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“Phenylktonuria Pipeline Insight, 2025“ report by DelveInsight outlines comprehensive insights into the present clinical development scenario and growth prospects across the Phenylktonuria Market.

The Phenylktonuria 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 Phenylktonuria Pipeline Report:

• Companies across the globe are diligently working toward developing novel Phenylktonuria treatment therapies with a considerable amount of success over the years.

• Phenylktonuria 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 Phenylktonuria treatment

• Emerging Phenylktonuria 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 Phenylktonuria market in the coming years.

• In December 2024, Alltrna has unveiled new preclinical findings, including data on its lead tRNA development candidate, AP003. This chemically engineered and modified tRNA oligonucleotide is delivered via a clinically validated, liver-targeted lipid nanoparticle (LNP) system. AP003 is designed to bypass the arginine to TGA (Arg-TGA) premature termination codon (PTC). In two transgenic mouse models of Stop Codon Diseases—methylmalonic acidemia (MMA) and phenylketonuria (PKU)—a single dose of AP003 demonstrated a strong in vivo ability to restore protein production to levels considered clinically meaningful.

• In October 2024, PTC Therapeutics, Inc. has announced that the FDA has accepted the New Drug Application (NDA) for sepiapterin, intended for treating both pediatric and adult patients with phenylketonuria (PKU). The Prescription Drug User Fee Act (PDUFA) target action date will be communicated in the Day 74 Letter.

• In July 2024, PTC Therapeutics, Inc. announced that it has submitted a New Drug Application (NDA) to the U.S. FDA for sepiapterin. The application seeks approval for the treatment of phenylketonuria (PKU) in both pediatric and adult patients, covering all age groups and disease subtypes.

• In September 2024, Jnana Therapeutics shared findings from the 150mg twice-daily (BID) second-dose cohort in its ongoing Phase I/II clinical trial evaluating JNT-517 in adults with phenylketonuria (PKU).

• In August 2024, Otsuka Pharmaceutical Co., Ltd. (Otsuka) and Jnana Therapeutics Inc. have entered into a definitive merger agreement under which Otsuka will acquire Jnana. Following the transaction, Jnana will become a wholly owned subsidiary of Otsuka through its fully owned subsidiary, Otsuka America, Inc. (OAI).

Phenylktonuria Overview

Phenylktonuria (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 Phenylktonuria 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

Phenylktonuria Route of Administration

Phenylktonuria 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

Phenylktonuria Molecule Type

Phenylktonuria Products have been categorized under various Molecule types, such as

• Oligonucleotide

• Peptide

• Small molecule

Phenylktonuria Pipeline Therapeutics Assessment

• Phenylktonuria Assessment by Product Type

• Phenylktonuria By Stage and Product Type

• Phenylktonuria Assessment by Route of Administration

• Phenylktonuria By Stage and Route of Administration

• Phenylktonuria Assessment by Molecule Type

• Phenylktonuria by Stage and Molecule Type

DelveInsight’s Phenylktonuria 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 Phenylktonuria product details are provided in the report. Download the Phenylktonuria pipeline report to learn more about the emerging Phenylktonuria therapies

Some of the key companies in the Phenylktonuria Therapeutics Market include:

Key companies developing therapies for Phenylktonuria 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.

Phenylktonuria Pipeline Analysis:

The Phenylktonuria pipeline report provides insights into

• The report provides detailed insights about companies that are developing therapies for the treatment of Phenylktonuria 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 Phenylktonuria Treatment.

• Phenylktonuria key companies are involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.

• Phenylktonuria 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 Phenylktonuria 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.

Download Sample PDF Report to know more about Phenylktonuria drugs and therapies

Phenylktonuria Pipeline Market Drivers

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

Phenylktonuria Pipeline Market Barriers

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

Scope of Phenylktonuria Pipeline Drug Insight

• Coverage: Global

• Key Phenylktonuria Companies: Nestlé Health Science, Homology Medicine, Jnana Therapeutics, BioMarin Pharmaceutical, PTC Therapeutics, Synlogic, and others

• Key Phenylktonuria Therapies: CDX 6114, HMI-103, JNT 517:, BMN 307, Sepiapterin, SYNB1934, and others

• Phenylktonuria Therapeutic Assessment: Phenylktonuria current marketed and Phenylktonuria emerging therapies

• Phenylktonuria Market Dynamics: Phenylktonuria market drivers and Phenylktonuria market barriers

Request for Sample PDF Report for Phenylktonuria Pipeline Assessment and clinical trials

Table of Contents

1. Phenylktonuria Report Introduction

2. Phenylktonuria Executive Summary

3. Phenylktonuria Overview

4. Phenylktonuria- Analytical Perspective In-depth Commercial Assessment

5. Phenylktonuria Pipeline Therapeutics

6. Phenylktonuria Late Stage Products (Phase II/III)

7. Phenylktonuria Mid Stage Products (Phase II)

8. Phenylktonuria Early Stage Products (Phase I)

9. Phenylktonuria Preclinical Stage Products

10. Phenylktonuria Therapeutics Assessment

11. Phenylktonuria Inactive Products

12. Company-University Collaborations (Licensing/Partnering) Analysis

13. Phenylktonuria Key Companies

14. Phenylktonuria Key Products

15. Phenylktonuria Unmet Needs

16 . Phenylktonuria Market Drivers and Barriers

17. Phenylktonuria Future Perspectives and Conclusion

18. Phenylktonuria Analyst Views

19. Appendix

20. About DelveInsight

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. It also offers Healthcare Consulting Services, which benefits in market analysis to accelerate business growth and overcome challenges with a practical approach.

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