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Tay-Sachs Disease - Pipeline Insight, 2019

Published on: Oct 2018 | From USD $1250 | Published By: DELVE INSIGHTS | Number Of Pages: 60

"Tay-Sachs Disease - Pipeline Insight, 2019" report by DelveInsight outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Tay - Sachs disease pipeline landscape is provided which includes the disease overview and Tay - Sachs disease treatment guidelines. The assessment part of the report embraces, in depth Tay - Sachs disease commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Tay-Sachs Disease collaborations, licensing, mergers and acquisition, funding, designations and other product related details.
Tay - Sachs disease Understanding
According to National Organization for Rare Disorders (NORD), Tay-Sachs disease is a rare, neurodegenerative disorder in which deficiency of an enzyme hexosaminidase A results in excessive accumulation of certain fats (lipids) known as gangliosides in the brain and nerve cells. This abnormal accumulation of gangliosides leads to progressive dysfunction of the central nervous system. This disorder is categorized as a lysosomal storage disease. Tay-Sachs disease is broken down into the classic or infantile form, the juvenile form, and the adult or late-onset form. In individuals with infantile Tay-Sachs disease, symptoms typically first appear between three and five months of age. In individuals with the late-onset form, symptoms may become apparent anytime from adolescence through the mid-30s.
Tay - Sachs disease Pipeline Development Activities
The report provides insights into different therapeutic candidates in discovery and preclinical, phase 1, phase 2, and phase 3 stage. Drugs under development as a monotherapy or combination therapy are also included. It also analyses key players involved in Tay - Sachs disease targeted therapeutics development with respective active and inactive (dormant or discontinued) projects with the appropriate reasons if available. Tay - Sachs disease pipeline report covers 6+ companies. Some of the key players include IntraBio Inc (IB1001-201), Castle Creek Pharmaceuticals (CCP 010) etc.
The report is built using data and information traced from the researcher's proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations and featured press releases from company/university web sites and industry-specific third party sources, etc.
Tay - Sachs disease Analytical Perspective by DelveInsight
• In-depth Tay-Sachs Disease Commercial Assessment of products
This report provides an in-depth Commercial Assessment of therapeutic drugs have been included which comprises of collaborations, Licensing, Acquisition –Deal Value Trends. The sub-segmentation is described in the report which includes Company-Company Collaborations (Licensing / Partnering), Company-Academia Collaborations, and Acquisition analysis in both Graphical and tabulated form.
• Tay-Sachs Disease Clinical Assessment of products
The report comprises of comparative clinical assessment of products by development stage, product type, route of administration, molecule type, and MOA type across this indication.
Scope of the report
• The Tay - Sachs disease report provides an overview of therapeutic pipeline activity for Tay - Sachs disease across the complete product development cycle including all clinical and non-clinical stages
• It comprises of detailed profiles of Tay - Sachs disease therapeutic products with key coverage of developmental activities including technology, collaborations, licensing, mergers and acquisition, funding, designations and other product related details
• Detailed Tay - Sachs disease Research and Development progress and trial details, results wherever available, are also included in the pipeline study
• Therapeutic assessment of the active pipeline products by development stage, product type, route of administration, molecule type, and MOA type
• Coverage of dormant and discontinued pipeline projects along with the reasons if available across Tay - Sachs disease
Reasons to Buy
• Establish a comprehensive understanding of the current pipeline scenario across Tay - Sachs disease to formulate effective R&D strategies
• Assess challenges and opportunities that influence Tay - Sachs disease R&D
• Develop strategic initiatives by understanding the focus areas of leading companies.
• Gather impartial perspective of strategies of the emerging competitors having potentially lucrative portfolio in this space and create effective counter strategies to gain competitive advantage
• Get in detail information of each product with updated information on each project along with key milestones
• Devise Tay - Sachs disease in licensing and out licensing strategies by identifying prospective partners with progressing projects for Tay - Sachs disease to enhance and expand business potential and scope
• Our extensive domain knowledge on therapy areas support the clients in decision-making process regarding their therapeutic portfolio by identifying the reason behind the inactive or discontinued drugs

1. Report Introduction
2. Tay-Sachs Disease (GM2 gangliosidosis, type 1, HexA deficiency, Hexosaminidase A deficiency, Hexosaminidase alpha-subunit deficiency (variant B))
2.1. Tay-Sachs Disease Disease Overview
2.2. Tay-Sachs Disease History
2.3. Tay-Sachs Disease Symptoms
2.4. Tay-Sachs Disease Causes
2.5. Tay-Sachs Disease Pathophysiology
2.6. Tay-Sachs Disease Diagnosis
2.6.1. Diagnostic Guidelines
3. Tay-Sachs Disease Current Treatment Patterns
3.1. Treatment Guidelines
4. Tay-Sachs Disease- DelveInsightÂ’s Analytical Perspective
4.1. In-depth Commercial Assessment
4.1.1. Tay-Sachs Disease companies collaborations, Licensing, Acquisition –Deal Value Trends
4.1.1.1. Assessment Summary
4.1.2. Tay-Sachs Disease Collaboration Deals
4.1.2.1. Company-Company Collaborations (Licensing / Partnering) Analysis
4.1.2.2. Tay-Sachs Disease Acquisition Analysis
4.2. Clinical Assessment of Pipeline Drugs
4.2.1. Assessment by Phase of Development
4.2.2. Assessment by Product Type (Mono / Combination)
4.2.2.1. Assessment by Stage and Product Type
4.2.3. Assessment by Route of Administration
4.2.3.1. Assessment by Stage and Route of Administration
4.2.4. Assessment by Molecule Type
4.2.4.1. Assessment by Stage and Molecule Type
4.2.5. Assessment by MOA
4.2.5.1. Assessment by Stage and MOA
5. Tay-Sachs Disease Pipeline Therapeutics
5.1. Late Stage Products (Phase-III)
5.1.1. Comparative Analysis
5.2. Mid Stage Products (Phase-II)
5.2.1. Comparative Analysis
5.3. Early Stage Products (Phase-I)
5.3.1. Comparative Analysis
5.4. Pre-clinical and Discovery Stage Products
5.4.1. Comparative Analysis
5.5. Inactive Products
6. Tay-Sachs Disease-Products Analysis
6.1. Product Profiles
6.1.1. IB1001-201- IntraBio
6.1.1.1. Product Description
6.1.1.1.1. Product Overview
6.1.1.1.2. Mechanism of Action
6.1.1.2. Research and Development
6.1.1.2.1. Clinical Studies
6.1.1.2.1.1. Detailed Study Description
6.1.1.2.1.2. Study Results
6.1.1.2.1.3. Clinical Trials: Tabular View
6.1.1.3. Product Development Activities
6.1.1.3.1. Tabulated Product Summary
6.1.1.3.1.1. General Description Table
6.1.2. CCP 010- Castle Creek Pharmaceuticals
6.1.2.1. Product Description
6.1.2.1.1. Product Overview
6.1.2.1.2. Mechanism of Action
6.1.2.2. Research and Development
6.1.2.2.1. Clinical Studies
6.1.2.2.1.1. Detailed Study Description
6.1.2.2.1.2. Study Results
6.1.2.2.1.3. Clinical Trials: Tabular View
6.1.2.3. Product Development Activities
6.1.2.3.1. Tabulated Product Summary
6.1.2.3.1.1. General Description Table
To be continued in the reportÂ…Â…Â…Â…Â…Â…Â…Â…Â…Â…
7. Recent Technologies
8. Tay-Sachs Disease Key Companies
8.1. Axovant Gene Therapies
8.2. Actelion Pharmaceuticals
8.3. Castle Creek Pharmaceuticals
8.4. Avigen
8.5. Gene Therapy Research Institution
8.6. Oxford Glycobiology Institute
9. Tay-Sachs Disease Key Products
9.1. AXO AAV GM2
9.2. Miglustat
9.3. CCP 010
9.4. Lysosomal storage disease gene therapy
9.5. Research programme: Tay-Sachs disease gene therapy
9.6. Acetylleucine
10. Dormant and Discontinued Products
10.1. Dormant Products
10.1.1. Reasons for being dormant
10.2. Discontinued Products
10.2.1. Reasons for the discontinuation
11. Tay-Sachs Disease- Unmet Needs
12. Tay-Sachs Disease- Future Perspectives
13. Appendix
14. Report Methodology
14.1. Secondary Research
14.2. Expert Panel Validation

Table 1. Diagnostic Guidelines
Table 2. Treatment Guidelines
Table 3. Assessment Summary
Table 4. Company-Company Collaborations (Licensing / Partnering) Analysis
Table 5. Tay-Sachs Disease Acquisition Analysis
Table 6. Assessment by Phase of Development
Table 7. Assessment by Product Type (Mono / Combination)
Table 8. Assessment by Stage and Product Type
Table 9. Assessment by Route of Administration
Table 10. Assessment by Stage and Route of Administration
Table 11. Assessment by Molecule Type
Table 12. Assessment by Stage and Molecule Type
Table 13. Assessment by MOA
Table 14. Assessment by Stage and MOA
Table 15. Late Stage Products (Phase-III)
Table 16. Mid Stage Products (Phase-II)
Table 17. Early Stage Products (Phase-I)
Table 18. Pre-clinical and Discovery Stage Products
Table 19. Inactive Products
Table 20. Dormant Products
Table 21. Discontinued Products


Figure 1. Disease Overview
Figure 2. History
Figure 3. Symptoms
Figure 4. Causes
Figure 5. Pathophysiology
Figure 6. Diagnostic Guidelines
Figure 7. Treatment Guidelines
Figure 8. Tay-Sachs Disease companies collaborations, Licensing, Acquisition –Deal Value Trends
Figure 9. Company-Company Collaborations (Licensing / Partnering) Analysis
Figure 10. Tay-Sachs Disease Acquisition Analysis
Figure 11. Assessment by Phase of Development
Figure 12. Assessment by Product Type (Mono / Combination)
Figure 13. Assessment by Stage and Product Type
Figure 14. Assessment by Route of Administration
Figure 15. Assessment by Stage and Route of Administration
Figure 16. Assessment by Molecule Type
Figure 17. Assessment by Stage and Molecule Type
Figure 18. Assessment by MOA
Figure 19. Assessment by Stage and MOA
Figure 20. Late Stage Products (Phase-III)
Figure 21. Mid Stage Products (Phase-II)
Figure 22. Early Stage Products (Phase-I)
Figure 23. Pre-clinical and Discovery Stage Products
Figure 24. Inactive Products
Figure 25. Dormant Products
Figure 26. Discontinued Products
Figure 27. Unmet Needs

SECONDARY RESEARCH
Secondary Research Information is collected from a number of publicly available as well as paid databases. Public sources involve publications by different associations and governments, annual reports and statements of companies, white papers and research publications by recognized industry experts and renowned academia etc. Paid data sources include third party authentic industry databases.

PRIMARY RESEARCH
Once data collection is done through secondary research, primary interviews are conducted with different stakeholders across the value chain like manufacturers, distributors, ingredient/input suppliers, end customers and other key opinion leaders of the industry. Primary research is used both to validate the data points obtained from secondary research and to fill in the data gaps after secondary research.

MARKET ENGINEERING
The market engineering phase involves analyzing the data collected, market breakdown and forecasting. Macroeconomic indicators and bottom-up and top-down approaches are used to arrive at a complete set of data points that give way to valuable qualitative and quantitative insights. Each data point is verified by the process of data triangulation to validate the numbers and arrive at close estimates.

EXPERT VALIDATION
The market engineered data is verified and validated by a number of experts, both in-house and external.

REPORT WRITING/ PRESENTATION
After the data is curated by the mentioned highly sophisticated process, the analysts begin to write the report. Garnering insights from data and forecasts, insights are drawn to visualize the entire ecosystem in a single report.

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