Homocystinuria Pipeline Appears Promising With 3+ Leading Biotech and Pharma Companies Driving Innovation in the Therapeutics Segment | DelveInsight

DelveInsight's, "Homocystinuria Pipeline Insight, 2025" report provides comprehensive insights about 3+ companies and 3+ pipeline drugs in Homocystinuria 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.

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Key Takeaways from the Homocystinuria Pipeline Report
• In April 2025, Travere Therapeutics Inc. announced a study visits will occur at regular intervals including home visits for study drug administration. Some study visits may be conducted remotely. Dietary protein intake and compliance with HCU treatments will continue to be monitored and recorded to ensure a stable diet and HCU treatment compliance.
• DelveInsight's Homocystinuria pipeline report depicts a robust space with 3+ active players working to develop 3+ pipeline therapies for Homocystinuria treatment.
• The leading Homocystinuria Companies such as Travere Therapeutics, Syntis Bio and others.
• Promising Homocystinuria Pipeline Therapies such as Pegtibatinase and others.

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Homocystinuria Emerging Drugs Profile
• TVT-058: Travere Therapeutics
Pegtibatinase (TVT-058) is an investigational PEGylated, recombinant enzyme replacement therapy designed to address the underlying cause of classical homocystinuria (HCU). In preclinical studies, pegtibatinase has demonstrated an ability to reduce total homocysteine levels and improve clinical parameters. Pegtibatinase is currently advancing in the Phase III Study to assess its safety, tolerability, pharmacokinetics, pharmacodynamics and clinical effects in patients with classical HCU. Pegtibatinase has been granted Rare Pediatric Disease and Fast Track designations by the US Food and Drug Administration (FDA), as well as Orphan Drug designation in the US and Europe.

The Homocystinuria pipeline report provides insights into
• The report provides detailed insights about companies that are developing therapies for the treatment of Homocystinuria with aggregate therapies developed by each company for the same.
• It accesses the Different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for Homocystinuria Treatment.
• Homocystinuria Companies are involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
• Homocystinuria Drugs under development based on the stage of development, route of administration, target receptor, monotherapy or combination therapy, a different mechanism of action, and molecular type.
• Detailed analysis of collaborations (company-company collaborations and company-academia collaborations), licensing agreement and financing details for future advancement of the Homocystinuria market.

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Homocystinuria Companies
Travere Therapeutics, Syntis Bio and others.

Homocystinuria 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
• Intravenous
• Subcutaneous
• Parenteral
• Topical

Homocystinuria Products have been categorized under various Molecule types such as
• Recombinant fusion proteins
• Small molecule
• Monoclonal antibody
• Peptide
• Polymer
• Gene therapy

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Scope of the Homocystinuria Pipeline Report
• Coverage- Global
• Homocystinuria Companies- Travere Therapeutics, Syntis Bio and others.
• Homocystinuria Pipeline Therapies- Pegtibatinase and others.
• Homocystinuria Therapeutic Assessment by Product Type: Mono, Combination, Mono/Combination
• Homocystinuria Therapeutic Assessment by Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III

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Table of Contents
1. Introduction
2. Executive Summary
3. Homocystinuria: Overview
4. Pipeline Therapeutics
5. Therapeutic Assessment
6. Homocystinuria - DelveInsight's Analytical Perspective
7. Late Stage Products (Phase III)
8. TVT-058: Travere Therapeutics
9. Drug profiles in the detailed report.....
10. Mid Stage Products (Phase II)
11. Drug name: Company name
12. Drug profiles in the detailed report.....
13. Early Stage Products (Phase I/II)
14. Drug name: Company name
15. Preclinical and Discovery Stage Products
16. SYNB 1353: Synlogic
17. Inactive Products
18. Homocystinuria Key Companies
19. Homocystinuria Key Products
20. Homocystinuria- Unmet Needs
21. Homocystinuria- Market Drivers and Barriers
22. Homocystinuria- Future Perspectives and Conclusion
23. Homocystinuria Analyst Views
24. Homocystinuria Key Companies
25. Appendix

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