Urea Cycle Disorders - Pipeline Insight, 2024

This “Urea Cycle Disorders - Pipeline Insight, 2024,” report provides comprehensive insights about 8+ companies and 8+ pipeline drugs in Urea Cycle Disorders 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

Urea Cycle Disorders Understanding

Urea Cycle Disorders: Overview

A urea cycle disorder (UCD) is an inherited disease. When we eat protein our bodies break it down into amino acids which we need to grow and stay healthy. Extra amino acids that our body doesn’t need are turned into a waste product called ammonia. Enzymes in the liver turn the ammonia into urea so the body can get rid of it through the urine. In someone with a UCD, the enzyme is missing or is not working correctly. Because of this, ammonia builds up in the blood and it can be harmful. It can travel to the brain and cause memory loss, behavior changes, coma, seizures, or brain damage. UCDs affect about 1 in 35,000 births in the United States. Just because it’s rare doesn’t mean there isn’t information to help you or someone you love who is living with a UCD.

Urea Cycle Disorders - Pipeline Insight, 2024 report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Urea Cycle Disorders pipeline landscape is provided which includes the disease overview and Urea Cycle Disorders treatment guidelines. The assessment part of the report embraces, in depth Urea Cycle Disorders 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, Urea Cycle Disorders 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 Urea Cycle Disorders R&D. The therapies under development are focused on novel approaches to treat/improve Urea Cycle Disorders.

Urea Cycle Disorders Emerging Drugs Chapters

This segment of the Urea Cycle Disorders 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.

Urea Cycle Disorders Emerging Drugs

ACER-001: Acer Therapeutics ACER-001 is a powder formulation of sodium phenylbutyrate (Na PB). The proprietary formulation is designed to be both taste-masked and immediate release. ACER-001 is being developed using a microencapsulation process for the treatment of various inborn errors of metabolism, including UCDs and MSUD. ACER-001 microparticles consist of a core center, a layer of active drug, and a taste-masking coating that quickly dissolves in the stomach, allowing taste to be neutralized while still allowing for rapid systemic release. Currently, it is in phase 3 of development stage for the treatment of inborn Urea CycleDisorders.

Avalotcagene ontaparvovec: Dimension Therapeutics Avalotcagene ontaparvovec is a Ornithine carbamoyltransferase replacement therapy being developed by Dimension Therapeutics for the treatment of Inborn Urea Cycle Disorders. Currently, the therapy is in phase 2 stage of development.

Urea Cycle Disorders: Therapeutic Assessment

This segment of the report provides insights about the different Urea Cycle Disorders drugs segregated based on following parameters that define the scope of the report, such as:

Major Players in Urea Cycle Disorders

There are approx. 8+ key companies which are developing the therapies for Urea Cycle Disorders. The companies which have their Urea Cycle Disorders drug candidates in the most advanced stage, i.e. phase III include, Acer Therapeutics.

Phases

This report covers around 8+ 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

Urea Cycle Disorders 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
• intravitreal
• Subretinal
• 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.

Urea Cycle Disorders: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Urea Cycle Disorders 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 Urea Cycle Disorders drugs.

Urea Cycle Disorders Report Insights

• Urea Cycle Disorders Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

Urea Cycle Disorders 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 Urea Cycle Disorders drugs?
• How many Urea Cycle Disorders drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Urea Cycle Disorders?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Urea Cycle Disorders 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 Urea Cycle Disorders and their status?
• What are the key designations that have been granted to the emerging drugs?

Key Players

• Acer Therapeutics
• Dimension Therapeutics
• Callitas Therapeutics
• Poseida Therapeutics
• Promethera Biosciences
• Arcturus Therapeutics
• Kaleido Biosciences
• Akaza Biopharma
• Evox Therapeutics
• Dipharma SA
• Sana Biotechnology
• Aeglea BioTherapeutics
• ERYTECH

Key Products

• ACER-001
• Avalotcagene ontaparvovec
• MACS
• P OTC 101
• Heparesc
• ARCT 810
• VS 01
• KB195
• HepaStem
• Resatorvid
• EVX-103
• SG 328
• Pegzilarginase
• ERY-ADI

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