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The publisher's latest Pharmaceutical and Healthcare disease pipeline guide Canavan Disease - Drugs In Development, 2022, provides an overview of the Canavan Disease (Central Nervous System) pipeline landscape.Speak directly to the analyst to clarify any post sales queries you may have.
Canavan disease is a gene-linked neurological disorder in which the brain degenerates into spongy tissue riddled with microscopic fluid-filled spaces. Cause includes a mutation in the gene which directs the production of the enzyme aspartoacylase (ASPA) allows the buildup of N-acetylaspartic acid (NAA) in the brain. The buildup of NAA causes damage to myelin. Symptoms include lack of motor development, feeding difficulties, abnormal muscle tone, and an abnormally large, poorly controlled head, paralysis, blindness, or hearing loss.
Report Highlights
The publisher's Pharmaceutical and Healthcare latest pipeline guide Canavan Disease - Drugs In Development, 2022, provides comprehensive information on the therapeutics under development for Canavan Disease (Central Nervous System), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The guide covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases.The Canavan Disease (Central Nervous System) pipeline guide also reviews of key players involved in therapeutic development for Canavan Disease and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies/Universities/Institutes, the molecules developed by Companies in Phase II and Unknown stages are 2 and 1 respectively. Similarly, the Universities portfolio in Phase I, Preclinical and Discovery stages comprises 1, 3 and 1 molecules, respectively.
Canavan Disease (Central Nervous System) pipeline guide 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. The guide is built using data and information sourced from the publisher'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. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.
Note: Certain content/sections in the pipeline guide may be removed or altered based on the availability and relevance of data.
Scope
- The pipeline guide provides a snapshot of the global therapeutic landscape of Canavan Disease (Central Nervous System).
- The pipeline guide reviews pipeline therapeutics for Canavan Disease (Central Nervous System) by companies and universities/research institutes based on information derived from company and industry-specific sources.
- The pipeline guide covers pipeline products based on several stages of development ranging from pre-registration till discovery and undisclosed stages.
- The pipeline guide features descriptive drug profiles for the pipeline products which comprise, product description, descriptive licensing and collaboration details, R&D brief, MoA & other developmental activities.
- The pipeline guide reviews key companies involved in Canavan Disease (Central Nervous System) therapeutics and enlists all their major and minor projects.
- The pipeline guide evaluates Canavan Disease (Central Nervous System) therapeutics based on mechanism of action (MoA), drug target, route of administration (RoA) and molecule type.
- The pipeline guide encapsulates all the dormant and discontinued pipeline projects.
- The pipeline guide reviews latest news related to pipeline therapeutics for Canavan Disease (Central Nervous System)
Reasons to Buy
- Procure strategically important competitor information, analysis, and insights to formulate effective R&D strategies.
- Recognize emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage.
- Find and recognize significant and varied types of therapeutics under development for Canavan Disease (Central Nervous System).
- Classify potential new clients or partners in the target demographic.
- Develop tactical initiatives by understanding the focus areas of leading companies.
- Plan mergers and acquisitions meritoriously by identifying key players and it's most promising pipeline therapeutics.
- Formulate corrective measures for pipeline projects by understanding Canavan Disease (Central Nervous System) pipeline depth and focus of Indication therapeutics.
- 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.
- Adjust the therapeutic portfolio by recognizing discontinued projects and understand from the know-how what drove them from pipeline.
Table of Contents
- Introduction
- Report Coverage
- Canavan Disease - Overview
- Canavan Disease - Therapeutics Development
- Pipeline Overview
- Pipeline by Companies
- Pipeline by Universities/Institutes
- Products under Development by Companies
- Products under Development by Universities/Institutes
- Canavan Disease - Therapeutics Assessment
- Assessment by Target
- Assessment by Mechanism of Action
- Assessment by Route of Administration
- Assessment by Molecule Type
- Canavan Disease - Companies Involved in Therapeutics Development
- Aspa Therapeutics Inc
- Myrtelle Inc
- Orphan Star Therapeutics LLC
- Canavan Disease - Drug Profiles
- BBP-812 - Drug Profile
- Product Description
- Mechanism Of Action
- Drugs to Target Aspartoacylase for Canavan Disease - Drug Profile
- Product Description
- Mechanism Of Action
- Gene Therapy to Activate Aspartoacylase for Canavan Disease - Drug Profile
- Product Description
- Mechanism Of Action
- Gene Therapy to Activate Aspartoacylase for Canavan Disease - Drug Profile
- Product Description
- Mechanism Of Action
- rAAV-Olig001-ASPA - Drug Profile
- Product Description
- Mechanism Of Action
- Recombinant Aspartoacylase Replacement for Canavan Disease - Drug Profile
- Product Description
- Mechanism Of Action
- Stem Cell Therapy to Activate Aspartoacylase for Canavan Disease - Drug Profile
- Product Description
- Mechanism Of Action
- Unnamed - Drug Profile
- Product Description
- Mechanism Of Action
- Canavan Disease - Dormant Projects
- Canavan Disease - Discontinued Products
- Canavan Disease - Product Development Milestones
- Featured News & Press Releases
- Oct 13, 2022: BridgeBio Pharma presents updated positive data from its BBP-812 Canavan disease gene therapy program at the 51st Annual Meeting of the Child Neurology Society
- Jun 22, 2022: BridgeBio Pharma announces early positive data for BBP-812, its investigational AAV9 gene therapy for Canavan disease
- Nov 03, 2021: BridgeBio Pharma announces dosing of first patient in phase 1/2 trial of investigational gene therapy for Canavan disease
- Oct 22, 2019: BridgeBio Pharma presents on its Central Nervous drug candidate BBP-812 at the European Society of Gene and Cell Therapy Conference
- Nov 09, 2017: New gene therapy developed with UNSW researchers targets brain disorders
- Appendix
- Methodology
- Coverage
- Secondary Research
- Primary Research
- Expert Panel Validation
- Contact Us
- Disclaimer
- Number of Products under Development for Canavan Disease, 2022
- Number of Products under Development by Companies, 2022
- Number of Products under Development by Universities/Institutes, 2022
- Products under Development by Companies, 2022
- Products under Development by Universities/Institutes, 2022
- Number of Products by Stage and Target, 2022
- Number of Products by Stage and Mechanism of Action, 2022
- Number of Products by Stage and Route of Administration, 2022
- Number of Products by Stage and Molecule Type, 2022
- Canavan Disease - Pipeline by Aspa Therapeutics Inc, 2022
- Canavan Disease - Pipeline by Myrtelle Inc, 2022
- Canavan Disease - Pipeline by Orphan Star Therapeutics LLC, 2022
- Canavan Disease - Dormant Projects, 2022
- Canavan Disease - Discontinued Products, 2022
- Number of Products under Development for Canavan Disease, 2022
- Number of Products under Development by Companies, 2022
- Number of Products under Development by Universities/Institutes, 2022
- Number of Products by Stage and Top 10 Targets, 2022
- Number of Products by Top 10 Mechanism of Actions, 2022
- Number of Products by Stage and Top 10 Mechanism of Actions, 2022
- Number of Products by Top 10 Routes of Administration, 2022
- Number of Products by Stage and Top 10 Routes of Administration, 2022
- Number of Products by Top 10 Molecule Types, 2022
- Number of Products by Stage and Top 10 Molecule Types, 2022
Companies Mentioned (Partial List)
A selection of companies mentioned in this report includes, but is not limited to:
- Aspa Therapeutics Inc
- Myrtelle Inc
- Orphan Star Therapeutics LLC
