Phenylketonuria Pipeline Landscape Analysis of 10+ Companies by DelveInsight

October 04 19:55 2022
Phenylketonuria Pipeline Landscape Analysis of 10+ Companies by DelveInsight

DelveInsight’s, “Phenylketonuria Pipeline Insight, 2022,” report provides comprehensive insights about 10+ companies and 15+ pipeline drugs in Phenylketonuria pipeline landscape.


Phenylketonuria Pipeline landscape is provided which includes the disease overview and Phenylketonuria treatment guidelines. The assessment part of the Phenylketonuria pipeline report embraces, in depth Phenylketonuria commercial assessment and clinical assessment of the pipeline products under development. In the Phenylketonuria Pipeline Report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Phenylketonuria collaborations, licensing, mergers and acquisition, funding, designations and other product related details.


Key takeaways from the Phenylketonuria Pipeline Report

  • DelveInsight’s Phenylketonuria Pipeline report depicts a robust space with 10+ active players working to develop 15+ pipeline therapies for various indications.
  • Leading Phenylketonuria Companies such as Homology Medicines, PTC Therapeutics, Synlogic, Rubius Therapeutics, Agios Pharmaceuticals, Generation Bio, Jnana Therapeutics, and others are developing potential drug candidates to improve the Phenylketonuria treatment scenario.
  • The promising Phenylketonuria therapies in the pipeline in various stages of development includes SYNB1618, HMI-102, and others.
  • SYNB1618 is an oral investigational Synthetic Biotic medicine designed to break down phenylalanine in the GI tract as a potential treatment for patients with PKU. A solid oral lyophilized powder formulation was evaluated in a Phase 1 study in healthy volunteers. The drug is currently being evaluated in Phase II stage of development for the treatment of Phenylketonuria.
  • HMI-102 is a gene therapy that uses a harmless adeno-associated viral vector called AAVHSC15, developed from human stem cells, to deliver a functional copy of the PAH gene to cells in the liver. It is designed to produce a lasting effect after a single intravenous (into-the-vein) dose. The drug is in Phase I/II clinical trials for phenylketonuria.


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The Phenylketonuria Pipeline report provides detailed profiles of pipeline assets, a comparative analysis of clinical and non-clinical stage Phenylketonuria, inactive and dormant assets, a comprehensive assessment of driving and restraining factors, and an assessment of opportunities and risks in the Phenylketonuria Pipeline Landscape.


Phenylketonuria Overview

Phenylketonuria (PKU) is an inborn error of metabolism that is detectable during the first days of life via routine newborn screening. PKU is inherited in an autosomal recessive pattern. PKU is characterized by absence or deficiency of an enzyme called phenylalanine hydroxylase (PAH), responsible for processing the amino acid phenylalanine. Infants with PKU typically appear normal at birth. With early screening and dietary treatment, affected individuals may never show symptoms of PKU. Symptoms may include vomiting, irritability, and/or a red skin rash with small pimples. Neurological symptoms are present in some untreated patients with PKU, including seizures, abnormal muscle movements, tight muscles, increased reflexes, involuntary movements, or tremor. The goal of treatment for PKU is to keep plasma phenylalanine levels within 120-360 umol/L (2-6 mg/dL). This is generally achieved through carefully planned and monitored diet.


Latest Breakthroughs and Developments in the Phenylketonuria Treatment Landscape

  • In August 2021, Janana Therapeutics announced the closing of a $50 million Series B financing To Advance Lead PKU Program And Small Molecule Pipeline Based On Next-Gen Chemoproteomic Platform.
  • In October 2020, the Food and Drug Administration (FDA) granted Fast Track designation to the investigational gene therapy BMN 307 (BioMarin Pharmaceutical) for the treatment of patients with phenylketonuria (PKU).
  • In May 2020, PTC Therapeutics announced that it agreed to acquire Censa Pharmaceuticals, focused on the development of CNSA-001 (sepiapterin), a clinical-stage investigational therapy for orphan metabolic diseases, including phenylketonuria (PKU) and other diseases.


Phenylketonuria Emerging Drugs

  • SYNB1618: Synlogic
  • HMI-102: Homology Medicines


Phenylketonuria Pipeline Analysis Report

  • The report provides detailed insights about emerging therapies for the Phenylketonuria treatment and the aggregate therapies developed by major pharma companies.
  • It accesses the different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for Phenylketonuria Treatment.
  • It outlines the key companies involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
  • The Phenylketonuria Pipeline Report evaluates the drugs that are 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.
  • It navigates the major collaborations (company-company collaborations and company-academia collaborations), licensing agreements, financing details, data presentation by the pharma giants, and regulatory approval in the Phenylketonuria market.

The Phenylketonuria Pipeline 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.


Scope of the Phenylketonuria Pipeline Report

Coverage– Global

Phenylketonuria Phases

  • Phenylketonuria Therapies Late-stage (Phase III) 
  • Phenylketonuria Therapies Mid-stage (Phase II)
  • Phenylketonuria Therapies Early-stage (Phase I)
  • Phenylketonuria Preclinical stage and Discovery candidates    
  • Discontinued and Inactive candidates 

Phenylketonuria Mechanism of Action

  • Phenylalanine ammonia-lyase customised replacements
  • Mixed function oxygenase modulators
  • Gastrointestinal microbiome modulators
  • SLC6A19 protein inhibitors

Phenylketonuria Molecule Types

  • Peptides
  • Monoclonal antibodies
  • Small molecules
  • Gene therapy

Phenylketonuria Route of Administration

  • Parenteral
  • Intravenous
  • Oral
  • Subcutaneous
  • Topical

Phenylketonuria Product Types

  • Monotherapy
  • Combination
  • Mono/Combination

Phenylketonuria Companies– Homology Medicines, PTC Therapeutics, Synlogic, Rubius Therapeutics, Agios Pharmaceuticals, Generation Bio, Jnana Therapeutics, and others

Phenylketonuria Therapies– SYNB1618, HMI-102, and others.


Dive deep into rich insights for Phenylketonuria Pipeline Assessment, Visit @ Phenylketonuria Pipeline Therapeutics Assessment


Table of Content

  1. Introduction
  2. Executive Summary
  3. Phenylketonuria: Overview
  4. Pipeline Therapeutics
  5. Therapeutic Assessment
  6. Phenylketonuria– DelveInsight’s Analytical Perspective
  7. Mid Stage Products (Phase III)
  8. Sepiapterin: PTC Therapeutics
  9. Mid Stage Products (Phase II)
  10. SYNB 1618: Synlogic
  11. Early Stage Products (Phase I/II)
  12. HMI-102: Homology Medicines
  13. Early Stage Products (Phase I)
  14. RTX 134: Rubius Therapeutics
  15. Preclinical Stage Products
  16. Drug name: Company name
  17. Inactive Products
  18. Phenylketonuria Key Companies
  19. Phenylketonuria Key Products
  20. Phenylketonuria- Unmet Needs
  21. Phenylketonuria- Market Drivers and Barriers
  22. Phenylketonuria- Future Perspectives and Conclusion
  23. Phenylketonuria Analyst Views
  24. Phenylketonuria Key Companies
  25. Appendix

 For further information on the Phenylketonuria Pipeline therapeutics, reach out @ Phenylketonuria Drugs


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