Leigh disease - Pipeline Insight, 2021
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DelveInsight’s, “Leigh disease - Pipeline Insight, 2021,” report provides comprehensive insights about 3+ companies and 3+ pipeline drugs in Leigh disease pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.
Geography Covered
Leigh disease: Overview
Leigh syndrome is a rare genetic neurometabolic disorder. It is characterized by the degeneration of the central nervous system (i.e., brain, spinal cord, and optic nerve). The symptoms of Leigh syndrome usually begin between the ages of three months and two years, but some patients do not exhibit signs and symptoms until several years later. Symptoms are associated with progressive neurological deterioration and may include loss of previously acquired motor skills, loss of appetite, vomiting, irritability, and/or seizure activity. As Leigh syndrome progresses, symptoms may also include generalized weakness, lack of muscle tone (hypotonia), and episodes of lactic acidosis, which may lead to impairment of respiratory and kidney function.
'Leigh disease - Pipeline Insight, 2021' report by DelveInsight outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Leigh disease pipeline landscape is provided which includes the disease overview and Leigh disease treatment guidelines. The assessment part of the report embraces, in depth Leigh 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, Leigh disease collaborations, licensing, mergers and acquisition, funding, designations and other product related details.
Report Highlights
This segment of the Leigh disease report encloses its detailed analysis of various drugs in different stages of clinical development, including phase II, I, preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.
Leigh disease Emerging Drugs
Further product details are provided in the report
Leigh disease: Therapeutic Assessment
This segment of the report provides insights about the different Leigh disease drugs segregated based on following parameters that define the scope of the report, such as:
Leigh disease: Pipeline Development Activities
The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Leigh disease therapeutic drugs key players involved in developing key drugs.
Pipeline Development Activities
The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Leigh disease drugs.
Leigh disease Report Insights
Current Treatment Scenario and Emerging Therapies:
DelveInsight’s, “Leigh disease - Pipeline Insight, 2021,” report provides comprehensive insights about 3+ companies and 3+ pipeline drugs in Leigh disease pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.
Geography Covered
- Global coverage
Leigh disease: Overview
Leigh syndrome is a rare genetic neurometabolic disorder. It is characterized by the degeneration of the central nervous system (i.e., brain, spinal cord, and optic nerve). The symptoms of Leigh syndrome usually begin between the ages of three months and two years, but some patients do not exhibit signs and symptoms until several years later. Symptoms are associated with progressive neurological deterioration and may include loss of previously acquired motor skills, loss of appetite, vomiting, irritability, and/or seizure activity. As Leigh syndrome progresses, symptoms may also include generalized weakness, lack of muscle tone (hypotonia), and episodes of lactic acidosis, which may lead to impairment of respiratory and kidney function.
'Leigh disease - Pipeline Insight, 2021' report by DelveInsight outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Leigh disease pipeline landscape is provided which includes the disease overview and Leigh disease treatment guidelines. The assessment part of the report embraces, in depth Leigh 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, Leigh disease collaborations, licensing, mergers and acquisition, funding, designations and other product related details.
Report Highlights
- The companies and academics are working to assess challenges and seek opportunities that could influence Leigh disease R&D. The therapies under development are focused on novel approaches to treat/improve Leigh disease.
This segment of the Leigh disease report encloses its detailed analysis of various drugs in different stages of clinical development, including phase II, I, preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.
Leigh disease Emerging Drugs
- Vatiquinone (EPI-743): PTC Therapeutics
- ABI-009: Khondrion
Further product details are provided in the report
Leigh disease: Therapeutic Assessment
This segment of the report provides insights about the different Leigh disease drugs segregated based on following parameters that define the scope of the report, such as:
- Major Players in Leigh disease
- Phases
- Late stage products (Phase III)
- Mid-stage products (Phase II)
- Early-stage product (Phase I) along with the details of
- Pre-clinical and Discovery stage candidates
- Discontinued & Inactive candidates
- Route of Administration
- Oral
- Parenteral
- intravitreal
- Subretinal
- Topical.
- Molecule Type
- Monoclonal Antibody
- Peptides
- Polymer
- Small molecule
- Gene therapy
- Product Type
Leigh disease: Pipeline Development Activities
The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Leigh disease therapeutic drugs key players involved in developing key drugs.
Pipeline Development Activities
The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Leigh disease drugs.
Leigh disease Report Insights
- Leigh disease Pipeline Analysis
- Therapeutic Assessment
- Unmet Needs
- Impact of Drugs
- Pipeline Product Profiles
- Therapeutic Assessment
- Pipeline Assessment
- Inactive drugs assessment
- Unmet Needs
Current Treatment Scenario and Emerging Therapies:
- How many companies are developing Leigh disease drugs?
- How many Leigh disease drugs are developed by each company?
- How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Leigh disease?
- What are the key collaborations (Industry–Industry, Industry–Academia), Mergers and acquisitions, licensing activities related to the Leigh disease therapeutics?
- What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies?
- What are the clinical studies going on for Leigh disease and their status?
- What are the key designations that have been granted to the emerging drugs?
- PTC Therapeutics
- Aadi, LLC
- Khondrion
- Mission therapeutics
- Taysha Gene Therapies
- Vatiquinone (EPI-743)
- ABI-009
- Sonlicromanol
- USP30 MITOCHONDRIAL DISEASE PROGRAM
- TSHA-104
Introduction
Executive Summary
Leigh disease: Overview
Causes
Mechanism of Action
Signs and Symptoms
Diagnosis
Disease Management
Pipeline Therapeutics
Comparative Analysis
Therapeutic Assessment
Assessment by Product Type
Assessment by Stage and Product Type
Assessment by Route of Administration
Assessment by Stage and Route of Administration
Assessment by Molecule Type
Assessment by Stage and Molecule Type
Leigh disease – DelveInsight’s Analytical Perspective
In-depth Commercial Assessment
Leigh disease companies’ collaborations, Licensing, Acquisition -Deal Value Trends
Leigh disease Collaboration Deals
Company-Company Collaborations (Licensing / Partnering) Analysis
Company-University Collaborations (Licensing / Partnering) Analysis
Late Stage Products (Phase III)
Comparative Analysis
Vatiquinone (EPI-743): PTC Therapeutics
Product Description
Research and Development
Product Development Activities
Drug profiles in the detailed report
Mid Stage Products (Phase II)
Comparative Analysis
ABI-009: Aadi, LLC
Product Description
Research and Development
Product Development Activities
Drug profiles in the detailed report
Inactive Products
Comparative Analysis
Leigh disease Key Companies
Leigh disease Key Products
Leigh disease- Unmet Needs
Leigh disease- Market Drivers and Barriers
Leigh disease- Future Perspectives and Conclusion
Leigh disease Analyst Views
Leigh disease Key Companies
Appendix
Executive Summary
Leigh disease: Overview
Causes
Mechanism of Action
Signs and Symptoms
Diagnosis
Disease Management
Pipeline Therapeutics
Comparative Analysis
Therapeutic Assessment
Assessment by Product Type
Assessment by Stage and Product Type
Assessment by Route of Administration
Assessment by Stage and Route of Administration
Assessment by Molecule Type
Assessment by Stage and Molecule Type
Leigh disease – DelveInsight’s Analytical Perspective
In-depth Commercial Assessment
Leigh disease companies’ collaborations, Licensing, Acquisition -Deal Value Trends
Leigh disease Collaboration Deals
Company-Company Collaborations (Licensing / Partnering) Analysis
Company-University Collaborations (Licensing / Partnering) Analysis
Late Stage Products (Phase III)
Comparative Analysis
Vatiquinone (EPI-743): PTC Therapeutics
Product Description
Research and Development
Product Development Activities
Drug profiles in the detailed report
Mid Stage Products (Phase II)
Comparative Analysis
ABI-009: Aadi, LLC
Product Description
Research and Development
Product Development Activities
Drug profiles in the detailed report
Inactive Products
Comparative Analysis
Leigh disease Key Companies
Leigh disease Key Products
Leigh disease- Unmet Needs
Leigh disease- Market Drivers and Barriers
Leigh disease- Future Perspectives and Conclusion
Leigh disease Analyst Views
Leigh disease Key Companies
Appendix
LIST OF TABLES
Table 1 Total Products for Leigh disease
Table 2 Late Stage Products
Table 3 Mid Stage Products
Table 4 Early Stage Products
Table 5 Pre-clinical & Discovery Stage Products
Table 6 Assessment by Product Type
Table 7 Assessment by Stage and Product Type
Table 8 Assessment by Route of Administration
Table 9 Assessment by Stage and Route of Administration
Table 10 Assessment by Molecule Type
Table 11 Assessment by Stage and Molecule Type
Table 12 Inactive Products
Table 1 Total Products for Leigh disease
Table 2 Late Stage Products
Table 3 Mid Stage Products
Table 4 Early Stage Products
Table 5 Pre-clinical & Discovery Stage Products
Table 6 Assessment by Product Type
Table 7 Assessment by Stage and Product Type
Table 8 Assessment by Route of Administration
Table 9 Assessment by Stage and Route of Administration
Table 10 Assessment by Molecule Type
Table 11 Assessment by Stage and Molecule Type
Table 12 Inactive Products
LIST OF FIGURES
Figure 1 Total Products for Leigh disease
Figure 2 Late Stage Products
Figure 3 Mid Stage Products
Figure 4 Early Stage Products
Figure 5 Preclinical and Discovery Stage Products
Figure 6 Assessment by Product Type
Figure 7 Assessment by Stage and Product Type
Figure 8 Assessment by Route of Administration
Figure 9 Assessment by Stage and Route of Administration
Figure 10 Assessment by Molecule Type
Figure 11 Assessment by Stage and Molecule Type
Figure 12 Inactive Products
Figure 1 Total Products for Leigh disease
Figure 2 Late Stage Products
Figure 3 Mid Stage Products
Figure 4 Early Stage Products
Figure 5 Preclinical and Discovery Stage Products
Figure 6 Assessment by Product Type
Figure 7 Assessment by Stage and Product Type
Figure 8 Assessment by Route of Administration
Figure 9 Assessment by Stage and Route of Administration
Figure 10 Assessment by Molecule Type
Figure 11 Assessment by Stage and Molecule Type
Figure 12 Inactive Products
