
Methylmalonic acidaemia - Pipeline Insight, 2025
Description
DelveInsight’s, “Methylmalonic acidaemia - Pipeline Insight, 2025,” report provides comprehensive insights about 5+ companies and 5+ pipeline drugs in Methylmalonic acidaemia 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
Methylmalonic acidaemia: Overview
Methylmalonic acidemia refers to a group of inherited conditions in which the body can’t breakdown certain parts of proteins and fats. This leads to a build-up of toxic substances and bouts of serious illness called decompensation events or metabolic crises. Symptoms of a decompensation event include poor feeding, vomiting, trouble breathing, and lack of energy (lethargy). These can occur at different ages and can range from mild to severe. Methylmalonic acidemia is caused by changes in several different genes and is inherited in an autosomal recessive fashion. Treatment includes aggressive management of decompensation events, a low-protein diet, certain medications, antibiotics and, in some cases, liver and kidney transplantation. Some subtypes of methylmalonic acidemia respond to vitamin B12. Long-term complications can include growth delay, intellectual disability, kidney disease, and pancreatitis. Methylmalonic acidemia can be isolated or may occur along with another condition called homocystinuria.
""Methylmalonic acidaemia - Pipeline Insight, 2025"" report by DelveInsight outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Methylmalonic acidaemia pipeline landscape is provided which includes the disease overview and Methylmalonic acidaemia treatment guidelines. The assessment part of the report embraces, in depth Methylmalonic acidaemia 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, Methylmalonic acidaemia collaborations, licensing, mergers and acquisition, funding, designations and other product related details.
Report Highlights
This segment of the Methylmalonic acidaemia 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.
Methylmalonic acidaemia Emerging Drugs
Further product details are provided in the report……..
Methylmalonic acidaemia: Therapeutic Assessment
This segment of the report provides insights about the different Methylmalonic acidaemia drugs segregated based on following parameters that define the scope of the report, such as:
Methylmalonic acidaemia: Pipeline Development Activities
The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Methylmalonic acidaemia 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 Methylmalonic acidaemia drugs.
Methylmalonic acidaemia Report Insights
Current Treatment Scenario and Emerging Therapies:
Geography Covered
- Global coverage
Methylmalonic acidaemia: Overview
Methylmalonic acidemia refers to a group of inherited conditions in which the body can’t breakdown certain parts of proteins and fats. This leads to a build-up of toxic substances and bouts of serious illness called decompensation events or metabolic crises. Symptoms of a decompensation event include poor feeding, vomiting, trouble breathing, and lack of energy (lethargy). These can occur at different ages and can range from mild to severe. Methylmalonic acidemia is caused by changes in several different genes and is inherited in an autosomal recessive fashion. Treatment includes aggressive management of decompensation events, a low-protein diet, certain medications, antibiotics and, in some cases, liver and kidney transplantation. Some subtypes of methylmalonic acidemia respond to vitamin B12. Long-term complications can include growth delay, intellectual disability, kidney disease, and pancreatitis. Methylmalonic acidemia can be isolated or may occur along with another condition called homocystinuria.
""Methylmalonic acidaemia - Pipeline Insight, 2025"" report by DelveInsight outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Methylmalonic acidaemia pipeline landscape is provided which includes the disease overview and Methylmalonic acidaemia treatment guidelines. The assessment part of the report embraces, in depth Methylmalonic acidaemia 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, Methylmalonic acidaemia 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 Methylmalonic acidaemia R&D. The therapies under development are focused on novel approaches to treat/improve Methylmalonic acidaemia.
This segment of the Methylmalonic acidaemia 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.
Methylmalonic acidaemia Emerging Drugs
- HST5040: HemoShear Therapeutics
- LB-001: LogicBio Therapeutics
Further product details are provided in the report……..
Methylmalonic acidaemia: Therapeutic Assessment
This segment of the report provides insights about the different Methylmalonic acidaemia drugs segregated based on following parameters that define the scope of the report, such as:
- Major Players in Methylmalonic acidaemia
- 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
- intravenous
- Subcutaneous
- Topical.
- Molecule Type
- Monoclonal Antibody
- Peptides
- Polymer
- Small molecule
- Gene therapy
- Product Type
Methylmalonic acidaemia: Pipeline Development Activities
The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Methylmalonic acidaemia 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 Methylmalonic acidaemia drugs.
Methylmalonic acidaemia Report Insights
- Methylmalonic acidaemia 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 Methylmalonic acidaemia drugs?
- How many Methylmalonic acidaemia drugs are developed by each company?
- How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Methylmalonic acidaemia?
- What are the key collaborations (Industry–Industry, Industry–Academia), Mergers and acquisitions, licensing activities related to the Methylmalonic acidaemia 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 Methylmalonic acidaemia and their status?
- What are the key designations that have been granted to the emerging drugs?
- HemoShear Therapeutics
- LogicBio Therapeutics
- Moderna Therapeutics
- Selecta Biosciences
- PTC Therapeutics
- Poseida Therapeutics
- HST5040
- LB-001
- mRNA 3705
- SEL 302
- Vatiquinone
- P MMUT 101
Table of Contents
40 Pages
- Introduction
- Executive Summary
- Methylmalonic acidaemia: 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
- Methylmalonic acidaemia – DelveInsight’s Analytical Perspective
- Mid Stage Products (Phase II)
- Comparative Analysis
- HST5040: HemoShear Therapeutics
- Product Description
- Research and Development
- Product Development Activities
- Drug profiles in the detailed report…..
- Early Stage Products (Phase I/II)
- Comparative Analysis
- LB-001: LogicBio Therapeutics
- Product Description
- Research and Development
- Product Development Activities
- Drug profiles in the detailed report…..
- Preclinical and Discovery Stage Products
- Comparative Analysis
- P MMUT 101: Poseida Therapeutics
- Product Description
- Research and Development
- Product Development Activities
- Drug profiles in the detailed report…..
- Inactive Products
- Comparative Analysis
- Methylmalonic acidaemia Key Companies
- Methylmalonic acidaemia Key Products
- Methylmalonic acidaemia- Unmet Needs
- Methylmalonic acidaemia- Market Drivers and Barriers
- Methylmalonic acidaemia- Future Perspectives and Conclusion
- Methylmalonic acidaemia Analyst Views
- Methylmalonic acidaemia Key Companies
- Appendix
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