Methylmalonic acidaemia - Pipeline Insight, 2024

This “Methylmalonic acidaemia - Pipeline Insight, 2024,” 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

• Global coverage

Methylmalonic acidaemia Understanding

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, 2024 report 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.

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: Hemo Shear Therapeutics 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 de?ciency 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 andPA.

LB-001: LogicBio Therapeutics LB-001 is an investigational, first-in-class, single-administration, gene editing therapy for early intervention in methylmalonic acidemia (MMA) using the Gene Ride™ platform. GeneRide™ technology utilizes a natural DNA repair process called homologous recombination that enables precise editing of the genome without the need for exogenous nucleases and promoters that are associated with an increased risk of immune response and cancer. LB-001 is designed to non-disruptively insert a corrective copy of the methylmalonyl-CoA mutase (MMUT) gene into the albumin locus to drive lifelong therapeutic levels of MMUT expression in the liver, the main site of MMUT expression and activity. LB-001 is delivered to hepatocytes via liver-targeted, engineered recombinant adeno-associated virus vector (r AAV-LK03). The U.S. Food and Drug Administration (FDA) granted Fast Track designation for LB-001 for the treatment of MMA. In addition, the Company has received rare pediatric disease designation and orphan drug designation from the FDAforLB-001.

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

There are approx. 5+ key companies which are developing the therapies for Methylmalonic acidaemia. The companies which have their Methylmalonic acidaemia drug candidates in the most advanced stage, i.e. phase II include, Hemo ShearTherapeutics.

Phases

This report covers around 5+ 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

Methylmalonic acidaemia 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

Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.

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

Methylmalonic acidaemia Report Assessment

• Pipeline Product Profiles
• Therapeutic Assessment
• Pipeline Assessment
• Inactive drugs assessment
• Unmet Needs

Key Questions

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

• Hemo ShearTherapeutics
• LogicBio Therapeutics
• Moderna Therapeutics
• Selecta Biosciences
• PTC Therapeutics
• Poseida Therapeutics

Key Products

• HST5040
• LB-001
• m RNA3705
• SEL 302
• Vatiquinone
• P MMUT 101

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