This “Barth syndrome - Pipeline Insight, 2021,” report provides comprehensive insights about 2+ companies and 2+ pipeline drugs in Barth syndrome 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.
Barth syndrome Understanding
Barth syndrome is a genetic condition that mainly affects males. Some of the symptoms of the condition include enlarged heart, low blood cell count, weakness of muscles, and fatigue. Additionally, there can be increased levels of chemicals like 3-methyglutaconic acid and 2-ethyl hydracrylic acid in the urine or blood. Barth syndrome is caused by changes (mutations) in the TAZ gene and has an X-linked inheritance pattern. Barth syndrome is mainly found in early infancy or childhood. However, in some patients, symptoms appear in adulthood. Symptoms can present differently and can vary from one person to another. Barth syndrome is caused by mutations in the TAZ gene.
"Barth syndrome - Pipeline Insight, 2021" report by the publisher outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Barth syndrome pipeline landscape is provided which includes the disease overview and Barth syndrome treatment guidelines. The assessment part of the report embraces, in depth Barth syndrome 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, Barth syndrome 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 Barth syndrome R&D. The therapies under development are focused on novel approaches to treat/ improve Barth syndrome.
This segment of the Barth syndrome 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.
Elamipretide (Stealth BioTherapeutics) is a novel tetrapeptide drug currently in development for both common and orphan diseases that involve dysfunction of the mitochondria, the cellular organelles that generates chemical energy in the form of adenosine triphosphate (ATP). At the cellular level, mitochondrial dysfunction causes loss of ATP production, increased levels of reactive oxygen species (eg, superoxide), calcium dysregulation, and, if prolonged, cell death. Elamipretide, given as systemic injection, penetrates tissues and cells and enters the mitochondria, where it reverses mitochondrial dysfunction (improved ATP production, restored mitochondrial membrane potential, normal calcium flux, and reduced superoxide generation). In August 2021, Stealth BioTherapeutics announced the submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for elamipretide for the treatment of Barth syndrome
This segment of the report provides insights about the different Barth syndrome drugs segregated based on following parameters that define the scope of the report, such as:
There are approx. 2+ key companies which are developing the therapies for Barth syndrome. The companies which have their Barth syndrome drug candidates in the most advanced stage, i.e. preregistration include, Stealth BioTherapeutics.
The drugs have been categorized under various product types like Mono, Combination and Mono/Combination.
The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Barth syndrome therapeutic drugs key players involved in developing key drugs.
The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Barth syndrome drugs.
Current Treatment Scenario and Emerging Therapies:
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Geography Covered
- Global coverage
Barth syndrome Understanding
Barth syndrome: Overview
Barth syndrome is a genetic condition that mainly affects males. Some of the symptoms of the condition include enlarged heart, low blood cell count, weakness of muscles, and fatigue. Additionally, there can be increased levels of chemicals like 3-methyglutaconic acid and 2-ethyl hydracrylic acid in the urine or blood. Barth syndrome is caused by changes (mutations) in the TAZ gene and has an X-linked inheritance pattern. Barth syndrome is mainly found in early infancy or childhood. However, in some patients, symptoms appear in adulthood. Symptoms can present differently and can vary from one person to another. Barth syndrome is caused by mutations in the TAZ gene.
"Barth syndrome - Pipeline Insight, 2021" report by the publisher outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Barth syndrome pipeline landscape is provided which includes the disease overview and Barth syndrome treatment guidelines. The assessment part of the report embraces, in depth Barth syndrome 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, Barth syndrome 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 Barth syndrome R&D. The therapies under development are focused on novel approaches to treat/ improve Barth syndrome.
Barth syndrome Emerging Drugs Chapters
This segment of the Barth syndrome 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.
Barth syndrome Emerging Drugs
Elamipretide: Stealth BioTherapeutics
Elamipretide (Stealth BioTherapeutics) is a novel tetrapeptide drug currently in development for both common and orphan diseases that involve dysfunction of the mitochondria, the cellular organelles that generates chemical energy in the form of adenosine triphosphate (ATP). At the cellular level, mitochondrial dysfunction causes loss of ATP production, increased levels of reactive oxygen species (eg, superoxide), calcium dysregulation, and, if prolonged, cell death. Elamipretide, given as systemic injection, penetrates tissues and cells and enters the mitochondria, where it reverses mitochondrial dysfunction (improved ATP production, restored mitochondrial membrane potential, normal calcium flux, and reduced superoxide generation). In August 2021, Stealth BioTherapeutics announced the submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for elamipretide for the treatment of Barth syndrome
Barth syndrome: Therapeutic Assessment
This segment of the report provides insights about the different Barth syndrome drugs segregated based on following parameters that define the scope of the report, such as:
Major Players in Barth syndrome
There are approx. 2+ key companies which are developing the therapies for Barth syndrome. The companies which have their Barth syndrome drug candidates in the most advanced stage, i.e. preregistration include, Stealth BioTherapeutics.
Phases
This report covers around 2+ products under different phases of clinical development like
- 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
Barth syndrome pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as
- Oral
- Parenteral
- intravenous
- Subcutaneous
- Topical.
- Molecule Type
Products have been categorized under various Molecule types such as
- Monoclonal Antibody
- Peptides
- Polymer
- Small molecule
- Gene therapy
- Product Type
The drugs have been categorized under various product types like Mono, Combination and Mono/Combination.
Barth syndrome: Pipeline Development Activities
The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Barth syndrome 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 Barth syndrome drugs.
Barth syndrome Report Insights
- Barth syndrome Pipeline Analysis
- Therapeutic Assessment
- Unmet Needs
- Impact of Drugs
Barth syndrome Report Assessment
- Pipeline Product Profiles
- Therapeutic Assessment
- Pipeline Assessment
- Inactive drugs assessment
- Unmet Needs
Key Questions Answered
Current Treatment Scenario and Emerging Therapies:
- How many companies are developing Barth syndrome drugs?
- How many Barth syndrome drugs are developed by each company?
- How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Barth syndrome?
- What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Barth syndrome 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 Barth syndrome and their status?
- What are the key designations that have been granted to the emerging drugs?
Key Players
- Stealth BioTherapeutics
- Healx
- Scenic Biotech
Key Products
- Elamipretide
Research programme: metabolic disorder therapeutics
Reserach programme: gene modulating therapeutics
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Table of Contents
IntroductionExecutive Summary
Barth syndrome: 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
Barth syndrome - Analytical Perspective
Late Stage Products (Preregistration)
- Comparative Analysis
Elamipretide: Stealth BioTherapeutics
- Product Description
- Research and Development
- Product Development Activities
Preclinical and Discovery Stage Products
- Comparative Analysis
Reserach programme: gene modulating therapeutics: Scenic Biotech
- Product Description
- Research and Development
- Product Development Activities
Inactive Products
- Comparative Analysis
Barth syndrome Key Companies
Barth syndrome Key Products
Barth syndrome- Unmet Needs
Barth syndrome- Market Drivers and Barriers
Barth syndrome- Future Perspectives and Conclusion
Barth syndrome Analyst Views
Barth syndrome Key Companies
AppendixList of Tables
Table 1 Total Products for Barth syndrome
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 ProductsList of Figures
Figure 1 Total Products for Barth syndrome
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
Companies Mentioned (Partial List)
A selection of companies mentioned in this report includes, but is not limited to:
- Stealth BioTherapeutics
- Healx
- Scenic Biotech
