Urea Cycle Disorders Pipeline 2025: Innovative Clinical Developments By 8+ Global Leaders Delveinsight Highlighting Acer Therapeutics, Dimension Therapeutics, Callitas Therapeutics, Poseida Therap

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"Urea Cycle Disorders Pipeline 2025"DelveInsight's,“Urea Cycle Disorders - Pipeline Insight, 2025,” 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.

With urea cycle disorders becoming increasingly prevalent worldwide and contributing to comorbidities such as diabetes, cardiovascular disease, and certain cancers, there is a growing need for safer and more effective treatment options. According to DelveInsight, more than eight pharmaceutical and biotech companies are actively developing over eight therapeutic candidates targeting urea cycle disorders. These therapies are at various stages of clinical and preclinical development, reflecting significant innovation and a strong commitment to addressing this critical public health challenge.

DelveInsight's“Urea Cycle Disorders Pipeline Insight 2025” offers a comprehensive and strategic assessment of the current R&D landscape. The report reviews clinical trial progress, emerging therapies, mechanisms of action, competitive dynamics, and key company initiatives. It serves as an essential resource for stakeholders-including researchers, healthcare investors, and decision-makers-seeking insights into the evolving urea cycle disorders therapeutics market and the innovations shaping its future.

Explore the Cutting-Edge Landscape of Urea cycle disorders Drug Development

Key Takeaways from the Urea cycle disorders Pipeline Report

• DelveInsight's urea cycle disorders pipeline report highlights a dynamic landscape, with more than eight active companies developing over eight therapeutic candidates for the treatment of urea cycle disorders.

• In May 2025, a landmark case involved a six-month-old infant with CPS1 deficiency, a type of urea cycle disorder, receiving a personalized CRISPR-based gene editing therapy. Developed by researchers at the University of Pennsylvania and Children's Hospital of Philadelphia, the therapy corrected a specific liver cell mutation using CRISPR-Cas9 technology. The FDA approved the treatment within weeks, representing a major step forward in personalized medicine for rare genetic disorders.

• In September 2024, the FDA granted Orphan Drug Designation to CMP-CPS-001, a candidate from CAMP4 Therapeutics for UCD treatment. This designation aims to accelerate the development of therapies for rare diseases by offering incentives such as tax credits and market exclusivity upon approval.

• Leading companies in the urea cycle disorders space-including 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 actively developing new therapies to enhance the treatment landscape. Promising pipeline candidates include ACER-001, Avalotcagene ontaparvovec, and several others at various stages of development.

Urea cycle disorders Overview:

Urea Cycle Disorder (UCD) is a hereditary condition. When we eat protein, the body breaks it down into amino acids, which are vital for growth and overall health. Excess amino acids are normally converted into ammonia, a waste product, which the liver enzymes then process into urea for elimination through urine. In people with UCD, the required enzyme is absent or malfunctioning, causing ammonia to build up in the bloodstream. This accumulation can be toxic and affect the brain, resulting in symptoms such as memory loss, behavioral changes, seizures, coma, or permanent brain damage. UCD affects roughly 1 in 35,000 births in the U.S. While rare, there are resources and support available for individuals 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:

Urea cycle disorders Emerging Drugs

ACER-001: Acer Therapeutics

ACER-001 is a powdered formulation of sodium phenylbutyrate (NaPB) designed to mask its taste while providing immediate release. Using a microencapsulation process, ACER-001 is being developed to treat inherited metabolic disorders such as urea cycle disorders (UCDs) and Maple Syrup Urine Disease (MSUD). Each microparticle consists of a core, an active drug layer, and a taste-masking coating that dissolves quickly in the stomach, neutralizing the taste while enabling rapid absorption into the bloodstream. The therapy is currently in Phase 3 clinical trials for UCDs.

Avalotcagene ontaparvovec: Dimension Therapeutics

Avalotcagene ontaparvovec is a replacement therapy targeting Ornithine carbamoyltransferase, developed by Dimension Therapeutics for the treatment of inherited urea cycle disorders. The therapy is currently undergoing Phase 2 clinical trials.

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

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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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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.

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