Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) - Pipeline Review, H2 2018

Publisher Name :
Date: 17-Jul-2018
No. of pages: 70

Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) - Pipeline Review, H2 2018

Summary

Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) pipeline Target constitutes close to 15 molecules. Out of which approximately 9 molecules are developed by companies and remaining by the universities/institutes. The latest report Survival Motor Neuron Protein - Pipeline Review, H2 2018, 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.

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 molecules developed by companies in Phase III and Preclinical stages are 3 and 6 respectively. Similarly, the universities portfolio in Preclinical and Discovery stages comprises 4 and 2 molecules, respectively. Report covers products from therapy areas Central Nervous System and Respiratory which include indications Spinal Muscular Atrophy (SMA) and Cystic Fibrosis.

Furthermore, this report 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. Driven by data and information sourced from 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.

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

Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) - Pipeline Review, H2 2018

Table of Contents
List of Tables
List of Figures
Introduction
Global Markets Direct Report Coverage
Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) - Overview
Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) - Therapeutics Development
Products under Development by Stage of Development
Products under Development by Therapy Area
Products under Development by Indication
Products under Development by Companies
Products under Development by Universities/Institutes
Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) - Therapeutics Assessment
Assessment by Mechanism of Action
Assessment by Route of Administration
Assessment by Molecule Type
Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) - Companies Involved in Therapeutics Development
AveXis Inc
F. Hoffmann-La Roche Ltd
Genethon SA
Genzyme Corp
Ionis Pharmaceuticals Inc
Novartis AG
Sarepta Therapeutics Inc
Spotlight Innovation Inc
Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) - Drug Profiles
ALB-111 - 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
branaplam - 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
IONISENAC-2.5Rx - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
onasemnogene abeparvovec - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
PMO-25 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
RG-7916 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Small Molecules 2 to Activate SMN2 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 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
STL-182 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) - Dormant Products
Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) - Discontinued Products
Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) - Product Development Milestones
Featured News & Press Releases
Jun 16, 2018: Updated Preliminary Data from SMA FIREFISH Program in Type 1 Babies Presented at the CureSMA Conference
May 08, 2018: AveXis Issues Community Statement on the SPR1NT Trial
May 08, 2018: Scientists investigate new strategy to treat spinal muscular atrophy in infants
Apr 25, 2018: AveXis Announces First Patient Dosed in Phase 3 Trial of AVXS-101 in Pre-Symptomatic SMA Types 1, 2 and 3
Apr 24, 2018: AveXis Presents Initial Data from Pivotal U.S. Trial for SMA Type 1 and 24-Month Follow-Up Data from Phase 1 Trial of AVXS-101 in SMA Type 1 at the Annual Meeting of the American Academy of Neurology
Apr 24, 2018: Preliminary Clinical Data from FIREFISH Trial in Type 1 SMA Patients Presented at the American Academy of Neurology Annual Meeting
Apr 19, 2018: AveXis to Present AVXS-101 Data at the Annual Meeting of the American Academy of Neurology
Apr 18, 2018: Preliminary Study Suggests Drug May Help Babies with Spinal Muscular Atrophy
Apr 15, 2018: Genentech Provided Update on RG-7916
Mar 27, 2018: AveXis Gene Therapy Awarded SAKIGAKE Designation for Spinal Muscular Atrophy Type 1
Mar 15, 2018: FIREFISH Transitions into Pivotal Phase
Mar 13, 2018: Newly Issued U.S. Patent Covers Spotlight Innovations Spinal Muscular Atrophy Drug Candidates
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
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer

List of Tables
Number of Products under Development by Stage of Development, H2 2018
Number of Products under Development by Therapy Areas, H2 2018
Number of Products under Development by Indication, H2 2018
Number of Products under Development by Companies, H2 2018
Products under Development by Companies, H2 2018
Number of Products under Investigation by Universities/Institutes, H2 2018
Products under Investigation by Universities/Institutes, H2 2018
Number of Products by Stage and Mechanism of Actions, H2 2018
Number of Products by Stage and Route of Administration, H2 2018
Number of Products by Stage and Molecule Type, H2 2018
Pipeline by AveXis Inc, H2 2018
Pipeline by F. Hoffmann-La Roche Ltd, H2 2018
Pipeline by Genethon SA, H2 2018
Pipeline by Genzyme Corp, H2 2018
Pipeline by Ionis Pharmaceuticals Inc, H2 2018
Pipeline by Novartis AG, H2 2018
Pipeline by Sarepta Therapeutics Inc, H2 2018
Pipeline by Spotlight Innovation Inc, H2 2018
Dormant Projects, H2 2018
Discontinued Products, H2 2018

List of Figures
Number of Products under Development by Stage of Development, H2 2018
Number of Products under Development by Therapy Areas, H2 2018
Number of Products under Development by Top 10 Indications, H2 2018
Number of Products by Stage and Mechanism of Actions, H2 2018
Number of Products by Routes of Administration, H2 2018
Number of Products by Stage and Routes of Administration, H2 2018
Number of Products by Molecule Types, H2 2018
Number of Products by Stage and Molecule Types, H2 2018
  • Global Compounding Pharmacy Market 2018 by Manufacturers, Countries, Type and Application, Forecast to 2023
    Published: 17-Aug-2018        Price: US 3480 Onwards        Pages: 129
    The traditional role of compounding pharmacies is to make drugs prescribed by doctors for specific patients with needs that can't be met by commercially available drugs. For example: A young child may need a small, liquid dose of a drug made only in adult-dosage tablets. A person may be allergic to one of the ingredients in the commercial version of a drug. A compounding pharmacy would make the proper dosage and form of the drug without any offending ingredients. Comp......
  • Global Human Prothrombin Complex Market 2018 by Manufacturers, Regions, Type and Application, Forecast to 2023
    Published: 17-Aug-2018        Price: US 3480 Onwards        Pages: 134
    Prothrombin complex concentrate (PCC), also known as factor IX complex, is a medication made up of blood clotting factors II, IX, and X. Some versions also contain factor VII. It is used to treat and prevent bleeding in hemophilia B if pure factor IX is not available. Scope of the Report: This report focuses on the Human Prothrombin Complex in global market, especially in North America, Europe and Asia-Pacific, South America, Middle East and Africa. This report categorizes ......
  • Global (North America, Europe, Asia-Pacific, South America, Middle East and Africa) Human Prothrombin Complex Market 2018, Forecast to 2023
    Published: 17-Aug-2018        Price: US 4880 Onwards        Pages: 137
    Prothrombin complex concentrate (PCC), also known as factor IX complex, is a medication made up of blood clotting factors II, IX, and X. Some versions also contain factor VII. It is used to treat and prevent bleeding in hemophilia B if pure factor IX is not available. Scope of the Report: This report focuses on the Human Prothrombin Complex in global market, especially in North America, Europe and Asia-Pacific, South America, Middle East and Africa. This report categorizes ......
  • Global Medical Rubber Stopper Market 2018 by Manufacturers, Regions, Type and Application, Forecast to 2023
    Published: 17-Aug-2018        Price: US 3480 Onwards        Pages: 145
    Medical Rubber Stopper is widely used for the pharmaceutical application which made from butyl rubber and are available in various sizes and styles depending on applications. Scope of the Report: This report focuses on the Medical Rubber Stopper in global market, especially in North America, Europe and Asia-Pacific, South America, Middle East and Africa. This report categorizes the market based on manufacturers, regions, type and application. The classification of Me......
  • Global (North America, Europe, Asia-Pacific, South America, Middle East and Africa) Medical Rubber Stopper Market 2018, Forecast to 2023
    Published: 17-Aug-2018        Price: US 4880 Onwards        Pages: 145
    Medical Rubber Stopper is widely used for the pharmaceutical application which made from butyl rubber and are available in various sizes and styles depending on applications. Scope of the Report: This report focuses on the Medical Rubber Stopper in global market, especially in North America, Europe and Asia-Pacific, South America, Middle East and Africa. This report categorizes the market based on manufacturers, regions, type and application. The classification of Me......
  • Global Internal Nasal Dilators Market 2018 by Manufacturers, Regions, Type and Application, Forecast to 2023
    Published: 17-Aug-2018        Price: US 3480 Onwards        Pages: 128
    Internal Nasal Dilators are small devices that are made from plastic, or rubber. They fit directly into the nostril and remain in place all night to aid your breathing. The dilators are easy to insert and do not cause any undue discomfort when you have them inserted. Scope of the Report: This report focuses on the Internal Nasal Dilators in global market, especially in North America, Europe and Asia-Pacific, South America, Middle East and Africa. This report categorizes the......
  • Global (North America, Europe, Asia-Pacific, South America, Middle East and Africa) Internal Nasal Dilators Market 2018, Forecast to 2023
    Published: 17-Aug-2018        Price: US 4880 Onwards        Pages: 128
    Internal Nasal Dilators are small devices that are made from plastic, or rubber. They fit directly into the nostril and remain in place all night to aid your breathing. The dilators are easy to insert and do not cause any undue discomfort when you have them inserted. Scope of the Report: This report focuses on the Internal Nasal Dilators in global market, especially in North America, Europe and Asia-Pacific, South America, Middle East and Africa. This report categorizes the......
  • Global Self-Monitoring Blood Glucose (SMBG) Devices Market 2018 by Manufacturers, Regions, Type and Application, Forecast to 2023
    Published: 17-Aug-2018        Price: US 3480 Onwards        Pages: 129
    Self-monitoring of blood glucose (SMBG) is an important component of modern therapy for diabetes mellitus. SMBG has been recommended for people with diabetes and their health care professionals in order to achieve a specific level of glycemic control and to prevent hypoglycemia. The goal of SMBG is to collect detailed information about blood glucose levels at many time points to enable maintenance of a more constant glucose level by more precise regimens. It can be used to aid in the adjustment ......
  • Global (North America, Europe, Asia-Pacific, South America, Middle East and Africa) Self-Monitoring Blood Glucose (SMBG) Devices Market 2018, Forecast to 2023
    Published: 17-Aug-2018        Price: US 4880 Onwards        Pages: 131
    Self-monitoring of blood glucose (SMBG) is an important component of modern therapy for diabetes mellitus. SMBG has been recommended for people with diabetes and their health care professionals in order to achieve a specific level of glycemic control and to prevent hypoglycemia. The goal of SMBG is to collect detailed information about blood glucose levels at many time points to enable maintenance of a more constant glucose level by more precise regimens. It can be used to aid in the adjustment ......
  • 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