Methylmalonic academia - Pipeline Insight, 2021
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DelveInsight’s, “Methylmalonic academia - Pipeline Insight, 2021,” report provides comprehensive insights about 6+ companies and 6+ pipeline drugs in Methylmalonic academia 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 academia: 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.
Report Highlights
This segment of the Methylmalonic academia report encloses its detailed analysis of various drugs in different stages of clinical development, including phase III, 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 academia Emerging Drugs
In December, 2020, HemoShear Therapeutics announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designations to HemoShear’s HST5040 oral small molecule for treatment of methylmalonic acidemia (MMA) and propionic acidemia (PA). HST5040 was granted Fast Track and Rare Pediatric Disease designations to treat MMA and PA earlier in 2020.
In November, 2020, LogicBio Therapeutics announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to its clinical candidate, LB-001 for the treatment methylmalonic acidemia (MMA). According to the FDA, the purpose of Fast Track designation is to get important new drugs to patients earlier by facilitating the development, and expediting the review, of drugs to treat serious conditions and fill an unmet medical need.
Further product details are provided in the report
Methylmalonic academia: Therapeutic Assessment
This segment of the report provides insights about the different Methylmalonic academia drugs segregated based on following parameters that define the scope of the report, such as:
Methylmalonic academia: Pipeline Development Activities
The report provides insights into different therapeutic candidates in phase III, II, I, preclinical and discovery stage. It also analyses Methylmalonic academia 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 academia drugs.
Methylmalonic academia Report Insights
Current Scenario and Emerging Therapies:
DelveInsight’s, “Methylmalonic academia - Pipeline Insight, 2021,” report provides comprehensive insights about 6+ companies and 6+ pipeline drugs in Methylmalonic academia 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
Methylmalonic academia: 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.
Report Highlights
- The companies and academics are working to assess challenges and seek opportunities that could influence Methylmalonic academia R&D. The therapies under development are focused on novel approaches for Methylmalonic acidemia.
This segment of the Methylmalonic academia report encloses its detailed analysis of various drugs in different stages of clinical development, including phase III, 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 academia Emerging Drugs
- HST5040: HemoShear Therapeutics
In December, 2020, HemoShear Therapeutics announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designations to HemoShear’s HST5040 oral small molecule for treatment of methylmalonic acidemia (MMA) and propionic acidemia (PA). HST5040 was granted Fast Track and Rare Pediatric Disease designations to treat MMA and PA earlier in 2020.
- LB-001: LogicBio Therapeutics
In November, 2020, LogicBio Therapeutics announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to its clinical candidate, LB-001 for the treatment methylmalonic acidemia (MMA). According to the FDA, the purpose of Fast Track designation is to get important new drugs to patients earlier by facilitating the development, and expediting the review, of drugs to treat serious conditions and fill an unmet medical need.
Further product details are provided in the report
Methylmalonic academia: Therapeutic Assessment
This segment of the report provides insights about the different Methylmalonic academia drugs segregated based on following parameters that define the scope of the report, such as:
- Major Players working on Methylmalonic academia
- Phases
- Late-stage products (Phase III and
- Mid-stage products (Phase II and
- Early-stage products (Phase I/II and Phase I) along with the details of
- Pre-clinical and Discovery stage candidates
- Discontinued & Inactive candidates
- Route of Administration
- Infusion
- Intradermal
- Intramuscular
- Intranasal
- Intravaginal
- Oral
- Parenteral
- Subcutaneous
- Topical
- Molecule Type
- Vaccines
- Monoclonal Antibody
- Peptides
- Polymer
- Small molecule
- Product Type
Methylmalonic academia: Pipeline Development Activities
The report provides insights into different therapeutic candidates in phase III, II, I, preclinical and discovery stage. It also analyses Methylmalonic academia 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 academia drugs.
Methylmalonic academia Report Insights
- Methylmalonic academia Pipeline Analysis
- Therapeutic Assessment
- Unmet Needs
- Impact of Drugs
- Pipeline Product Profiles
- Therapeutic Assessment
- Pipeline Assessment
- Inactive drugs assessment
- Unmet Needs
Current Scenario and Emerging Therapies:
- How many companies are developing Methylmalonic academia drugs?
- How many Methylmalonic academia drugs are developed by each company?
- How many emerging drugs are in mid-stage, and late-stage of development for Methylmalonic academia?
- What are the key collaborations (Industry–Industry, Industry–Academia), Mergers and acquisitions, licensing activities related to the Methylmalonic academia 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 academia and their status?
- What are the key designations that have been granted to the emerging drugs?
- HemoShear Therapeutics
- LogicBio Therapeutics
- Selecta Biosciences
- Asklepios BioPharmaceutical
- Moderna Therapeutics
- Poseida Therapeutics
- HST5040
- LB-001
- AAV8
- MMA-101
- mRNA-3705
- P-MMUT-101
Introduction
Executive Summary
Methylmalonic academia: Overview
Structure
Mechanism of Action
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 acidemia– DelveInsight’s Analytical Perspective
In-depth Commercial Assessment
Methylmalonic academia companies’ collaborations, Licensing, Acquisition -Deal Value Trends
Methylmalonic acidemiaCollaboration Deals
Company-Company Collaborations (Licensing / Partnering) Analysis
Company-University Collaborations (Licensing / Partnering) Analysis
Late Stage Products (Phase III)
Comparative Analysis
Drug Name: Company Name
Product Description
Research and Development
Product Development Activities
Drug profiles in the detailed report
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
Pre-clinical and Discovery Stage Products
Comparative Analysis
mRNA-3705: Moderna Therapeutics
Product Description
Research and Development
Product Development Activities
Drug profiles in the detailed report
Inactive Products
Comparative Analysis
Methylmalonic acidemiaKey Companies
Methylmalonic acidemiaKey Products
Methylmalonic academia- Unmet Needs
Methylmalonic academia- Market Drivers and Barriers
Methylmalonic academia- Future Perspectives and Conclusion
Methylmalonic academia Analyst Views
Methylmalonic academia Key Companies
Appendix
Executive Summary
Methylmalonic academia: Overview
Structure
Mechanism of Action
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 acidemia– DelveInsight’s Analytical Perspective
In-depth Commercial Assessment
Methylmalonic academia companies’ collaborations, Licensing, Acquisition -Deal Value Trends
Methylmalonic acidemiaCollaboration Deals
Company-Company Collaborations (Licensing / Partnering) Analysis
Company-University Collaborations (Licensing / Partnering) Analysis
Late Stage Products (Phase III)
Comparative Analysis
Drug Name: Company Name
Product Description
Research and Development
Product Development Activities
Drug profiles in the detailed report
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
Pre-clinical and Discovery Stage Products
Comparative Analysis
mRNA-3705: Moderna Therapeutics
Product Description
Research and Development
Product Development Activities
Drug profiles in the detailed report
Inactive Products
Comparative Analysis
Methylmalonic acidemiaKey Companies
Methylmalonic acidemiaKey Products
Methylmalonic academia- Unmet Needs
Methylmalonic academia- Market Drivers and Barriers
Methylmalonic academia- Future Perspectives and Conclusion
Methylmalonic academia Analyst Views
Methylmalonic academia Key Companies
Appendix
LIST OF TABLES
Table 1 Total Products for Methylmalonic academia
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 Methylmalonic academia
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 Methylmalonic academia
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 Methylmalonic academia
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