SYNB-1618 is the most anticipated emerging therapy, awaiting launch. Apart from this, other products in the Phenylketonuria (PKU) therapeutic pipeline segment include CNSA-001, BMN 307, and one gene therapy, HMI-102. Some of these products have also received Fast track and Orphan Designations. Key players, such as Synlogic, Censa Pharmaceuticals, Codexis, Homology Medicines, etc., are developing therapies for the treatment of patients with Phenylketonuria.
As per DelveInsight, the increasing awareness, growing research in the field of genomics and bioinformatics, changing lifestyles, advancements in the healthcare industry, and rising per capita income in emerging markets are expected to drive the Phenylketonuria diagnosis and treatment market in the coming year’s. Moreover, the rising mergers and acquisitions and collaboration activities among the major market participants are also estimated to drive the Phenylketonuria therapeutics market growth.
“Phenylketonuria (PKU) Pipeline Insight, 2022” report by DelveInsight provides a comprehensive analysis of the ongoing clinical development activities and growth prospects across the Phenylketonuria Therapeutics Market.
The report provides a detailed description of the drug, including the mechanism of action, type, stage, route of administration, molecule type, clinical studies, inactive pipeline products, and NDA approvals (if any).
The report also covers commercial and clinical activities of the pipeline products from the pre-clinical developmental phase to the marketed stage. Additionally, it presents the ongoing developments in the therapeutics segment, including collaborations, licensing, mergers & acquisitions (M&A), funding, designations, and other product-related details.
Phenylketonuria (PKU) Pipeline Analysis
The report provides insights into:
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The report provides detailed insights into the key companies that are developing therapies in the Phenylketonuria Market.
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The report also evaluates different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for Phenylketonuria treatment.
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It analyzes the key companies involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
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It navigates the emerging 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.
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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.
The Report Covers the Emerging Pipeline Products Under Different Phases of Clinical Development Like –
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Late-stage products (Phase III)
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Mid-stage products (Phase II)
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Early-stage products (Phase I)
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Pre-clinical and Discovery stage candidates
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Discontinued & Inactive candidates
Phenylketonuria (PKU) – Mechanism of Action (MOA)
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Mixed-function oxygenase modulator
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Gastrointestinal microbiome modulator
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Phenylalanine ammonia-lyase replacement
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Gene transference
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SLC6A19 protein inhibitor
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Phenylalanine hydroxylase stimulant
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Phenylalanine hydroxylase replacement
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Phenylketonuria (PKU) Therapeutics Analysis
The management of Phenylketonuria is complex and dietary non-adherence often increases in adolescence and early adulthood, particularly due to social issues. Besides a low Phe diet, promising drugs are being developed for PKU. Oral sapropterin dihydrochloride (KUVAN), a synthetic form of BH4, could help lower Phe levels in some PKU patients. To further improve the treatment scenario, companies across the globe are diligently working toward the development of novel treatment therapies with a considerable amount of success over the years.
There are approx. 10+ key companies which are developing therapies for Phenylketonuria. PTC Therapeutics is leading the therapeutics market with its Phenylketonuria drug candidates in the most advanced stage of clinical development.
Phenylketonuria (PKU) Therapies Covered in the Report Include:
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Homology Medicines
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PTC Therapeutics
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Synlogic
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Rubius Therapeutics
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Agios Pharmaceuticals
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Generation Bio
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Jnana Therapeutics
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Sepiapterin: PTC therapeutics
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BioMarin Pharmaceuticals
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Jnana Therapeutics
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APR Applied Pharma Research
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Censa Pharmaceuticals
And many more
Phenylketonuria (PKU) Drugs Covered in the Report Include
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SYNB1618: Synlogic
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HMI-102: Homology Medicines
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HMI 103: Homology Medicines
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SYNB1618: Synlogic
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RTX-134: Rubius Therapeutics
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Sepiapterin: PTC therapeutics
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BMN-307: BioMarin Pharmaceuticals
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JNT 517: Jnana Therapeutics
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APR-OD031: APR Applied Pharma Research
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CNSA-001: Censa Pharmaceuticals
And More Others
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Table of Content
1. Report Introduction
2. Executive Summary
3. Phenylketonuria Current Treatment Patterns
4. Phenylketonuria – DelveInsight’s Analytical Perspective
5. Therapeutic Assessment
6. Phenylketonuria Late Stage Products (Phase-III)
7. Phenylketonuria 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. Phenylketonuria Discontinued Products
13. Phenylketonuria Product Profiles
14. Phenylketonuria Key Companies
15. Phenylketonuria Key Products
16. Dormant and Discontinued Products
17. Phenylketonuria Unmet Needs
18. Phenylketonuria Future Perspectives
19. Phenylketonuria Analyst Review
20. Appendix
21. Report Methodology
*The table of contents is not exhaustive; the final content may vary.
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