most promising pipeline therapeutics
  • 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
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
Astellas Pharma Inc.
AveXis, Inc.
Bioblast Pharma Ltd.
Cytokinetics, Inc.
F. Hoffmann-La Roche Ltd.
Genethon
Genzyme Corporation
GMP-Orphan SAS
Ionis Pharmaceuticals, Inc.
Longevity Biotech, Inc
Neurodyn Inc.
Neurotune AG
Novartis AG
Sarepta Therapeutics, Inc.
Voyager Therapeutics, Inc.
Vybion, Inc.
WAVE Life Sciences Ltd.
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 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
ARM-210 - Drug Profile
Product Description
Mechanism of Action
R&D Progress
AVXS-101 - Drug Profile
Product Description
Mechanism of Action
R&D Progress
azithromycin - Drug Profile
Product Description
Mechanism of Action
R&D Progress
CK-2127107 - Drug Profile
Product Description
Mechanism of Action
R&D Progress
Gene Therapy to Activate SMN 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
LBT-3627 - 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
Oligonucleotides to Activate SMN2 for Spinal Muscular Atrophy - Drug Profile
Product Description
Mechanism of Action
R&D Progress
PMO-25 - Drug Profile
Product Description
Mechanism of Action
R&D Progress
RG-7800 - Drug Profile
Product Description
Mechanism of Action
R&D Progress
RG-7916 - Drug Profile
Product Description
Mechanism of Action
R&D Progress
Small Molecules for Central Nervous System Disorders - Drug Profile
Product Description
Mechanism of Action
R&D Progress
Small Molecules to Activate SMN2 for Spinal Muscular Atrophy - Drug Profile
Product Description
Mechanism of Action
R&D Progress
Small Molecules to Activate SMN2 for Spinal Muscular Atrophy - Drug Profile
Product Description
Mechanism of Action
R&D Progress
Small Molecules to Activate Troponin for Non-Neuromuscular and Neuromuscular Disorders - Drug Profile
Product Description
Mechanism of Action
R&D Progress
sodium phenylbutyrate - Drug Profile
Product Description
Mechanism of Action
R&D Progress
tirasemtiv - Drug Profile
Product Description
Mechanism of Action
R&D Progress
VYSMN-101 - 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) - Discontinued Products
Spinal Muscular Atrophy (SMA) - Product Development Milestones
Featured News & Press Releases
Jan 12, 2016: Ionis Pharmaceuticals Completes Target Enrollment for Nusinersen Phase 3 Study, CHERISH, in Children with Spinal Muscular Atrophy
Jan 07, 2016: Spinal Muscular Atrophy Program Advances with Additional Product Candidate Entering Clinical Development
Jan 04, 2016: Cytokinetics Announces Start Of Phase 2 Clinical Trial Of Ck-2127107 In Patients With Spinal Muscular Atrophy
Oct 15, 2015: Isis Pharmaceuticals Initiates SHINE Study to Provide ISIS-SMN for Patients With SMA Who Have Completed the Phase 3 ENDEAR and CHERISH Studies
Oct 04, 2015: Phase 2 Data from Spinal Muscular Atrophy Program Presented at the 20th International Annual Congress of the World Muscle Society
Jul 14, 2015: Isis Pharmaceuticals Earns $2.15M for Advancing ISIS-SMN in Children With Spinal Muscular Atrophy
Jun 22, 2015: BioBlast Announces Positive Preclinical Proof-Of-Concept Results Of Its Novel Read- through Platform in Spinal Muscular Atrophy
Jun 22, 2015: Isis Pharmaceuticals Reports Data from ISIS-SMN Rx in Children with Spinal Muscular Atrophy
Jun 19, 2015: Cytokinetics Presents Results From Phase I Studies Of Ck-2127107
Jun 18, 2015: BioBlast Pharma to Present Preclinical Proofs of Concept in Spinal Muscular Atrophy at Two Leading Scientific Meetings in June
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 2016
Number of Products under Development for Spinal Muscular Atrophy (SMA) - Comparative Analysis, H1 2016
Number of Products under Development by Companies, H1 2016
Number of Products under Investigation by Universities/Institutes, H1 2016
Comparative Analysis by Late Stage Development, H1 2016
Comparative Analysis by Clinical Stage Development, H1 2016
Comparative Analysis by Early Stage Development, H1 2016
Products under Development by Companies, H1 2016
Products under Investigation by Universities/Institutes, H1 2016
Spinal Muscular Atrophy (SMA) - Pipeline by Astellas Pharma Inc., H1 2016
Spinal Muscular Atrophy (SMA) - Pipeline by AveXis, Inc., H1 2016
Spinal Muscular Atrophy (SMA) - Pipeline by Bioblast Pharma Ltd., H1 2016
Spinal Muscular Atrophy (SMA) - Pipeline by Cytokinetics, Inc., H1 2016
Spinal Muscular Atrophy (SMA) - Pipeline by F. Hoffmann-La Roche Ltd., H1 2016
Spinal Muscular Atrophy (SMA) - Pipeline by Genethon, H1 2016
Spinal Muscular Atrophy (SMA) - Pipeline by Genzyme Corporation, H1 2016
Spinal Muscular Atrophy (SMA) - Pipeline by GMP-Orphan SAS, H1 2016
Spinal Muscular Atrophy (SMA) - Pipeline by Ionis Pharmaceuticals, Inc., H1 2016
Spinal Muscular Atrophy (SMA) - Pipeline by Longevity Biotech, Inc, H1 2016
Spinal Muscular Atrophy (SMA) - Pipeline by Neurodyn Inc., H1 2016
Spinal Muscular Atrophy (SMA) - Pipeline by Neurotune AG, H1 2016
Spinal Muscular Atrophy (SMA) - Pipeline by Novartis AG, H1 2016
Spinal Muscular Atrophy (SMA) - Pipeline by Sarepta Therapeutics, Inc., H1 2016
Spinal Muscular Atrophy (SMA) - Pipeline by Voyager Therapeutics, Inc., H1 2016
Spinal Muscular Atrophy (SMA) - Pipeline by Vybion, Inc., H1 2016
Spinal Muscular Atrophy (SMA) - Pipeline by WAVE Life Sciences Ltd., H1 2016
Assessment by Monotherapy Products, H1 2016
Number of Products by Stage and Target, H1 2016
Number of Products by Stage and Mechanism of Action, H1 2016
Number of Products by Stage and Route of Administration, H1 2016
Number of Products by Stage and Molecule Type, H1 2016
Spinal Muscular Atrophy (SMA) Therapeutics - Recent Pipeline Updates, H1 2016
Spinal Muscular Atrophy (SMA) - Dormant Projects, H1 2016
Spinal Muscular Atrophy (SMA) - Discontinued Products, H1 2016
List of Figures
Number of Products under Development for Spinal Muscular Atrophy (SMA), H1 2016
Number of Products under Development for Spinal Muscular Atrophy (SMA) - Comparative Analysis, H1 2016
Number of Products under Development by Companies, H1 2016
Comparative Analysis by Clinical Stage Development, H1 2016
Comparative Analysis by Early Stage Products, H1 2016
Assessment by Monotherapy Products, H1 2016
Number of Products by Targets, H1 2016
Number of Products by Stage and Targets, H1 2016
Number of Products by Mechanism of Actions, H1 2016
Number of Products by Stage and Mechanism of Actions, H1 2016
Number of Products by Routes of Administration, H1 2016
Number of Products by Stage and Routes of Administration, H1 2016
Number of Products by Molecule Types, H1 2016
Number of Products by Stage and Molecule Types, H1 2016

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