Methylmalonic Acidaemia Pipeline Research Report 2023: Comprehensive Insights About 4+ Companies and 4+ Pipeline Drugs
14 Dec 2023
Orphan DrugPhase 2Fast Track
DUBLIN, Dec. 13, 2023 /PRNewswire/ -- The "Methylmalonic Acidaemia - Pipeline Insight, 2023" clinical trials has been added to
ResearchAndMarkets.com's offering.
This report provides comprehensive insights about 4+ companies and 4+ 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.
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.
Methylmalonic Acidaemia Emerging Drugs Chapters
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 is an investigational oral small molecule therapy being developed by HemoShear to reduce the levels of toxins associated with methylmalonic acidemia (MMA) and propionic acidemia (PA), rare genetic disorders caused by the deficiency of certain enzymes required to metabolize amino acids. HST5040 is formulated for convenient daily administration at home as a liquid taken either orally or through a gastrostomy tube. The FDA has granted HST5040 Orphan Drug, Fast Track and Rare Pediatric Disease designations for the treatment of MMA and PA.Currently, the drug is in Phase II stage of Clinical trial evaluation for the treatment of Methylmalonic Acidemia.
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.
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?
Key Players
Key Products
mRNA-3705
Methylmalonic Acidaemia: Therapeutic Assessment
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
Products have been categorized under various ROAs such as
Intra-articular
Intraocular
Intrathecal
Intravenous
Ophthalmic
Oral
Parenteral
Subcutaneous
Topical
Transdermal
Molecule Type
Products have been categorized under various Molecule types such as
Oligonucleotide
Peptide
Small molecule
Product Type
Methylmalonic Acidaemia Report Insights
Methylmalonic Acidaemia Pipeline Analysis
Therapeutic Assessment
Unmet Needs
Impact of Drugs
For more information about this clinical trials report visit https://www.researchandmarkets.com/r/hidgfv
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