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Published: Dec, 2016 | Pages:
82 | Publisher: Global Markets Direct
Industry: Pharmaceuticals & Healthcare | Report Format: Electronic (PDF)
Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) - Pipeline Review, H2 2016 Summary Global Markets Direct's, 'Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) - Pipeline Review, H2 2016', provides in depth analysis on Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) targeted pipeline therapeutics. The report provides 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. 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 Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) targeted therapeutics development 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. *Updated report will be delivered in 48 hours of order confirmation. 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 ands scope
Table of Contents
Table of Contents 2 List of Tables 5 List of Figures 5 Introduction 6 Global Markets Direct Report Coverage 6 Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) Overview 7 Therapeutics Development 8 Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) - Products under Development by Stage of Development 8 Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) - Products under Development by Therapy Area 9 Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) - Products under Development by Indication 10 Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) - Pipeline Products Glance 11 Late Stage Products 11 Early Stage Products 12 Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) - Products under Development by Companies 13 Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) - Products under Development by Universities/Institutes 15 Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) - Therapeutics Assessment 17 Assessment by Monotherapy/Combination Products 17 Assessment by Mechanism of Action 18 Assessment by Route of Administration 19 Assessment by Molecule Type 21 Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) - Companies Involved in Therapeutics Development 23 AveXis Inc 23 Biogen Inc 24 F. Hoffmann-La Roche Ltd 25 Genethon SA 26 Genzyme Corp 27 Novartis AG 28 Sarepta Therapeutics Inc 29 Spotlight Innovation Inc 30 Voyager Therapeutics Inc 31 WAVE Life Sciences Ltd 32 Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) - Drug Profiles 33 ALB-111 - Drug Profile 33 Product Description 33 Mechanism Of Action 33 R&D Progress 33 Antisense Oligonucleotide to Activate SMN2 for Spinal Muscular Atrophy - Drug Profile 34 Product Description 34 Mechanism Of Action 34 R&D Progress 34 AVXS-101 - Drug Profile 35 Product Description 35 Mechanism Of Action 35 R&D Progress 35 Gene Therapy to Activate SMN for Spinal Muscular Atrophy - Drug Profile 39 Product Description 39 Mechanism Of Action 39 R&D Progress 39 Gene Therapy to Activate SMN1 for Spinal Muscular Atrophy - Drug Profile 40 Product Description 40 Mechanism Of Action 40 R&D Progress 40 Gene Therapy to Activate SMN1 for Spinal Muscular Atrophy - Drug Profile 41 Product Description 41 Mechanism Of Action 41 R&D Progress 41 LMI-070 - Drug Profile 42 Product Description 42 Mechanism Of Action 42 R&D Progress 42 nusinersen - Drug Profile 43 Product Description 43 Mechanism Of Action 43 R&D Progress 43 Oligonucleotides to Activate SMN2 for Spinal Muscular Atrophy Type 1 - Drug Profile 50 Product Description 50 Mechanism Of Action 50 R&D Progress 50 PMO-25 - Drug Profile 51 Product Description 51 Mechanism Of Action 51 R&D Progress 51 RG-7800 - Drug Profile 52 Product Description 52 Mechanism Of Action 52 R&D Progress 52 RG-7916 - Drug Profile 54 Product Description 54 Mechanism Of Action 54 R&D Progress 54 Small Molecules for Spinal Muscular Atrophy - Drug Profile 56 Product Description 56 Mechanism Of Action 56 R&D Progress 56 Small Molecules to Activate SMN2 for Spinal Muscular Atrophy - Drug Profile 57 Product Description 57 Mechanism Of Action 57 R&D Progress 57 Small Molecules to Activate SMN2 for Spinal Muscular Atrophy - Drug Profile 58 Product Description 58 Mechanism Of Action 58 R&D Progress 58 STL-182 - Drug Profile 59 Product Description 59 Mechanism Of Action 59 R&D Progress 59 VYSMN-101 - Drug Profile 60 Product Description 60 Mechanism Of Action 60 R&D Progress 60 Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) - Dormant Projects 61 Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) - Featured News & Press Releases 62 Dec 06, 2016: Study Finds New Treatment For Spinal Muscular Atrophy Safe For Infants 62 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 63 Nov 01, 2016: AveXis Announces Single-Arm Design for U.S. Pivotal Study of AVXS-101 in SMA Type 1 Patients 64 Oct 28, 2016: Biogen's Regulatory Applications for Nusinersen as a Treatment for Spinal Muscular Atrophy Accepted by FDA and EMA 65 Oct 20, 2016: Spinal Muscular Atrophy Program Advances into Phase 2 Clinical Studies in SMA Patients with RG7916 66 Oct 08, 2016: New Data Presented at World Muscle Society Congress Support Potential Benefit of Investigational Treatment Nusinersen in Spinal Muscular Atrophy 67 Oct 08, 2016: AveXis Reports Interim Data from Ongoing Phase 1 Clinical Trial of AVXS-101 in Spinal Muscular Atrophy Type 1 as Presented at the International Annual Congress of the World Muscle Society 69 Sep 26, 2016: Biogen Completes Rolling Submission of New Drug Application to FDA for Nusinersen as a Treatment for Spinal Muscular Atrophy 71 Sep 23, 2016: CHMP of the EMA has Granted Nusinersen Accelerated Assessment 72 Aug 11, 2016: AveXis Reports Interim Data from Ongoing Phase 1 Trial of AVXS-101 in Spinal Muscular Atrophy Type 1 72 Aug 04, 2016: Candidate Drug for Spinal Muscular Atrophy, with Roots in CSHL Research, Passes Major Hurdle 75 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 76 Jul 25, 2016: Update on LMI070 for the Treatment of Type 1 SMA 77 Jul 20, 2016: AveXis Receives U.S. FDA Breakthrough Therapy Designation for AVXS-101 Gene Replacement Therapy for Spinal Muscular Atrophy Type 1 78 May 16, 2016: AveXis Presents Pulmonary Data from Ongoing Phase 1 Trial of AVXS-101 in Spinal Muscular Atrophy Type 1 78 Appendix 81 Methodology 81 Coverage 81 Secondary Research 81 Primary Research 81 Expert Panel Validation 81 Contact Us 81 Disclaimer 82
List of Tables Number of Products under Development for, H2 2016 8 Number of Products under Development by Therapy Area, H2 2016 9 Number of Products under Development by Indication, H2 2016 10 Comparative Analysis by Late Stage Development, H2 2016 11 Comparative Analysis by Early Stage Products, H2 2016 12 Number of Products under Development by Companies, H2 2016 13 Products under Development by Companies, H2 2016 14 Number of Products under Investigation by Universities/Institutes, H2 2016 15 Products under Investigation by Universities/Institutes, H2 2016 16 Assessment by Monotherapy/Combination Products, H2 2016 17 Number of Products by Stage and Mechanism of Action, H2 2016 18 Number of Products by Stage and Route of Administration, H2 2016 20 Number of Products by Stage and Molecule Type, H2 2016 22 Pipeline by AveXis Inc, H2 2016 23 Pipeline by Biogen Inc, H2 2016 24 Pipeline by F. Hoffmann-La Roche Ltd, H2 2016 25 Pipeline by Genethon SA, H2 2016 26 Pipeline by Genzyme Corp, H2 2016 27 Pipeline by Novartis AG, H2 2016 28 Pipeline by Sarepta Therapeutics Inc, H2 2016 29 Pipeline by Spotlight Innovation Inc, H2 2016 30 Pipeline by Voyager Therapeutics Inc, H2 2016 31 Pipeline by WAVE Life Sciences Ltd, H2 2016 32 Dormant Projects, H2 2016 61
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