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

  • ID: 4464937
  • Report
  • 113 pages
  • Global Markets Direct
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  • AveXis Inc
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  • F. Hoffmann-La Roche Ltd
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Spinal Muscular Atrophy (SMA) - Pipeline Review, H1 2018

Summary:

This 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

The Publisher'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 sourcedfrom the publisher'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 for Purchase:
  • 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.
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Note: Product cover images may vary from those shown
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FEATURED COMPANIES

  • AveXis Inc
  • Cytokinetics Inc
  • Exicure Inc
  • F. Hoffmann-La Roche Ltd
  • Genethon SA
  • Genzyme Corp
  • MORE
Introduction

The Publisher's 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

Jan 03, 2018: Cytokinetics Provides on CK-212

Dec 13, 2017: AveXis Announces Plan to Initiate Phase 1 Trial in SMA Type 2 Utilizing Intrathecal Delivery of AVXS-

Dec 11, 2017: Cytokinetics Publishes Clinical Trial Data for CK-212

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

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
Note: Product cover images may vary from those shown
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  • 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
  • Sare
Note: Product cover images may vary from those shown
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Note: Product cover images may vary from those shown
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