Survival Motor Neuron Protein - Pipeline Review, H2 2019
Survival Motor Neuron Protein - Pipeline Review, H2 2019
SUMMARY
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.
Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) pipeline Target constitutes close to 19 molecules. Out of which approximately 11 molecules are developed by companies and remaining by the universities/institutes. The molecules developed by companies in Pre-Registration, Phase III, Phase II and Preclinical stages are 1, 1, 2 and 7 respectively. Similarly, the universities portfolio in Phase II, Preclinical and Discovery stages comprises 2, 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.
The latest report Survival Motor Neuron Protein - Pipeline Review, H2 2019, 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. 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.
The report is built using 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
SUMMARY
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.
Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) pipeline Target constitutes close to 19 molecules. Out of which approximately 11 molecules are developed by companies and remaining by the universities/institutes. The molecules developed by companies in Pre-Registration, Phase III, Phase II and Preclinical stages are 1, 1, 2 and 7 respectively. Similarly, the universities portfolio in Phase II, Preclinical and Discovery stages comprises 2, 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.
The latest report Survival Motor Neuron Protein - Pipeline Review, H2 2019, 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. 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.
The report is built using 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
- 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
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
AurimMed Pharma Inc
AveXis Inc
Biogen Inc
Exicure Inc
F. Hoffmann-La Roche Ltd
Ionis Pharmaceuticals Inc
Novartis AG
Sarepta Therapeutics Inc
Shift Pharmaceuticals
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
BIIB-089 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
branaplam - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
E-1V111 - 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
GTP-201 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
IONISENAC-2.5Rx - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
LDN-5178 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Oligonucleotide to Activate SMN2 for Spinal Muscular Atrophy - 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
risdiplam - 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
Nov 12, 2019: Roche and PTC report positive data for risdiplam in SMA
Oct 30, 2019: Novartis’ Zolgensma experiences further safety issues
Oct 02, 2019: Risdiplam Spinal Muscular Atrophy data demonstrating continued benefit presented at World Muscle Society Congress
Sep 19, 2019: Novartis presents additional Zolgensma data in children with SMA
Aug 14, 2019: Brother scientists no longer with Novartis after alleged data manipulation
Aug 13, 2019: US Senator seeks records linked to Novartis Zolgensma data issues
Aug 07, 2019: Novartis defends Zolgensma in face of FDA’s data concerns
Aug 06, 2019: Statement on data accuracy issues with recently approved gene therapy
Jul 31, 2019: Connecticut Childrens administers first gene therapy for spinal muscular atrophy (SMA) in the state
Jul 25, 2019: AveXis Statement on Access to Zolgensma (onasemnogene abeparvovec-xioi)
Jul 22, 2019: BIA Separations selected for manufacturing process development support for gene therapy pipeline
Jul 16, 2019: Catalent Biologics announces agreement to provide additional production of approved gene therapy treatment for spinal muscular atrophy
Jun 13, 2019: UF Health patient first to receive new gene therapy for neuromuscular disease since FDA approval
Jun 05, 2019: New gene therapy poised to transform care for spinal muscular atrophy
May 30, 2019: Newly approved Spinal Muscular Atrophy Gene Therapy, Zolgensma, validates AskBio Gene Therapy Platform
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer
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
AurimMed Pharma Inc
AveXis Inc
Biogen Inc
Exicure Inc
F. Hoffmann-La Roche Ltd
Ionis Pharmaceuticals Inc
Novartis AG
Sarepta Therapeutics Inc
Shift Pharmaceuticals
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
BIIB-089 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
branaplam - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
E-1V111 - 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
GTP-201 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
IONISENAC-2.5Rx - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
LDN-5178 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Oligonucleotide to Activate SMN2 for Spinal Muscular Atrophy - 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
risdiplam - 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
Nov 12, 2019: Roche and PTC report positive data for risdiplam in SMA
Oct 30, 2019: Novartis’ Zolgensma experiences further safety issues
Oct 02, 2019: Risdiplam Spinal Muscular Atrophy data demonstrating continued benefit presented at World Muscle Society Congress
Sep 19, 2019: Novartis presents additional Zolgensma data in children with SMA
Aug 14, 2019: Brother scientists no longer with Novartis after alleged data manipulation
Aug 13, 2019: US Senator seeks records linked to Novartis Zolgensma data issues
Aug 07, 2019: Novartis defends Zolgensma in face of FDA’s data concerns
Aug 06, 2019: Statement on data accuracy issues with recently approved gene therapy
Jul 31, 2019: Connecticut Childrens administers first gene therapy for spinal muscular atrophy (SMA) in the state
Jul 25, 2019: AveXis Statement on Access to Zolgensma (onasemnogene abeparvovec-xioi)
Jul 22, 2019: BIA Separations selected for manufacturing process development support for gene therapy pipeline
Jul 16, 2019: Catalent Biologics announces agreement to provide additional production of approved gene therapy treatment for spinal muscular atrophy
Jun 13, 2019: UF Health patient first to receive new gene therapy for neuromuscular disease since FDA approval
Jun 05, 2019: New gene therapy poised to transform care for spinal muscular atrophy
May 30, 2019: Newly approved Spinal Muscular Atrophy Gene Therapy, Zolgensma, validates AskBio Gene Therapy Platform
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 2019
Number of Products under Development by Therapy Areas, H2 2019
Number of Products under Development by Indication, H2 2019
Number of Products under Development by Companies, H2 2019
Products under Development by Companies, H2 2019
Number of Products under Investigation by Universities/Institutes, H2 2019
Products under Investigation by Universities/Institutes, H2 2019
Number of Products by Stage and Mechanism of Actions, H2 2019
Number of Products by Stage and Route of Administration, H2 2019
Number of Products by Stage and Molecule Type, H2 2019
Pipeline by AurimMed Pharma Inc, H2 2019
Pipeline by AveXis Inc, H2 2019
Pipeline by Biogen Inc, H2 2019
Pipeline by Exicure Inc, H2 2019
Pipeline by F. Hoffmann-La Roche Ltd, H2 2019
Pipeline by Ionis Pharmaceuticals Inc, H2 2019
Pipeline by Novartis AG, H2 2019
Pipeline by Sarepta Therapeutics Inc, H2 2019
Pipeline by Shift Pharmaceuticals, H2 2019
Pipeline by Spotlight Innovation Inc, H2 2019
Dormant Projects, H2 2019
Discontinued Products, H2 2019
Number of Products under Development by Stage of Development, H2 2019
Number of Products under Development by Therapy Areas, H2 2019
Number of Products under Development by Indication, H2 2019
Number of Products under Development by Companies, H2 2019
Products under Development by Companies, H2 2019
Number of Products under Investigation by Universities/Institutes, H2 2019
Products under Investigation by Universities/Institutes, H2 2019
Number of Products by Stage and Mechanism of Actions, H2 2019
Number of Products by Stage and Route of Administration, H2 2019
Number of Products by Stage and Molecule Type, H2 2019
Pipeline by AurimMed Pharma Inc, H2 2019
Pipeline by AveXis Inc, H2 2019
Pipeline by Biogen Inc, H2 2019
Pipeline by Exicure Inc, H2 2019
Pipeline by F. Hoffmann-La Roche Ltd, H2 2019
Pipeline by Ionis Pharmaceuticals Inc, H2 2019
Pipeline by Novartis AG, H2 2019
Pipeline by Sarepta Therapeutics Inc, H2 2019
Pipeline by Shift Pharmaceuticals, H2 2019
Pipeline by Spotlight Innovation Inc, H2 2019
Dormant Projects, H2 2019
Discontinued Products, H2 2019
LIST OF FIGURES
Number of Products under Development by Stage of Development, H2 2019
Number of Products under Development by Therapy Areas, H2 2019
Number of Products under Development by Top 10 Indications, H2 2019
Number of Products by Stage and Mechanism of Actions, H2 2019
Number of Products by Routes of Administration, H2 2019
Number of Products by Stage and Routes of Administration, H2 2019
Number of Products by Top 10 Molecule Types, H2 2019
Number of Products by Stage and Top 10 Molecule Types, H2 2019
COMPANIES MENTIONED
AurimMed Pharma Inc
AveXis Inc
Biogen Inc
Exicure Inc
F. Hoffmann-La Roche Ltd
Ionis Pharmaceuticals Inc
Novartis AG
Sarepta Therapeutics Inc
Shift Pharmaceuticals
Spotlight Innovation Inc
Number of Products under Development by Stage of Development, H2 2019
Number of Products under Development by Therapy Areas, H2 2019
Number of Products under Development by Top 10 Indications, H2 2019
Number of Products by Stage and Mechanism of Actions, H2 2019
Number of Products by Routes of Administration, H2 2019
Number of Products by Stage and Routes of Administration, H2 2019
Number of Products by Top 10 Molecule Types, H2 2019
Number of Products by Stage and Top 10 Molecule Types, H2 2019
COMPANIES MENTIONED
AurimMed Pharma Inc
AveXis Inc
Biogen Inc
Exicure Inc
F. Hoffmann-La Roche Ltd
Ionis Pharmaceuticals Inc
Novartis AG
Sarepta Therapeutics Inc
Shift Pharmaceuticals
Spotlight Innovation Inc
