Phenylketonuria Pipeline and Clinical Trials Assessment 2024: FDA Approvals, Therapies and Key Companies involved by DelveInsight |Erytech Pharma SA, Homology Medicines Inc., Synlogic Inc., BioMarin Pharmaceuticals Inc., Censa Pharmaceuticals (PTC Therape

(Las Vegas, Nevada, United States) As per DelveInsight's assessment, globally, Phenylketonuria pipeline constitutes 18+ key companies continuously working towards developing 20+ Phenylketonuria treatment therapies, analysis of Clinical Trials, Therapies, Mechanism of Action, Route of Administration, and Developments analyzes DelveInsight.

"Phenylketonuria Pipeline Insight, 2024" report by DelveInsight outlines comprehensive insights into the present clinical development scenario and growth prospects across the Phenylketonuria Market.

The Phenylketonuria 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.

Some of the key takeaways from the Phenylketonuria Pipeline Report: https://www.delveinsight.com/sample-request/phenylketonuria-pku-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=gpr
• Companies across the globe are diligently working toward developing novel Phenylketonuria treatment therapies with a considerable amount of success over the years.
• Phenylketonuria companies working in the treatment market are Nestlé Health Science, Homology Medicine, Jnana Therapeutics, BioMarin Pharmaceutical, PTC Therapeutics, Synlogic, and others, are developing therapies for the Phenylketonuria treatment
• Emerging Phenylketonuria therapies in the different phases of clinical trials are- CDX 6114, HMI-103, JNT 517:, BMN 307, Sepiapterin, SYNB1934, and others are expected to have a significant impact on the Phenylketonuria market in the coming years.
• In January 2024, Jnana Therapeutics, a clinical-stage biotech company using its advanced chemoproteomics platform to develop drugs for difficult targets, announced positive, statistically significant interim results from its ongoing clinical study of JNT-517 in phenylketonuria (PKU) patients. JNT-517, a small molecule inhibitor targeting the phenylalanine (Phe) transporter SLC6A19, is being tested as a potential first-in-class oral treatment for PKU in all ages and genotypes. Based on these encouraging interim results, Jnana has modified the Phase 1b trial design to potentially expedite the development of JNT-517.
• In July 2023, The US Food and Drug Administration (FDA) has granted fast track designation to Synlogic's labafenogene marselecobac (SYNB1934), a clinical-stage biotechnology company's treatment for phenylketonuria (PKU). This oral therapy, which is not systemically absorbed, targets and breaks down phenylalanine (Phe) in the gastrointestinal tract.

Phenylketonuria Overview
Phenylketonuria (PKU) is a rare genetic disorder characterized by the body's inability to properly process an amino acid called phenylalanine (Phe). This condition is caused by a defect in the gene responsible for producing an enzyme called phenylalanine hydroxylase (PAH). PAH is essential for breaking down phenylalanine into other substances that the body needs.

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Emerging Phenylketonuria Drugs Under Different Phases of Clinical Development Include:
• CDX 6114: Nestlé Health Science
• HMI-103: Homology Medicine
• JNT 517: Jnana Therapeutics
• BMN 307: BioMarin Pharmaceutical
• Sepiapterin: PTC Therapeutics
• SYNB1934: Synlogic

Phenylketonuria Route of Administration
Phenylketonuria pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs, such as
• Intra-articular
• Intraocular
• Intrathecal
• Intravenous
• Ophthalmic
• Oral
• Parenteral
• Subcutaneous
• Topical
• Transdermal

Phenylketonuria Molecule Type
Phenylketonuria Products have been categorized under various Molecule types, such as
• Oligonucleotide
• Peptide
• Small molecule

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

DelveInsight's Phenylketonuria Report covers around 20+ 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 Phenylketonuria product details are provided in the report. Download the Phenylketonuria pipeline report to learn more about the emerging Phenylketonuria therapies at:
https://www.delveinsight.com/report-store/phenylketonuria-pku-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=gpr

Some of the key companies in the Phenylketonuria Therapeutics Market include:
Key companies developing therapies for Phenylketonuria are - Erytech Pharma SA, Homology Medicines Inc., Synlogic Inc., BioMarin Pharmaceuticals Inc., Censa Pharmaceuticals (PTC Therapeutics), Codexis Inc., SOM Innovation Biotech SL, Ultragenyx (Dimension Therapeutics), and others.

Phenylketonuria Pipeline Analysis:
The Phenylketonuria pipeline report provides insights into
• The report provides detailed insights about companies that are developing therapies for the treatment of Phenylketonuria 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 Phenylketonuria Treatment.
• Phenylketonuria key companies are involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
• Phenylketonuria 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 Phenylketonuria market.
The report is built using data and information traced from the researcher's proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations, and featured press releases from company/university websites and industry-specific third-party sources, etc.

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Phenylketonuria Pipeline Market Drivers
• Rising awareness about the disease, growing Research and Development Activities are some of the important factors that are fueling the Phenylketonuria Market.

Phenylketonuria Pipeline Market Barriers
• However, neurobehavioral Problems Associated with Phenylketonuria, limitations associated with the current treatment and other factors are creating obstacles in the Phenylketonuria Market growth.

Scope of Phenylketonuria Pipeline Drug Insight
• Coverage: Global
• Key Phenylketonuria Companies: Nestlé Health Science, Homology Medicine, Jnana Therapeutics, BioMarin Pharmaceutical, PTC Therapeutics, Synlogic, and others
• Key Phenylketonuria Therapies: CDX 6114, HMI-103, JNT 517:, BMN 307, Sepiapterin, SYNB1934, and others
• Phenylketonuria Therapeutic Assessment: Phenylketonuria current marketed and Phenylketonuria emerging therapies
• Phenylketonuria Market Dynamics: Phenylketonuria market drivers and Phenylketonuria market barriers

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

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