Spinal Muscular Atrophy (SMA) - Pipeline Review, H1 2018

Publisher Name :
Date: 20-Feb-2018
No. of pages: 113
Inquire Before Buying

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Spinal Muscular Atrophy (SMA) - Pipeline Review, H1 2018, 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 2018, 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 Phase III, Phase II, Phase I, Preclinical and Discovery stages are 4, 3, 1, 13 and 3 respectively. Similarly, the Universities portfolio in Preclinical and Discovery stages comprises 7 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.

Spinal Muscular Atrophy (SMA) - Pipeline Review, H1 2018

Table of Contents

List of Tables
List of Figures
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
BioMarin Pharmaceutical Inc
Cytokinetics Inc
Exicure Inc
F. Hoffmann-La Roche Ltd
Genethon SA
Genzyme Corp
Kowa Co Ltd
Neurotune AG
Novartis AG
Paratek Pharmaceuticals Inc
Recursion Pharmaceuticals Inc
Sarepta Therapeutics Inc
Scholar Rock Inc
Spotlight Innovation Inc
Voyager Therapeutics Inc
Spinal Muscular Atrophy (SMA) - Drug Profiles
AAD-2004 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
ALB-111 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
amifampridine phosphate - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Antisense Oligonucleotide to Activate SMN2 for Spinal Muscular Atrophy - 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
AVXS-101 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
branaplam - 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
K-828SP - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
NT-1654 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
olesoxime - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Oligonucleotides for Spinal Muscular Atrophy and Huntington Disease - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
PMO-25 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
PTKSMA-1 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
REC-0000716 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
REC-0001202 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
RG-7916 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Small Molecule to Inhibit GSK-3 for Spinal Muscular Atrophy - 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 for Spinal Muscular Atrophy - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Small Molecules for Spinal Muscular Atrophy - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Small Molecules for X-Linked Infantile 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 SMN2 for Spinal Muscular Atrophy - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Small Molecules to Inhibit SRSF10 and BCLAF1 for HIV Infection, Spinal Muscular Atrophy and Colon Cancer - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
SRK-105 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
STL-182 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
VYSMN-101 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Xcel-hNu - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Xcel-hNuP - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Spinal Muscular Atrophy (SMA) - Dormant Projects
Spinal Muscular Atrophy (SMA) - Discontinued Products
Spinal Muscular Atrophy (SMA) - Product Development Milestones
Featured News & Press Releases
Feb 02, 2018: Scholar Rock Announces Publication of Comprehensive Pharmacological Study of SRK-015, a Novel Inhibitor of Myostatin Activation, Supporting the Treatment of Muscle Atrophy Disorders
Jan 30, 2018: AveXis to Initiate Screening for Remaining Patients in Pivotal Trial of AVXS-101 for SMA Type 1 Following Review of Preliminary Data from First Three Patients
Jan 27, 2018: Preliminary Data from FIREFISH trial in Type 1 SMA Infants Presented at the International Scientific Congress on Spinal Muscular Atrophy
Jan 23, 2018: Data from RG7916 Programs in SMA to be Presented at the International Scientific Congress on Spinal Muscular Atrophy
Jan 23, 2018: Scholar Rock and Collaborators at Harvard Medical School and Northeastern University Elucidate Molecular Basis of Myostatin Activation - a Key Physiological Process in Muscle Health
Jan 16, 2018: AveXis Announces Expanded Clinical Development Program for AVXS-101 in Spinal Muscular Atrophy
Jan 04, 2018: AveXis Announces Alignment with FDA on Next Steps Toward a BLA Submission for AVXS-101 in SMA Type 1
Jan 03, 2018: Cytokinetics Provides on CK-2127107
Dec 13, 2017: AveXis Announces Plan to Initiate Phase 1 Trial in SMA Type 2 Utilizing Intrathecal Delivery of AVXS-101
Dec 11, 2017: Cytokinetics Publishes Clinical Trial Data for CK-2127107
Nov 21, 2017: Catalyst Pharmaceuticals Announces Phase 2 Study of Firdapse in Ambulatory Patients with Spinal Muscular Atrophy (SMA)
Nov 01, 2017: AveXis Announces New England Journal of Medicine Publication of Phase 1 Data of AVXS-101 Gene Replacement Therapy in Spinal Muscular Atrophy Type 1
Oct 12, 2017: Spinal Muscular Atrophy Program Advances into Pivotal Study in SMA Patients
Oct 03, 2017: AveXis to Report Top-line Data from the Phase 1 Clinical Trial of AVXS-101 in SMA Type 1 at the International Annual Congress of the World Muscle Society
Oct 03, 2017: RG7916 Increased SMN Protein Production in SUNFISH Clinical Trial in Patients with Type 2/3 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 2018
Number of Products under Development by Companies, H1 2018
Number of Products under Development by Companies, H1 2018 (Contd..1), H1 2018
Number of Products under Development by Universities/Institutes, H1 2018
Products under Development by Companies, H1 2018
Products under Development by Companies, H1 2018 (Contd..1), H1 2018
Products under Development by Universities/Institutes, H1 2018
Number of Products by Stage and Target, H1 2018
Number of Products by Stage and Mechanism of Action, H1 2018
Number of Products by Stage and Route of Administration, H1 2018
Number of Products by Stage and Molecule Type, H1 2018
Spinal Muscular Atrophy (SMA) - Pipeline by AveXis Inc, H1 2018
Spinal Muscular Atrophy (SMA) - Pipeline by BioMarin Pharmaceutical Inc, H1 2018
Spinal Muscular Atrophy (SMA) - Pipeline by Cytokinetics Inc, H1 2018
Spinal Muscular Atrophy (SMA) - Pipeline by Exicure Inc, H1 2018
Spinal Muscular Atrophy (SMA) - Pipeline by F. Hoffmann-La Roche Ltd, H1 2018
Spinal Muscular Atrophy (SMA) - Pipeline by Genethon SA, H1 2018
Spinal Muscular Atrophy (SMA) - Pipeline by Genzyme Corp, H1 2018
Spinal Muscular Atrophy (SMA) - Pipeline by Kowa Co Ltd, H1 2018
Spinal Muscular Atrophy (SMA) - Pipeline by Neurotune AG, H1 2018
Spinal Muscular Atrophy (SMA) - Pipeline by Novartis AG, H1 2018
Spinal Muscular Atrophy (SMA) - Pipeline by Paratek Pharmaceuticals Inc, H1 2018
Spinal Muscular Atrophy (SMA) - Pipeline by Recursion Pharmaceuticals Inc, H1 2018
Spinal Muscular Atrophy (SMA) - Pipeline by Sarepta Therapeutics Inc, H1 2018
Spinal Muscular Atrophy (SMA) - Pipeline by Scholar Rock Inc, H1 2018
Spinal Muscular Atrophy (SMA) - Pipeline by Spotlight Innovation Inc, H1 2018
Spinal Muscular Atrophy (SMA) - Pipeline by Voyager Therapeutics Inc, H1 2018
Spinal Muscular Atrophy (SMA) - Dormant Projects, H1 2018
Spinal Muscular Atrophy (SMA) - Dormant Projects, H1 2018 (Contd..1), H1 2018
Spinal Muscular Atrophy (SMA) - Discontinued Products, H1 2018

List of Figures

Number of Products under Development for Spinal Muscular Atrophy (SMA), H1 2018
Number of Products under Development by Companies, H1 2018
Number of Products under Development by Universities/Institutes, H1 2018
Number of Products by Top 10 Targets, H1 2018
Number of Products by Stage and Top 10 Targets, H1 2018
Number of Products by Top 10 Mechanism of Actions, H1 2018
Number of Products by Stage and Top 10 Mechanism of Actions, H1 2018
Number of Products by Routes of Administration, H1 2018
Number of Products by Stage and Routes of Administration, H1 2018
Number of Products by Molecule Types, H1 2018
Number of Products by Stage and Molecule Types, H1 2018
  • Global Oligonucleotide Therapeutics Market 2018-2022
    Published: 13-Jun-2018        Price: US 3500 Onwards        Pages: 103
    About Oligonucleotide Therapeutics Oligonucleotides are short nucleotides sequences of DNA or RNA molecules called oligomers. Oligonucleotide therapeutics target and inhibit the expression of certain diseases-causing genes, thus forming a new class of therapeutics to treat chronic diseases. The analysts forecast Global Oligonucleotide Therapeutics Market to grow at a CAGR of 9.52% during the period 2018-2022. Covered in this report This repor......
  • Global Migraine Therapeutics Market 2018-2022
    Published: 13-Jun-2018        Price: US 3500 Onwards        Pages: 102
    About migraine therapeutics Migraine therapeutics caters to the treatment of acute or chronic migraine. It can be either preventive or abortive, that determines the treatment of migraine. The analysts forecast Global Migraine Therapeutics Market to grow at a CAGR of 5.38% during the period 2018-2022. Covered in this report This report covers the present scenario and the growth prospects of the migraine therapeutics market. To calcula......
  • Global Sclerotherapy Market 2018 by Manufacturers, Regions, Type and Application, Forecast to 2023
    Published: 11-Jun-2018        Price: US 3480 Onwards        Pages: 135
    Sclerotherapy is a procedure used to treat blood vessels or blood vessel malformations (vascular malformations) and also those of the lymphatic system. A medicine is injected into the vessels, which makes them shrink. It is used for children and young adults with vascular or lymphatic malformations. In adults, sclerotherapy is often used to treat spider veins, smaller varicose veins, and hemorrhoids. Scope of the Report: This report focuses on the Sclerotherapy in g......
  • Global (North America, Europe, Asia-Pacific, South America, Middle East and Africa) Sclerotherapy Market 2018, Forecast to 2023
    Published: 11-Jun-2018        Price: US 4880 Onwards        Pages: 130
    Sclerotherapy is a procedure used to treat blood vessels or blood vessel malformations (vascular malformations) and also those of the lymphatic system. A medicine is injected into the vessels, which makes them shrink. It is used for children and young adults with vascular or lymphatic malformations. In adults, sclerotherapy is often used to treat spider veins, smaller varicose veins, and hemorrhoids. Scope of the Report: This report focuses on the Sclerotherapy in g......
  • Global Brachytherapy Afterloaders, Brachytherapy Seeds Market by Manufacturers, Regions, Type and Application, Forecast to 2023
    Published: 09-Jun-2018        Price: US 3480 Onwards        Pages: 136
    Brachytherapy Afterloaders, is kind of machine used in brachytherapy to deliverer radioactive sources into the treatment site, to avoid risk of radiation exposure to clinical staff. Brachytherapy Seeds is radiation sources enclosed within a non-radioactive capsule. The common radiation sources are Iodine-125 Palladium-103 Cesium-137. Scope of the Report: This report focuses on the Brachytherapy Afterloaders, Brachytherapy Seeds in global market, especially in N......
  • Global (North America, Europe, Asia-Pacific, South America, Middle East and Africa) Brachytherapy Afterloaders, Brachytherapy Seeds Market 2018, Forecast to 2023
    Published: 09-Jun-2018        Price: US 4880 Onwards        Pages: 130
    Brachytherapy Afterloaders, is kind of machine used in brachytherapy to deliverer radioactive sources into the treatment site, to avoid risk of radiation exposure to clinical staff. Brachytherapy Seeds is radiation sources enclosed within a non-radioactive capsule. The common radiation sources are Iodine-125 Palladium-103 Cesium-137. Scope of the Report: This report focuses on the Brachytherapy Afterloaders, Brachytherapy Seeds in global market, especially in N......
  • United States Human Microbiome Therapeutics Market Report 2018
    Published: 04-Jun-2018        Price: US 3800 Onwards        Pages: 97
    In this report, the United States Human Microbiome Therapeutics market is valued at USD XX million in 2017 and is expected to reach USD XX million by the end of 2025, growing at a CAGR of XX% between 2017 and 2025. Geographically, this report splits the United States market into seven regions: - The West - Southwest - The Middle Atlantic - New England - The South - The Midwest with sales (volume), revenue (value), market share and g......
  • Psoriasis Therapeutics in Asia-Pacific Markets to 2024 - Increasing Prevalence, Advent of Novel Biologics and Pipeline Drugs to Drive the Market
    Published: 01-Jun-2018        Price: US 4995 Onwards        Pages: 125
    Psoriasis is a common, chronic inflammatory skin condition affecting around 2-3% of the world’s population. While the main cause of psoriasis remains uncertain, immunologic, genetic, and environmental factors appear to play a role. Common symptoms of psoriasis include inflammation, swelling, and patches of red, itchy, flaky skin called skin lesions. The most common sites of involvement are the scalp, elbows and knees, followed by nails, hands, feet and trunk. It is estimated tha......
  • Global Acute Ischemic Stroke Therapeutics Market Professional Survey Report 2018
    Published: 01-Jun-2018        Price: US 3500 Onwards        Pages: 104
    This report studies Acute Ischemic Stroke Therapeutics in Global market, especially in North America, China, Europe, Southeast Asia, Japan and India, with production, revenue, consumption, import and export in these regions, from 2013 to 2018, and forecast to 2025. This report focuses on top manufacturers in global market, with production, price, revenue and market share for each manufacturer, covering - Bayer - Boehringer Ingelheim - Roche - Sanofi......
  • SERVICES
    Value for Money
    We believe in "optimum utilization of available budget and resources". While servicing our clients' (your) market research requirements, we keep the same approach in focus to help you get the best value for your $$s.
    Ever Growing Inventory
    Ranging from the smallest feasible / required data (datasheets, data facts, SWOT analysis, company profiles, etc) to full research reports that help you make decisions, our inventory is updated almost on a daily basis with the latest industry reports from domain experts that track more than 5000 niche sectors.
    One Stop Solution
    Need a custom research report on medical devices market? Require all available business intelligence on 3D printing industry? Exploring F&B sector of a particular country/region? RnRMarketResearch.com is your one-stop-solution to all market intelligence needs. We not only offer custom research and consulting services, we also "bundle" reports to meet your needs and help you fetch the data analysis you require for your business.
    Dedicated Client Engagement
    Not limited to only "finding" relevant reports for you, our client engagement team dedicates its efforts to understand your "business need" and accordingly maps available research data to help you move forward. Call "your" client engagement executive any time of your day and get your questions answered in order to make the correct business decision.
    Saving Time and Efforts
    Simply share your research requirement details with us and let us do all the hard work to find required intelligence for you. When you add up our "one stop solution" and "dedicated client engagement" services mentioned above, you obviously know the time and effort saving you do by working with us.
    Payment Flexibility
    Working with Fortune 500 organizations, we understand the importance of being flexible for payments. Share your payment terms with us and we will surely match up to them to ensure you get access to required business intelligence data without having to wait for the payment to be done.
    Post-Purchase Research Support
    Have questions after reading a report / datasheet bought through us? Not sure about the methodology used for data available in the research? Talk to us / Share your questions with us and if required, we will connect you with the analyst(s)/author(s) of the report(s) and ensure you get satisfactory answers for the same. Need more data / analysis / report(s) on the topic of your research/project? The RnRMarketResearch.com team is here for you 24X7 to support you with your post-purchase requirements. Subscription Offers & Packages (Get in touch with us for more details - sales@rnrmarketresearch.com / +1 888 391 5441 )
    • Ad Hoc
    • Pay - as - you - go / Bucket Subscriptions
    • Fixed Cost for #of reports
    • Customize / Personalize as per your needs