Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) Drugs in Development by Stages, Target, MoA, RoA, Molecule Type and Key Players, 2022 Update

December 2022 | 83 pages | ID: S77749DECA3DEN
Global Markets Direct

Abstracts

Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) Drugs in Development by Stages, Target, MoA, RoA, Molecule Type and Key Players, 2022 Update

SUMMARY

Survival Motor Neuron Protein (Component Of Gems 1 or Gemin 1 or SMN1 or SMN2) pipeline Target constitutes close to 20 molecules. Out of which approximately 13 molecules are developed by companies and remaining by the universities/institutes. The latest report Survival Motor Neuron Protein - Drugs In Development, 2022, 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 Pre-Registration, Phase III, Phase II, Phase I, Preclinical and Discovery stages are 1, 1, 1, 1, 7 and 2 respectively. Similarly, the universities portfolio in Phase II, Preclinical and Discovery stages comprises 1, 4 and 2 molecules, respectively. Report covers products from therapy areas Central Nervous System which include indications Spinal Muscular Atrophy (SMA) and Huntington Disease.

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

Contents

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
Amogen Pharma Pvt Ltd
Beijing GeneCradle Technology Co Ltd
Biocad
CANbridge Life Sciences Ltd
F. Hoffmann-La Roche Ltd
Huida (Shanghai) Biotechnology Co Ltd
Novartis AG
Novartis Gene Therapies
Sarepta Therapeutics Inc
Shift Pharmaceuticals
Spotlight Innovation Inc (Inactive)
Suzhou GenAssist Therapeutics Co Ltd
Voyager Therapeutics 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
ANB-4 - Drug Profile
Product Description
Mechanism Of Action
Antisense Oligonucleotide to Activate SMN2 for Spinal Muscular Atrophy - Drug Profile
Product Description
Mechanism Of Action
History of Events
branaplam hydrochloride - Drug Profile
Product Description
Mechanism Of Action
History of Events
E-1V111 - Drug Profile
Product Description
Mechanism Of Action
History of Events
GC-101 - Drug Profile
Product Description
Mechanism Of Action
GEN-8010 - Drug Profile
Product Description
Mechanism Of Action
Gene Therapy to Activate SMN1 for Spinal Muscular Atrophy - Drug Profile
Product Description
Mechanism Of Action
HG-001 - Drug Profile
Product Description
Mechanism Of Action
onasemnogene abeparvovec - Drug Profile
Product Description
Mechanism Of Action
History of Events
onasemnogene abeparvovec - Drug Profile
Product Description
Mechanism Of Action
PMO-25 - Drug Profile
Product Description
Mechanism Of Action
History of Events
Resagen - Drug Profile
Product Description
Mechanism Of Action
History of Events
risdiplam - Drug Profile
Product Description
Mechanism Of Action
History of Events
Second-Generation scAAV9 Gene Therapy - Drug Profile
Product Description
Mechanism Of Action
History of Events
Small Molecules 2 to Activate SMN2 for Spinal Muscular Atrophy - Drug Profile
Product Description
Mechanism Of Action
Small Molecules for Spinal Muscular Atrophy - Drug Profile
Product Description
Mechanism Of Action
Small Molecules to Activate SMN2 for Spinal Muscular Atrophy - Drug Profile
Product Description
Mechanism Of Action
Spinal Muscular Atrophy - Drug Profile
Product Description
Mechanism Of Action
STL-182 - Drug Profile
Product Description
Mechanism Of Action
History of Events
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
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer

List Of Tables

LIST OF TABLES

Number of Products under Development by Stage of Development, 2022
Number of Products under Development by Therapy Areas, 2022
Number of Products under Development by Indication, 2022
Number of Products under Development by Companies, 2022
Products under Development by Companies, 2022
Number of Products under Investigation by Universities/Institutes, 2022
Products under Investigation by Universities/Institutes, 2022
Number of Products by Stage and Mechanism of Actions, 2022
Number of Products by Stage and Route of Administration, 2022
Number of Products by Stage and Molecule Type, 2022
Pipeline by Amogen Pharma Pvt Ltd, 2022
Pipeline by Beijing GeneCradle Technology Co Ltd, 2022
Pipeline by Biocad, 2022
Pipeline by CANbridge Life Sciences Ltd, 2022
Pipeline by F. Hoffmann-La Roche Ltd, 2022
Pipeline by Huida (Shanghai) Biotechnology Co Ltd, 2022
Pipeline by Novartis AG, 2022
Pipeline by Novartis Gene Therapies, 2022
Pipeline by Sarepta Therapeutics Inc, 2022
Pipeline by Shift Pharmaceuticals, 2022
Pipeline by Spotlight Innovation Inc (Inactive), 2022
Pipeline by Suzhou GenAssist Therapeutics Co Ltd, 2022
Pipeline by Voyager Therapeutics Inc, 2022
Dormant Projects, 2022
Discontinued Products, 2022

List Of Figures

LIST OF FIGURES

Number of Products under Development by Stage of Development, 2022
Number of Products under Development by Top 10 Indications, 2022
Number of Products by Stage and Mechanism of Actions, 2022
Number of Products by Routes of Administration, 2022
Number of Products by Stage and Routes of Administration, 2022
Number of Products by Molecule Types, 2022
Number of Products by Stage and Molecule Types, 2022