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    Survival Motor Neuron Protein Pipeline Review H2 2017

    Survival Motor Neuron Protein Pipeline Review H2 2017

    • Report Code ID: RW0001884179
    • Category Pharmaceuticals
    • No. of Pages 77
    • Publication Month Jul-17
    • Publisher Name Global Markets Direct
    Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) - Pipeline Review, H2 2017

    Summary

    According to the recently published report 'Survival Motor Neuron Protein - Pipeline Review, H2 2017'; Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) pipeline Target constitutes close to 17 molecules. Out of which approximately 12 molecules are developed by companies and remaining by the universities/institutes.

    Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) - Survival of motor neuron or survival motor neuron (SMN) is a protein encoded by the SMN1 and SMN2 genes. The SMN complex plays a catalyst role in the assembly of small nuclear ribonucleoproteins (snRNPs) . They play an important role in the splicing of cellular pre-mRNAs. They ensure the correct splicing of U12 intron-containing genes that is important for normal motor and proprioceptive neurons development. They are required for resolving RNA-DNA hybrids created by RNA polymerase II. They play a role in the metabolism of small nucleolar ribonucleoprotein.

    The report 'Survival Motor Neuron Protein - Pipeline Review, H2 2017' outlays comprehensive information on the Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) targeted therapeutics, complete with analysis by indications, stage of development, mechanism of action (MoA) , route of administration (RoA) and molecule type; that are being developed by Companies / Universities.

    It also reviews key players involved in Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) targeted therapeutics development with respective active and dormant or discontinued projects. Currently, The molecules developed by companies in Pre-Registration, Phase II and Preclinical stages are 1, 3 and 8 respectively. Similarly, the universities portfolio in Preclinical and Discovery stages comprises 4 and 1 molecules, respectively. Report covers products from therapy areas Central Nervous System and Respiratory which include indications Spinal Muscular Atrophy (SMA) and Cystic Fibrosis.

    Note: Certain content / sections in the pipeline guide may be removed or altered based on the availability and relevance of data.

    Scope

    - The report provides a snapshot of the global therapeutic landscape for Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2)
    - The report reviews Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) targeted therapeutics under development 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 in Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) targeted therapeutics and enlists all their major and minor projects
    - The report assesses Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) targeted therapeutics based on 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 and deals related to Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) targeted therapeutics

    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 the targeted therapy areas and indications for Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2)
    - Identify the use of drugs for target identification and drug repurposing
    - 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 Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) development landscape
    - 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
    Table of Contents 2
    List of Tables 4
    List of Figures 4
    Introduction 5
    Publisher Report Coverage 5
    Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) - Overview 6
    Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) - Therapeutics Development 7
    Products under Development by Stage of Development 7
    Products under Development by Therapy Area 8
    Products under Development by Indication 9
    Products under Development by Companies 10
    Products under Development by Universities/Institutes 12
    Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) - Therapeutics Assessment 14
    Assessment by Mechanism of Action 14
    Assessment by Route of Administration 15
    Assessment by Molecule Type 17
    Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) - Companies Involved in Therapeutics Development 19
    AveXis Inc 19
    Biogen Inc 19
    F. Hoffmann-La Roche Ltd 20
    Genethon SA 20
    Genzyme Corp 21
    Ionis Pharmaceuticals Inc 21
    Novartis AG 22
    Sarepta Therapeutics Inc 22
    Spotlight Innovation Inc 23
    Translate Bio Inc 23
    WAVE Life Sciences Ltd 24
    Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) - Drug Profiles 25
    ALB-111 - Drug Profile 25
    Antisense Oligonucleotide to Activate SMN2 for Spinal Muscular Atrophy - Drug Profile 26
    AVXS-101 - Drug Profile 27
    Gene Therapy to Activate SMN for Spinal Muscular Atrophy - Drug Profile 33
    Gene Therapy to Activate SMN1 for Spinal Muscular Atrophy - Drug Profile 34
    Gene Therapy to Activate SMN1 for Spinal Muscular Atrophy - Drug Profile 35
    IONISENAC-2.5Rx - Drug Profile 36
    LMI-070 - Drug Profile 37
    nusinersen - Drug Profile 38
    Oligonucleotide to Activate SMN2 for Spinal Muscular Atrophy - Drug Profile 47
    Oligonucleotides to Activate SMN2 for Spinal Muscular Atrophy Type 1 - Drug Profile 48
    PMO-25 - Drug Profile 49
    RG-7916 - Drug Profile 50
    Small Molecules for Spinal Muscular Atrophy - Drug Profile 52
    Small Molecules to Activate SMN2 for Spinal Muscular Atrophy - Drug Profile 53
    Small Molecules to Activate SMN2 for Spinal Muscular Atrophy - Drug Profile 54
    STL-182 - Drug Profile 55
    Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) - Dormant Products 56
    Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) - Discontinued Products 57
    Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) - Product Development Milestones 58
    Featured News & Press Releases 58
    Appendix 75
    Methodology 75
    Coverage 75
    Secondary Research 75
    Primary Research 75
    Expert Panel Validation 75
    Contact Us 75
    Disclaimer 76

    List of Tables

    Number of Products under Development by Stage of Development, H2 2017
    Number of Products under Development by Therapy Areas, H2 2017
    Number of Products under Development by Indication, H2 2017
    Number of Products under Development by Companies, H2 2017
    Products under Development by Companies, H2 2017
    Number of Products under Investigation by Universities/Institutes, H2 2017
    Products under Investigation by Universities/Institutes, H2 2017
    Number of Products by Stage and Mechanism of Actions, H2 2017
    Number of Products by Stage and Route of Administration, H2 2017
    Number of Products by Stage and Molecule Type, H2 2017
    Pipeline by AveXis Inc, H2 2017
    Pipeline by Biogen Inc, H2 2017
    Pipeline by F. Hoffmann-La Roche Ltd, H2 2017
    Pipeline by Genethon SA, H2 2017
    Pipeline by Genzyme Corp, H2 2017
    Pipeline by Ionis Pharmaceuticals Inc, H2 2017
    Pipeline by Novartis AG, H2 2017
    Pipeline by Sarepta Therapeutics Inc, H2 2017
    Pipeline by Spotlight Innovation Inc, H2 2017
    Pipeline by Translate Bio Inc, H2 2017
    Pipeline by WAVE Life Sciences Ltd, H2 2017
    Dormant Projects, H2 2017
    Discontinued Products, H2 2017

    List of Figures

    Number of Products under Development by Stage of Development, H2 2017
    Number of Products under Development by Therapy Areas, H2 2017
    Number of Products under Development by Indications, H2 2017
    Number of Products by Stage and Mechanism of Actions, H2 2017
    Number of Products by Routes of Administration, H2 2017
    Number of Products by Stage and Routes of Administration, H2 2017
    Number of Products by Molecule Types, H2 2017
    Number of Products by Stage and Molecule Types, H2 2017
    AveXis Inc
    Biogen Inc
    F. Hoffmann-La Roche Ltd
    Genethon SA
    Genzyme Corp
    Ionis Pharmaceuticals Inc
    Novartis AG
    Sarepta Therapeutics Inc
    Spotlight Innovation Inc
    Translate Bio Inc
    WAVE Life Sciences Ltd

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