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Homocystinuria Clinical Trials and Pipeline 2025: EMA, PDMA, FDA Approvals, Medication, IND, NDA Approval, Therapies, Mechanism of Action, Route of Administration and Companies by DelveInsight

02-17-2025 06:27 PM CET | Health & Medicine

Press release from: DelveInsight Business Research

Homocystinuria Clinical Trials

Homocystinuria Clinical Trials

(New York, United States) "Homocystinuria Pipeline Insight, 2025" report by DelveInsight outlines comprehensive insights into the present clinical development scenario and growth prospects across the Homocystinuria Market.

As per DelveInsight's assessment, globally, Homocystinuria pipeline constitutes 3+ key companies continuously working towards developing 3+ Homocystinuria treatment therapies, analysis of Clinical Trials, Therapies, Mechanism of Action, Route of Administration, and Developments analyzes DelveInsight.

The Homocystinuria Pipeline report embraces in-depth commercial and clinical assessment of the pipeline products from the pre-clinical developmental phase to the marketed phase. The report also covers a detailed description of the drug, including the mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, collaborations, mergers acquisition, funding, designations, and other product-related details.

Request for Sample Report here @ https://www.delveinsight.com/report-store/homocystinuria-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=apr

Some of the key takeaways from the Homocystinuria Pipeline Report:
• Homocystinuria Companies across the globe are diligently working toward developing novel Homocystinuria treatment therapies with a considerable amount of success over the years.
• Homocystinuria companies working in the treatment market are Travere Therapeutics, Synlogic, Syntis Bio, and others, are developing therapies for the Homocystinuria treatment.
• Emerging Homocystinuria therapies such as TVT-058, SYNB 1353, and others are expected to have a significant impact on the Homocystinuria market in the coming years.
• Travere Therapeutics' HARMONY Study: Travere Therapeutics initiated the Phase III HARMONY Study in December 2023 to evaluate pegtibatinase for the treatment of classical Homocystinuria. However, enrolment was paused in September 2024 due to challenges in the drug substance profile during the scale-up process. The company is working on process improvements to resume enrolment.
• Aeglea BioTherapeutics' Pegtarviliase Trial: Aeglea BioTherapeutics is conducting a Phase 1/2 clinical trial for pegtarviliase, another enzyme replacement therapy for classical Homocystinuria. Dosing in the third cohort began in October 2022.

Homocystinuria Overview
Homocystinuria is a rare genetic disorder affecting the metabolism of the amino acid methionine, leading to an abnormal accumulation of homocysteine and its byproducts in the blood and urine. This condition is primarily caused by mutations in the CBS (cystathionine beta-synthase) gene, resulting in a deficiency or dysfunction of the CBS enzyme. Other less common forms involve defects in methionine synthase or cobalamin metabolism.
Clinical manifestations of homocystinuria are multisystemic, often impacting the eyes, skeletal system, central nervous system, and vascular system. Common symptoms include nearsightedness, lens dislocation, skeletal abnormalities such as scoliosis or osteoporosis, developmental delays, and an increased risk of thromboembolic events, which can be life-threatening.
Diagnosis is based on elevated plasma homocysteine levels, increased methionine, and confirmatory genetic testing. Early detection through newborn screening programs is crucial for timely intervention.
Treatment focuses on lowering homocysteine levels to prevent complications. This often involves a combination of dietary restrictions (low methionine diet), supplementation with vitamin B6 (pyridoxine), folate, and vitamin B12, as well as betaine therapy in some cases. Regular monitoring and a multidisciplinary approach are essential to optimize outcomes and manage complications associated with the disorder.

Get a Free Sample PDF Report to know more about Homocystinuria Pipeline Therapeutic Assessment- https://www.delveinsight.com/sample-request/homocystinuria-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=apr

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

Homocystinuria Pipeline Therapeutics Assessment
• Homocystinuria Assessment by Product Type
• Homocystinuria By Stage and Product Type
• Homocystinuria Assessment by Route of Administration
• Homocystinuria By Stage and Route of Administration
• Homocystinuria Assessment by Molecule Type
• Homocystinuria by Stage and Molecule Type

DelveInsight's Homocystinuria Report covers around 3+ products under different phases of clinical development like-
• Late-stage products (Phase III)
• Mid-stage products (Phase II)
• Early-stage product (Phase I)
• Pre-clinical and Discovery stage candidates
• Discontinued & Inactive candidates
• Route of Administration

Further Homocystinuria product details are provided in the report. Download the Homocystinuria pipeline report to learn more about the emerging Homocystinuria therapies- https://www.delveinsight.com/report-store/homocystinuria-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=apr

Homocystinuria Pipeline Analysis:
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 key 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.

Download Sample PDF Report to know more about Homocystinuria drugs and therapies- https://www.delveinsight.com/sample-request/homocystinuria-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=apr

Scope of Homocystinuria Pipeline Drug Insight
• Coverage: Global
• Key Homocystinuria Companies: Travere Therapeutics, Synlogic, Syntis Bio, and others.
• Key Homocystinuria Therapies: TVT-058, SYNB 1353, and others.
• Homocystinuria Therapeutic Assessment: Homocystinuria current marketed and Homocystinuria emerging therapies
• Homocystinuria Market Dynamics: Homocystinuria market drivers and Homocystinuria market barriers

Request for Sample PDF Report for Homocystinuria Pipeline Assessment and clinical trials - https://www.delveinsight.com/sample-request/homocystinuria-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=apr

Table of Contents
1. Homocystinuria Report Introduction
2. Homocystinuria Executive Summary
3. Homocystinuria Overview:
4. Homocystinuria- Analytical Perspective In-depth Commercial Assessment
5. Homocystinuria Pipeline Therapeutics
6. Homocystinuria Late Stage Products (Phase II/III)
7. Homocystinuria Mid Stage Products (Phase II)
8. Homocystinuria Early Stage Products (Phase I)
9. Homocystinuria Preclinical Stage Products
10. Homocystinuria Therapeutics Assessment
11. Homocystinuria Inactive Products
12. Company-University Collaborations (Licensing/Partnering) Analysis
13. Homocystinuria Companies
14. Homocystinuria Key Products
15. Homocystinuria Unmet Needs
16 . Homocystinuria Market Drivers and Barriers
17. Homocystinuria Future Perspectives and Conclusion
18. Homocystinuria Analyst Views
19. Appendix
20. About DelveInsight

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Contact Us:
Ankit Nigam
Manager Marketing
info@delveinsight.com
+14699457679
https://www.delveinsight.com/consulting/primary-research-services
Case study: https://www.delveinsight.com/case-study/esmo-conference-coverage

About DelveInsight
DelveInsight is a leading Life Science market research and business consulting company recognized for its off-the-shelf syndicated market research reports and customized solutions to firms in the healthcare sector.

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