Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2)-Pipeline Review, H1 2018

Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2)-Pipeline Review, H1 2018

Jan 2018 Global Markets Direct Pharmaceutical70 Pages Price :
$ 3500
Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) - Pipeline Review, H1 2018

Summary

Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) pipeline Target constitutes close to 16 molecules. Out of which approximately 11 molecules are developed by companies and remaining by the universities/institutes. The latest report Survival Motor Neuron Protein - Pipeline Review, H1 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, Phase II and Preclinical stages are 2, 1 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.

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 its 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
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
Voyager Therapeutics Inc
WAVE Life Sciences Ltd
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
AVXS-101 - 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
Oligonucleotides to Activate SMN2 for Spinal Muscular Atrophy Type 1 - 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 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
VYSMN-101 - 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
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 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
Dec 13, 2017: AveXis Announces Plan to Initiate Phase 1 Trial in SMA Type 2 Utilizing Intrathecal Delivery of AVXS-101
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
Sep 29, 2017: AveXis Announces Plan to Initiate Pivotal Trial of AVXS-101 in SMA Type 1 Using Product from New GMP Commercial Process
Aug 24, 2017: Iowa State University biomedical researcher conducts promising trial of potential therapy for spinal muscular atrophy
Jul 01, 2017: Preliminary Phase 2 Data from Spinal Muscular Atrophy Program Presented at CureSMA Conference
Jun 22, 2017: Spotlight Innovation to Present Poster at the Cure SMA 2017 Annual SMA Conference
Jun 14, 2017: AveXis Announces Alignment with FDA on GMP Commercial Manufacturing Process for AVXS-101
Apr 25, 2017: AveXis Presents Results from Phase 1 Trial of AVXS-101 in SMA Type 1 at the Annual Meeting of the American Academy of Neurology
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, H1 2018
Number of Products under Development by Therapy Areas, H1 2018
Number of Products under Development by Indication, H1 2018
Number of Products under Development by Companies, H1 2018
Products under Development by Companies, H1 2018
Number of Products under Investigation by Universities/Institutes, H1 2018
Products under Investigation by Universities/Institutes, H1 2018
Number of Products by Stage and Mechanism of Actions, H1 2018
Number of Products by Stage and Route of Administration, H1 2018
Number of Products by Stage and Molecule Type, H1 2018
Pipeline by AveXis Inc, H1 2018
Pipeline by F. Hoffmann-La Roche Ltd, H1 2018
Pipeline by Genethon SA, H1 2018
Pipeline by Genzyme Corp, H1 2018
Pipeline by Ionis Pharmaceuticals Inc, H1 2018
Pipeline by Novartis AG, H1 2018
Pipeline by Sarepta Therapeutics Inc, H1 2018
Pipeline by Spotlight Innovation Inc, H1 2018
Pipeline by Voyager Therapeutics Inc, H1 2018
Pipeline by WAVE Life Sciences Ltd, H1 2018
Dormant Projects, H1 2018
Discontinued Products, H1 2018

List of Figures
Number of Products under Development by Stage of Development, H1 2018
Number of Products under Development by Therapy Areas, H1 2018
Number of Products under Development by Indications, H1 2018
Number of Products by Stage and 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

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