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Spinal Muscular Atrophy (SMA) - Pipeline Review, H1 2017

  • March 2017
  • -
  • Global Markets Direct
  • -
  • 111 pages

Spinal Muscular Atrophy (SMA) - Pipeline Review, H1 2017

Summary

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Spinal Muscular Atrophy (SMA) - Pipeline Review, H1 2017, provides an overview of the Spinal Muscular Atrophy (SMA) (Central Nervous System) pipeline landscape.

Spinal muscular atrophy (SMA) is a genetic disease that attacks nerve cells, called motor neurons, in the spinal cord. These critically important cells are responsible for supplying electrical and chemical messages to muscle cells. Without the proper input from the motor neurons, muscle cells cannot function properly. The muscle cells will, therefore, become much smaller (atrophy) and will produce symptoms of muscle weakness. This can affect walking, crawling, breathing, swallowing, and head and neck control.

Report Highlights

Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Spinal Muscular Atrophy (SMA) - Pipeline Review, H1 2017, provides comprehensive information on the therapeutics under development for Spinal Muscular Atrophy (SMA) (Central Nervous System), 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 Spinal Muscular Atrophy (SMA) (Central Nervous System) pipeline guide also reviews of key players involved in therapeutic development for Spinal Muscular Atrophy (SMA) and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Pre-Registration, Phase III, Phase II, Phase I, Preclinical and Discovery stages are 1, 2, 4, 2, 9 and 2 respectively. Similarly, the Universities portfolio in Preclinical and Discovery stages comprises 6 and 2 molecules, respectively.

Spinal Muscular Atrophy (SMA) (Central Nervous System) 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 Spinal Muscular Atrophy (SMA) (Central Nervous System).
- The pipeline guide reviews pipeline therapeutics for Spinal Muscular Atrophy (SMA) (Central Nervous System) 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 Spinal Muscular Atrophy (SMA) (Central Nervous System) therapeutics and enlists all their major and minor projects.
- The pipeline guide evaluates Spinal Muscular Atrophy (SMA) (Central Nervous System) 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 Spinal Muscular Atrophy (SMA) (Central Nervous System)

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 Spinal Muscular Atrophy (SMA) (Central Nervous System).
- 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 Spinal Muscular Atrophy (SMA) (Central Nervous System) 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

Spinal Muscular Atrophy (SMA) - Pipeline Review, H1 2017
Introduction
Global Markets Direct Report Coverage
Spinal Muscular Atrophy (SMA) - Overview
Spinal Muscular Atrophy (SMA) - Therapeutics Development
Pipeline Overview
Pipeline by Companies
Pipeline by Universities/Institutes
Products under Development by Companies
Products under Development by Universities/Institutes
Spinal Muscular Atrophy (SMA) - Therapeutics Assessment
Assessment by Target
Assessment by Mechanism of Action
Assessment by Route of Administration
Assessment by Molecule Type
Spinal Muscular Atrophy (SMA) - Companies Involved in Therapeutics Development
AveXis Inc
Cytokinetics Inc
F. Hoffmann-La Roche Ltd
Genethon SA
Genzyme Corp
Ionis Pharmaceuticals Inc
Kowa Company Ltd
Neurotune AG
Novartis AG
Paratek Pharmaceuticals Inc
RaNA Therapeutics Inc
Sarepta Therapeutics Inc
Spotlight Innovation Inc
WAVE Life Sciences Ltd
Spinal Muscular Atrophy (SMA) - Drug Profiles
AAD-2004 - Drug Profile
Product Description
Mechanism Of Action
RandD Progress
ALB-111 - Drug Profile
Product Description
Mechanism Of Action
RandD Progress
Antisense Oligonucleotide to Activate SMN2 for Spinal Muscular Atrophy - Drug Profile
Product Description
Mechanism Of Action
RandD Progress
Antisense Oligonucleotides for Spinal Muscular Atrophy - Drug Profile
Product Description
Mechanism Of Action
RandD Progress
AVXS-101 - Drug Profile
Product Description
Mechanism Of Action
RandD Progress
CK-2127107 - Drug Profile
Product Description
Mechanism Of Action
RandD Progress
Gene Therapy to Activate SMN for Spinal Muscular Atrophy - Drug Profile
Product Description
Mechanism Of Action
RandD Progress
Gene Therapy to Activate SMN1 for Spinal Muscular Atrophy - Drug Profile
Product Description
Mechanism Of Action
RandD Progress
Gene Therapy to Activate SMN1 for Spinal Muscular Atrophy - Drug Profile
Product Description
Mechanism Of Action
RandD Progress
K-828SP - Drug Profile
Product Description
Mechanism Of Action
RandD Progress
LMI-070 - Drug Profile
Product Description
Mechanism Of Action
RandD Progress
NT-1654 - Drug Profile
Product Description
Mechanism Of Action
RandD Progress
nusinersen - Drug Profile
Product Description
Mechanism Of Action
RandD Progress
olesoxime - Drug Profile
Product Description
Mechanism Of Action
RandD Progress
Oligonucleotide to Activate SMN2 for Spinal Muscular Atrophy - Drug Profile
Product Description
Mechanism Of Action
RandD Progress
Oligonucleotides to Activate SMN2 for Spinal Muscular Atrophy Type 1 - Drug Profile
Product Description
Mechanism Of Action
RandD Progress
PMO-25 - Drug Profile
Product Description
Mechanism Of Action
RandD Progress
PTKSMA-1 - Drug Profile
Product Description
Mechanism Of Action
RandD Progress
RG-7800 - Drug Profile
Product Description
Mechanism Of Action
RandD Progress
RG-7916 - Drug Profile
Product Description
Mechanism Of Action
RandD Progress
Small Molecules for Central Nervous System Disorders - Drug Profile
Product Description
Mechanism Of Action
RandD Progress
Small Molecules for Spinal Muscular Atrophy - Drug Profile
Product Description
Mechanism Of Action
RandD Progress
Small Molecules for Spinal Muscular Atrophy - Drug Profile
Product Description
Mechanism Of Action
RandD Progress
Small Molecules to Activate SMN2 for Spinal Muscular Atrophy - Drug Profile
Product Description
Mechanism Of Action
RandD Progress
Small Molecules to Activate SMN2 for Spinal Muscular Atrophy - Drug Profile
Product Description
Mechanism Of Action
RandD Progress
Small Molecules to Inhibit GSK-3 for Spinal Muscular Atrophy - Drug Profile
Product Description
Mechanism Of Action
RandD Progress
STL-182 - Drug Profile
Product Description
Mechanism Of Action
RandD Progress
tirasemtiv - Drug Profile
Product Description
Mechanism Of Action
RandD Progress
Spinal Muscular Atrophy (SMA) - Dormant Projects
Spinal Muscular Atrophy (SMA) - Discontinued Products
Spinal Muscular Atrophy (SMA) - Product Development Milestones
Featured News and Press Releases
Feb 08, 2017: Spotlight Innovation Engages Top-Tier Contract Research Organization to Conduct Preclinical Studies of STL-182
Jan 13, 2017: New Data Show SPINRAZA (nusinersen) Significantly Reduces Risk of Death or Permanent Ventilation in Infantile-Onset Spinal Muscular Atrophy
Jan 06, 2017: RG7916 Granted Orphan Drug Designation in the U.S. for the Treatment of Spinal Muscular Atrophy
Jan 05, 2017: PTC Therapeutics Announces Initiation of FIREFISH Study in Infant (Type I) SMA Patients
Dec 28, 2016: Biogen's atrophy drug SPINRAZA to cost $750,000 per patient
Dec 23, 2016: FDA approves first drug for spinal muscular atrophy
Dec 06, 2016: Study Finds New Treatment For Spinal Muscular Atrophy Safe For Infants
Nov 07, 2016: Biogen and Ionis Pharmaceuticals Announce SPINRAZA (nusinersen) Meets Primary Endpoint at Interim Analysis of Phase 3 CHERISH Study in Later-Onset Spinal Muscular Atrophy
Oct 28, 2016: Biogen's Regulatory Applications for Nusinersen as a Treatment for Spinal Muscular Atrophy Accepted by FDA and EMA
Oct 20, 2016: Spinal Muscular Atrophy Program Advances into Phase 2 Clinical Studies in SMA Patients with RG7916
Oct 08, 2016: New Data Presented at World Muscle Society Congress Support Potential Benefit of Investigational Treatment Nusinersen in Spinal Muscular Atrophy
Sep 26, 2016: Biogen Completes Rolling Submission of New Drug Application to FDA for Nusinersen as a Treatment for Spinal Muscular Atrophy
Sep 23, 2016: CHMP of the EMA has Granted Nusinersen Accelerated Assessment
Aug 04, 2016: Candidate Drug for Spinal Muscular Atrophy, with Roots in CSHL Research, Passes Major Hurdle
Aug 01, 2016: Biogen and Ionis Pharmaceuticals Report Nusinersen Meets Primary Endpoint at Interim Analysis of Phase 3 ENDEAR Study in Infantile-Onset 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 2017
Number of Products under Development by Companies, H1 2017
Number of Products under Development by Universities/Institutes, H1 2017
Products under Development by Companies, H1 2017
Products under Development by Companies, H1 2017 (Contd..1), H1 2017
Products under Development by Universities/Institutes, H1 2017
Number of Products by Stage and Target, H1 2017
Number of Products by Stage and Mechanism of Action, H1 2017
Number of Products by Stage and Route of Administration, H1 2017
Number of Products by Stage and Molecule Type, H1 2017
Spinal Muscular Atrophy (SMA) - Pipeline by AveXis Inc, H1 2017
Spinal Muscular Atrophy (SMA) - Pipeline by Cytokinetics Inc, H1 2017
Spinal Muscular Atrophy (SMA) - Pipeline by F. Hoffmann-La Roche Ltd, H1 2017
Spinal Muscular Atrophy (SMA) - Pipeline by Genethon SA, H1 2017
Spinal Muscular Atrophy (SMA) - Pipeline by Genzyme Corp, H1 2017
Spinal Muscular Atrophy (SMA) - Pipeline by Ionis Pharmaceuticals Inc, H1 2017
Spinal Muscular Atrophy (SMA) - Pipeline by Kowa Company Ltd, H1 2017
Spinal Muscular Atrophy (SMA) - Pipeline by Neurotune AG, H1 2017
Spinal Muscular Atrophy (SMA) - Pipeline by Novartis AG, H1 2017
Spinal Muscular Atrophy (SMA) - Pipeline by Paratek Pharmaceuticals Inc, H1 2017
Spinal Muscular Atrophy (SMA) - Pipeline by RaNA Therapeutics Inc, H1 2017
Spinal Muscular Atrophy (SMA) - Pipeline by Sarepta Therapeutics Inc, H1 2017
Spinal Muscular Atrophy (SMA) - Pipeline by Spotlight Innovation Inc, H1 2017
Spinal Muscular Atrophy (SMA) - Pipeline by WAVE Life Sciences Ltd, H1 2017
Spinal Muscular Atrophy (SMA) - Dormant Projects, H1 2017
Spinal Muscular Atrophy (SMA) - Dormant Projects, H1 2017 (Contd..1), H1 2017
Spinal Muscular Atrophy (SMA) - Discontinued Products, H1 2017

List of Figures
Number of Products under Development for Spinal Muscular Atrophy (SMA), H1 2017
Number of Products under Development by Companies, H1 2017
Number of Products under Development by Universities/Institutes, H1 2017
Number of Products by Targets, H1 2017
Number of Products by Stage and Targets, H1 2017
Number of Products by Mechanism of Actions, H1 2017
Number of Products by Stage and Mechanism of Actions, H1 2017
Number of Products by Routes of Administration, H1 2017
Number of Products by Stage and Routes of Administration, H1 2017
Number of Products by Molecule Types, H1 2017
Number of Products by Stage and Molecule Types, H1 2017

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