
Primary Mitochondrial Myopathies - Pipeline Insight, 2024
Description
Primary Mitochondrial Myopathies - Pipeline Insight, 2024
DelveInsight’s, “Primary Mitochondrial Myopathies - Pipeline Insight, 2024,” report provides comprehensive insights about 3+ companies and 3+ pipeline drugs in Primary Mitochondrial Myopathies 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
Primary Mitochondrial Myopathies: Overview
Primary mitochondrial myopathies (PMM) are a group of disorders that are associated with changes in genetic material (e.g. depletions, deletions, or mutations) found within the DNA of mitochondria (mtDNA) or with genes outside the mitochondria (nuclear DNA), affecting predominantly the skeletal muscle. Mitochondria, found by the hundreds within every cell of the body, regulate the production of cellular energy and carry the genetic blueprints for this process within their own unique DNA (mtDNA). These disorders often hamper the ability of affected cells to break down food and oxygen and produce energy. Mitochondria provide more than 90% of the energy used by the body’s tissues; mitochondrial disorders are characterized by a lack of sufficient energy for cells of the body to function properly. High-energy tissues like muscle, brain, or heart tissue are most likely to be affected by mitochondrial disorders. In most mitochondrial disorders, abnormally high numbers of defective mitochondria are present in the cells of the body. Mitochondrial diseases often affect more than one organ system of the body. Most mitochondrial diseases affect the muscles (myopathy). Sometimes, muscle disease is the only or predominant sign of a mitochondrial disorder, thus defined as PMM. There are no disease-modifying treatments for PMM; treatment is aimed to improving or resolving specific symptoms.
""Primary Mitochondrial Myopathies - Pipeline Insight, 2024"" report by DelveInsight outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Primary Mitochondrial Myopathies pipeline landscape is provided which includes the disease overview and Primary Mitochondrial Myopathies treatment guidelines. The assessment part of the report embraces, in depth Primary Mitochondrial Myopathies 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, Primary Mitochondrial Myopathies collaborations, licensing, mergers and acquisition, funding, designations and other product related details.
Report Highlights
- A better understanding of disease pathogenesis contributing to the development of novel therapeutics for Primary Mitochondrial Myopathies.
- In the coming years, the Primary Mitochondrial Myopathies market is set to change due to the rising awareness of the disease, and incremental healthcare spending across the world; which would expand the size of the market to enable the drug manufacturers to penetrate more into the market.
- The companies and academics that are working to assess challenges and seek opportunities that could influence Primary Mitochondrial Myopathies R&D. The therapies under development are focused on novel approaches to treat/improve the disease condition.
- A detailed portfolio of major pharma players who are involved in fueling the Primary Mitochondrial Myopathies treatment market. Several potential therapies for Primary Mitochondrial Myopathies are under investigation. With the expected launch of these emerging therapies, it is expected that there will be a significant impact on the Primary Mitochondrial Myopathies market size in the coming years.
- Our in-depth analysis of the pipeline assets (in early-stage, mid-stage and late stage of development for the treatment of Primary Mitochondrial Myopathies) includes therapeutic assessment and comparative analysis. This will support the clients in the decision-making process regarding their therapeutic portfolio by identifying the overall scenario of the research and development activities.
This segment of the Primary Mitochondrial Myopathies 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.
Primary Mitochondrial Myopathies Emerging Drugs
- REN001: Reneo Pharma Ltd
- Elamipretide: Stealth BioTherapeutics
Further product details are provided in the report……..
Primary Mitochondrial Myopathies: Therapeutic Assessment
This segment of the report provides insights about the different Primary Mitochondrial Myopathies drugs segregated based on following parameters that define the scope of the report, such as:
- Major Players in Primary Mitochondrial Myopathies
- 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
- Inhalation
- Inhalation/Intravenous/Oral
- Intranasal
- Intravenous
- Intravenous/ Subcutaneous
- NA
- Oral
- Oral/intranasal/subcutaneous
- Parenteral
- Subcutaneous
- Molecule Type
- Antibody
- Antisense oligonucleotides
- Immunotherapy
- Monoclonal antibody
- Peptides
- Protein
- Recombinant protein
- Small molecule
- Stem Cell
- Vaccine
- Product Type
Primary Mitochondrial Myopathies: Pipeline Development Activities
The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Primary Mitochondrial Myopathies 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 Primary Mitochondrial Myopathies drugs.
Primary Mitochondrial Myopathies Report Insights
- Primary Mitochondrial Myopathies 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 Primary Mitochondrial Myopathies drugs?
- How many Primary Mitochondrial Myopathies drugs are developed by each company?
- How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Primary Mitochondrial Myopathies?
- What are the key collaborations (Industry–Industry, Industry–Academia), Mergers and acquisitions, licensing activities related to the Primary Mitochondrial Myopathies 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 Primary Mitochondrial Myopathies and their status?
- What are the key designations that have been granted to the emerging drugs?
- Reneo Pharma Ltd
- Stealth BioTherapeutics
- Astellas Pharma Inc
- Abliva AB
- REN001
- Elamipretide
- Bocidelpar
- KL 1333
Table of Contents
60 Pages
- Introduction
- Executive Summary
- Primary Mitochondrial Myopathies: 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
- Primary Mitochondrial Myopathies – DelveInsight’s Analytical Perspective
- Late Stage Products (Phase III)
- Comparative Analysis
- Elamipretide: Stealth BioTherapeutics
- Product Description
- Research and Development
- Product Development Activities
- Drug profiles in the detailed report…..
- Mid Stage Products (Phase II/III)
- Comparative Analysis
- REN 001 - Reneo Pharmaceuticals
- Product Description
- Research and Development
- Product Development Activities
- Drug profiles in the detailed report…..
- Early Stage Products (Phase I)
- Comparative Analysis
- KL 1333: Abliva AB
- Product Description
- Research and Development
- Product Development Activities
- Drug profiles in the detailed report…..
- Preclinical and Discovery Stage Products
- Comparative Analysis
- Drug name: Company name
- Product Description
- Research and Development
- Product Development Activities
- Drug profiles in the detailed report…..
- Inactive Products
- Comparative Analysis
- Primary Mitochondrial Myopathies Key Companies
- Primary Mitochondrial Myopathies Key Products
- Primary Mitochondrial Myopathies- Unmet Needs
- Primary Mitochondrial Myopathies- Market Drivers and Barriers
- Primary Mitochondrial Myopathies- Future Perspectives and Conclusion
- Primary Mitochondrial Myopathies Analyst Views
- Primary Mitochondrial Myopathies Key Companies
- Appendix
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