Urea cycle disorders Pipeline 2025: Groundbreaking Clinical Advancements by 8+ Global Leaders - DelveInsight | Featuring Acer Therapeutics, Dimension Therapeutics, Callitas Therapeutics, Poseida Therapeutics, Promethera Biosciences, Arcturus Therapeutics,

With Urea cycle disorders reaching epidemic proportions globally and contributing significantly to comorbid conditions such as diabetes, cardiovascular disease, and certain cancers, there is a growing demand for safer, more effective treatment options. According to DelveInsight, the Urea cycle disorders pipeline comprises 8+ pharmaceutical and biotech companies actively developing 8+ therapeutic candidates targeting Urea cycle disorders. These therapies span various stages of clinical and non-clinical development, underscoring the intense innovation and commitment to addressing one of the most pressing public health challenges of our time.
DelveInsight's "Urea cycle disorders Pipeline Insight 2025" report provides a detailed and strategic evaluation of the ongoing R&D landscape. It covers clinical trial progression, emerging therapies, mechanisms of action, competitive positioning, and key company initiatives. The report serves as a crucial resource for stakeholders-including researchers, healthcare investors, and decision-makers, seeking insights into the evolving Urea cycle disorders Therapeutics Market and the breakthroughs shaping its future trajectory.

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Key Takeaways from the Urea cycle disorders Pipeline Report

DelveInsight's Urea cycle disorders pipeline report depicts a robust space with 8+ active players working to develop 8+ pipeline therapies for Urea cycle disorders treatment.
In May 2025, a groundbreaking case was reported where a six-month-old infant with a urea cycle disorder, specifically CPS1 deficiency, received a personalized CRISPR-based gene editing therapy. Developed by researchers at the University of Pennsylvania and Children's Hospital of Philadelphia, this therapy involved correcting a specific liver cell mutation using CRISPR-Cas9 technology. The FDA approved the therapy within weeks, marking a significant advancement in personalized medicine for rare genetic disorders.
In September 2024, the FDA granted Orphan Drug Designation to CMP-CPS-001, a therapeutic candidate developed by CAMP4 Therapeutics for the treatment of UCDs. This designation is intended to facilitate the development of treatments for rare diseases by providing incentives such as tax credits and market exclusivity upon approval.
Key Urea cycle disorders companies such as 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, and others are evaluating new drugs for Urea cycle disorders to improve the treatment landscape.
Promising Urea cycle disorders pipeline therapies in various stages of development include ACER-001, Avalotcagene ontaparvovec, and others.

Urea cycle disorders Overview:

Urea Cycle Disorder (UCD) is a genetic condition. When we consume protein, our bodies break it down into amino acids, which are essential for growth and health. Any excess amino acids are converted into ammonia, a waste product. Normally, enzymes in the liver convert ammonia into urea, which is then eliminated from the body through urine. In individuals with UCD, the necessary enzyme is either missing or not functioning properly. As a result, ammonia accumulates in the bloodstream, which can be toxic. This excess ammonia can reach the brain, leading to symptoms such as memory loss, changes in behavior, seizures, coma, or brain damage. UCD occurs in approximately 1 in 35,000 births in the U.S. Although it is rare, there are resources and information available to support those living with UCD and their families.

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Urea cycle disorders Pipeline Analysis
The Urea cycle disorders pipeline insights report 2025, provides insights into:
Provides comprehensive insights into key companies developing therapies in the Urea cycle disorders Market.

Categorizes Urea cycle disorders therapeutic companies by development stage: early, mid, and late-stage.

Highlights major companies involved in targeted therapy development, including both active and inactive (paused/discontinued) projects.

Reviews emerging Urea cycle disorders drugs under development based on:

Stage of development

Urea cycle disorders Route of administration

Target receptor

Monotherapy vs. combination therapy

Urea cycle disorders Mechanism of action

Molecular type

Offers detailed analysis of:

Company-to-company and company-academia collaborations

Urea cycle disorders Licensing agreements

Funding and investment activities supporting future Urea cycle disorders market advancement.

Unlock key insights into emerging Urea cycle disorders therapies and market strategies here: https://www.delveinsight.com/report-store/urea-cycle-disorders-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=kpr

Urea cycle disorders Emerging Drugs

ACER-001: Acer Therapeutics

ACER-001 is a powdered form of sodium phenylbutyrate (NaPB) with a unique formulation designed to mask its taste while providing immediate release. Developed through a microencapsulation technique, ACER-001 is intended to treat several inherited metabolic disorders, including Urea Cycle Disorders (UCDs) and Maple Syrup Urine Disease (MSUD). The microparticles of ACER-001 have a core, an active drug layer, and a taste-masking coating that dissolves quickly in the stomach, neutralizing the taste but allowing for fast absorption into the bloodstream. The drug is currently in Phase 3 clinical trials for the treatment of UCDs.

Avalotcagene ontaparvovec: Dimension Therapeutics

Avalotcagene ontaparvovec is a replacement therapy for Ornithine carbamoyltransferase, being developed by Dimension Therapeutics to treat inherited Urea Cycle Disorders. The therapy is currently in Phase 2 of clinical development.

Urea cycle disorders Pipeline Therapeutic Assessment

Urea cycle disorders Assessment by Product Type
• Mono
• Combination
• Mono/Combination

Urea cycle disorders By Stage
• 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

Urea cycle disorders Assessment by Route of Administration
• Oral
• Parenteral
• Intravenous
• Subcutaneous
• Topical

Urea cycle disorders Assessment by Molecule Type
• Recombinant fusion proteins
• Small molecule
• Monoclonal antibody
• Peptide
• Polymer
• Gene therapy

Download sample pages to get an in-depth assessment of the emerging Urea cycle disorders therapies and key Urea cycle disorders companies: https://www.delveinsight.com/report-store/urea-cycle-disorders-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=kpr

Table of Contents

1. Report Introduction
2. Executive Summary
3. Urea cycle disorders Current Treatment Patterns
4. Urea cycle disorders - DelveInsight's Analytical Perspective
5. Therapeutic Assessment
6. Urea cycle disorders Late-Stage Products (Phase-III)
7. Urea cycle disorders Mid-Stage Products (Phase-II)
8. Early Stage Products (Phase-I)
9. Pre-clinical Products and Discovery Stage Products
10. Inactive Products
11. Dormant Products
12. Urea cycle disorders Discontinued Products
13. Urea cycle disorders Product Profiles
14. Urea cycle disorders Key Companies
15. Urea cycle disorders Key Products
16. Dormant and Discontinued Products
17. Urea cycle disorders Unmet Needs
18. Urea cycle disorders Future Perspectives
19. Urea cycle disorders Analyst Review
20. Appendix
21. Report Methodology

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Contact Info:
Kritika Rehani
Assistant Manager, Marketing & Branding
krehani@delveinsight.com
info@delveinsight.com
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About DelveInsight

DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing comprehensive end-to-end solutions to improve their performance.

This release was published on openPR.