Phenylketonuria (PKU) Drugs in Development by Stages, Target, MoA, RoA, Molecule Type and Key Players, 2022 Update

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Published: March 31, 2022 Report Code: GMDGDHC22070IDB-ST

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Phenylketonuria (PKU) pipeline market research report provides comprehensive information on the therapeutics under development for phenylketonuria (PKU), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The report also covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases. Additionally, the report provides an overview of key players involved in therapeutic development for Phenylketonuria (PKU) and features dormant and discontinued projects.

Key Targets in the Phenylketonuria Pipeline Market

The key targets in the phenylketonuria pipeline market are Phenylalanine 4 Hydroxylase, Phenylalanine Ammonia Lyase, and Tetrahydrobiopterin. Phenylalanine 4 Hydroxylase has the highest number of pipeline products.

Phenylketonuria Pipeline Market, by Targets

Phenylketonuria Pipeline Market, by Targets

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Key MoA in the Phenylketonuria Pipeline Market

The key MoA in the phenylketonuria pipeline market are Phenylalanine 4 Hydroxylase Activator, Phenylalanine 4 Hydroxylase Replacement, Phenylalanine Ammonia Lyase Replacement, and Tetrahydrobiopterin Replacement. Phenylalanine 4 Hydroxylase Activator has the highest number of pipeline products.

Phenylketonuria Pipeline Market, by MoA

Phenylketonuria Pipeline Market, by MoA

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Key RoA in the Phenylketonuria Pipeline Market

The key RoA in the phenylketonuria pipeline market are oral, intravenous, and subcutaneous. Oral has the highest number of pipeline products.

Phenylketonuria Pipeline Market, by RoA

Phenylketonuria Pipeline Market, by RoA

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Key Molecule Types in the Phenylketonuria Pipeline Market

The key molecule types in the phenylketonuria pipeline market are gene therapy, small molecule, biologic, recombinant enzyme, oligonucleotide, cell therapy, gene-modified cell therapy, polymer, and protein. Gene therapy has the highest number of pipeline products.

Phenylketonuria Pipeline Market, by Molecule Type

Phenylketonuria Pipeline Market, by Molecule Type

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Key Companies in the Phenylketonuria Pipeline Market

The key companies in the phenylketonuria pipeline market are Synlogic Inc, Homology Medicines Inc, Agios Pharmaceuticals Inc, American Gene Technologies International Inc, BioMarin Pharmaceutical Inc, Castle Creek Biosciences Inc, and CommBio Therapeutics Co Ltd. Synlogic Inc has the highest number of pipeline products.

Phenylketonuria Pipeline Market, by Companies

Phenylketonuria Pipeline Market, by Companies

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Market report overview

Key targets Phenylalanine 4 Hydroxylase, Phenylalanine Ammonia Lyase, and Tetrahydrobiopterin
Key MoA Phenylalanine 4 Hydroxylase Activator, Phenylalanine 4 Hydroxylase Replacement, Phenylalanine Ammonia Lyase Replacement, and Tetrahydrobiopterin Replacement
Key RoA Oral, Intravenous, and Subcutaneous
Key molecule types Gene Therapy, Small Molecule, Biologic, Recombinant Enzyme, Oligonucleotide, Cell Therapy, Gene-Modified Cell Therapy, Polymer, and Protein
Key companies Synlogic Inc, Homology Medicines Inc, Agios Pharmaceuticals Inc, American Gene Technologies International Inc, BioMarin Pharmaceutical Inc, Castle Creek Biosciences Inc, and CommBio Therapeutics Co Ltd

Scope

  • The pipeline guide provides a snapshot of the global therapeutic landscape of Phenylketonuria (PKU) (Metabolic Disorders).
  • The pipeline guide reviews pipeline therapeutics for Phenylketonuria (PKU) (Metabolic Disorders) by companies and universities/research institutes based on information derived from company and industry-specific sources.
  • The pipeline guide covers pipeline products based on several stages of development ranging from pre-registration till discovery and undisclosed stages.
  • The pipeline guide features descriptive drug profiles for the pipeline products which comprise, product description, descriptive licensing and collaboration details, R&D brief, MoA & other developmental activities.
  • The pipeline guide reviews key companies involved in Phenylketonuria (PKU) (Metabolic Disorders) therapeutics and enlists all their major and minor projects.
  • The pipeline guide evaluates Phenylketonuria (PKU) (Metabolic Disorders) therapeutics based on mechanism of action (MoA), drug target, route of administration (RoA) and molecule type.
  • The pipeline guide encapsulates all the dormant and discontinued pipeline projects.
  • The pipeline guide reviews latest news related to pipeline therapeutics for Phenylketonuria (PKU) (Metabolic Disorders)

Reasons to Buy

  • Procure strategically important competitor information, analysis, and insights to formulate effective R&D strategies.

  • Recognize emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage.

  • Find and recognize significant and varied types of therapeutics under development for Phenylketonuria (PKU) (Metabolic Disorders).

  • Classify potential new clients or partners in the target demographic.

  • Develop tactical initiatives by understanding the focus areas of leading companies.

  • Plan mergers and acquisitions meritoriously by identifying key players and it’s most promising pipeline therapeutics.

  • Formulate corrective measures for pipeline projects by understanding Phenylketonuria (PKU) (Metabolic Disorders) pipeline depth and focus of Indication therapeutics.

  • Develop and design in-licensing and out-licensing strategies by identifying prospective partners with the most attractive projects to enhance and expand business potential and scope.

  • Adjust the therapeutic portfolio by recognizing discontinued projects and understand from the know-how what drove them from pipeline.

Agios Pharmaceuticals Inc
American Gene Technologies International Inc
BioMarin Pharmaceutical Inc
Castle Creek Biosciences Inc
CommBio Therapeutics Co Ltd
Evox Therapeutics Ltd
Generation Bio Co
Hepatx Corp
Homology Medicines Inc
iECURE
Jnana Therapeutics Inc
Maze Therapeutics Inc
MipSalus ApS
Moderna Inc
Nestle Health Science
Peg-Bio BioPharma Co Ltd
Pluvia AS
Prosit Sole Biotechnology (Beijing) Co Ltd
PTC Therapeutics Inc
Relief Therapeutics Holding AG
SOM Biotech SL
Synlogic Inc
Ultragenyx Pharmaceutical Inc
Vera Therapeutics Inc

Table of Contents

Table of Contents

List of Tables

List of Figures

Introduction

Global Markets Direct Report Coverage

Phenylketonuria (PKU) – Overview

Phenylketonuria (PKU) – Therapeutics Development

Pipeline Overview

Pipeline by Companies

Products under Development by Companies

Phenylketonuria (PKU) – Therapeutics Assessment

Assessment by Target

Assessment by Mechanism of Action

Assessment by Route of Administration

Assessment by Molecule Type

Phenylketonuria (PKU) – Companies Involved in Therapeutics Development

Agios Pharmaceuticals Inc

American Gene Technologies International Inc

BioMarin Pharmaceutical Inc

Castle Creek Biosciences Inc

CommBio Therapeutics Co Ltd

Evox Therapeutics Ltd

Generation Bio Co

Hepatx Corp

Homology Medicines Inc

iECURE

Jnana Therapeutics Inc

Maze Therapeutics Inc

MipSalus ApS

Moderna Inc

Nestle Health Science

Peg-Bio BioPharma Co Ltd

Pluvia AS

Prosit Sole Biotechnology (Beijing) Co Ltd

PTC Therapeutics Inc

Relief Therapeutics Holding AG

SOM Biotech SL

Synlogic Inc

Ultragenyx Pharmaceutical Inc

Vera Therapeutics Inc

Phenylketonuria (PKU) – Drug Profiles

AGT-324 – Drug Profile

Product Description

Mechanism Of Action

History of Events

APROD-031 – Drug Profile

Product Description

Mechanism Of Action

History of Events

CDX-6114 – Drug Profile

Product Description

Mechanism Of Action

History of Events

Gene Therapy for Phenylketonuria – Drug Profile

Product Description

Mechanism Of Action

Gene Therapy to Target Phenylalanine Hydroxylase for Phenylketonuria – Drug Profile

Product Description

Mechanism Of Action

History of Events

Gene-Modified Cell Therapy to Activate PAH for Phenylketonuria – Drug Profile

Product Description

Mechanism Of Action

HMI-103 – Drug Profile

Product Description

Mechanism Of Action

History of Events

HX-003 – Drug Profile

Product Description

Mechanism Of Action

JNT-517 – Drug Profile

Product Description

Mechanism Of Action

mRNA-3283 – Drug Profile

Product Description

Mechanism Of Action

Oligonucleotides to Activate PAH for Phenylketonuria – Drug Profile

Product Description

Mechanism Of Action

History of Events

PAH – Drug Profile

Product Description

Mechanism Of Action

pegvaliase – Drug Profile

Product Description

Mechanism Of Action

History of Events

Phelimin – Drug Profile

Product Description

Mechanism Of Action

History of Events

Phenylketonuria – Drug Profile

Product Description

Mechanism Of Action

PJ-008 – Drug Profile

Product Description

Mechanism Of Action

PKU – Drug Profile

Product Description

Mechanism Of Action

PSP-007 – Drug Profile

Product Description

Mechanism Of Action

sepiapterin – Drug Profile

Product Description

Mechanism Of Action

History of Events

Small Molecule for Phenylketonuria – Drug Profile

Product Description

Mechanism Of Action

Small Molecules to Activate Phenylalanine Hydroxylase for Phenylketonuria – Drug Profile

Product Description

Mechanism Of Action

SOM-1311 – Drug Profile

Product Description

Mechanism Of Action

SYNB-1618 – Drug Profile

Product Description

Mechanism Of Action

History of Events

SYNB-1934 – Drug Profile

Product Description

Mechanism Of Action

History of Events

UX-501 – Drug Profile

Product Description

Mechanism Of Action

History of Events

Phenylketonuria (PKU) – Dormant Projects

Phenylketonuria (PKU) – Discontinued Products

Phenylketonuria (PKU) – Product Development Milestones

Featured News & Press Releases

Feb 23, 2022: Jnana Therapeutics to advance JNT-517 as development candidate and potential first-in-class oral treatment for PKU

Nov 22, 2021: Synlogic presents data demonstrating reductions in plasma phenylalanine levels in patients with phenylketonuria treated with SYNB1934

Nov 22, 2021: Synlogic presents data demonstrating reductions in plasma phenylalanine levels in patients with phenylketonuria treated with SYNB1618

Nov 10, 2021: Synlogic announces presentation of data on SYNB1934 at 14th International Congress of Inborn Errors of Metabolism Meeting

Nov 10, 2021: Synlogic announces presentation of data on SYNB1618 at 14th International Congress of Inborn Errors of Metabolism Meeting

Oct 28, 2021: Synlogic announces Nature Communications publication highlighting use of synthetic biotic platform to optimize therapies for phenylketonuria

Oct 12, 2021: Homology Medicines announces world’s first gene editing clinical trial for PKU

Sep 11, 2021: Synlogic presents at Global PKU Patient Meeting

Jul 22, 2021: Synlogic publishes papers in Nature journals demonstrating proof-of-mechanism and potential of synthetic biotic platform for the treatment of phenylketonuria (PKU)

Jul 15, 2021: Synlogic provides update on SYNB1618 , a next-generation strain for the treatment of Phenylketonuria (PKU)

Jul 15, 2021: Synlogic announces initiation of phase 1 study of SYNB1934, a next-generation strain for the treatment of Phenylketonuria (PKU)

Jun 15, 2021: Synlogic presents additional preclinical data on therapeutic candidate SYNB1934 for Phenylketonuria (PKU) at Synthetic Biology: Engineering, Evolution & Design (SEED) Conference

Apr 13, 2021: Synlogic presents data demonstrating activity of a solid oral formulation of SYNB1618 at American College of Medical Genetics (ACMG) annual meeting

Oct 07, 2020: BioMarin, pioneer in rare disease treatments for Phenylketonuria (PKU), receives FDA approval of label expansion to allow maximum dose of 60 mg for Palynziq (pegvaliase-pqpz) injection for treatment of adults with PKU

Mar 26, 2020: FDA grants Orphan Drug Designation to APR-OD031 for the treatment of phenylketonuria

Appendix

Methodology

Coverage

Secondary Research

Primary Research

Expert Panel Validation

Contact Us

Disclaimer

Table

List of Tables

Number of Products under Development for Phenylketonuria (PKU), 2022

Number of Products under Development by Companies, 2022

Products under Development by Companies, 2022

Products under Development by Companies, 2022 (Contd..1)

Number of Products by Stage and Target, 2022

Number of Products by Stage and Mechanism of Action, 2022

Number of Products by Stage and Route of Administration, 2022

Number of Products by Stage and Molecule Type, 2022

Phenylketonuria (PKU) – Pipeline by Agios Pharmaceuticals Inc, 2022

Phenylketonuria (PKU) – Pipeline by American Gene Technologies International Inc, 2022

Phenylketonuria (PKU) – Pipeline by BioMarin Pharmaceutical Inc, 2022

Phenylketonuria (PKU) – Pipeline by Castle Creek Biosciences Inc, 2022

Phenylketonuria (PKU) – Pipeline by CommBio Therapeutics Co Ltd, 2022

Phenylketonuria (PKU) – Pipeline by Evox Therapeutics Ltd, 2022

Phenylketonuria (PKU) – Pipeline by Generation Bio Co, 2022

Phenylketonuria (PKU) – Pipeline by Hepatx Corp, 2022

Phenylketonuria (PKU) – Pipeline by Homology Medicines Inc, 2022

Phenylketonuria (PKU) – Pipeline by iECURE, 2022

Phenylketonuria (PKU) – Pipeline by Jnana Therapeutics Inc, 2022

Phenylketonuria (PKU) – Pipeline by Maze Therapeutics Inc, 2022

Phenylketonuria (PKU) – Pipeline by MipSalus ApS, 2022

Phenylketonuria (PKU) – Pipeline by Moderna Inc, 2022

Phenylketonuria (PKU) – Pipeline by Nestle Health Science, 2022

Phenylketonuria (PKU) – Pipeline by Peg-Bio BioPharma Co Ltd, 2022

Phenylketonuria (PKU) – Pipeline by Pluvia AS, 2022

Phenylketonuria (PKU) – Pipeline by Prosit Sole Biotechnology (Beijing) Co Ltd, 2022

Phenylketonuria (PKU) – Pipeline by PTC Therapeutics Inc, 2022

Phenylketonuria (PKU) – Pipeline by Relief Therapeutics Holding AG, 2022

Phenylketonuria (PKU) – Pipeline by SOM Biotech SL, 2022

Phenylketonuria (PKU) – Pipeline by Synlogic Inc, 2022

Phenylketonuria (PKU) – Pipeline by Ultragenyx Pharmaceutical Inc, 2022

Phenylketonuria (PKU) – Pipeline by Vera Therapeutics Inc, 2022

Phenylketonuria (PKU) – Dormant Projects, 2022

Phenylketonuria (PKU) – Discontinued Products, 2022

Figures

List of Figures

Number of Products under Development for Phenylketonuria (PKU), 2022

Number of Products under Development by Companies, 2022

Number of Products by Targets, 2022

Number of Products by Stage and Targets, 2022

Number of Products by Mechanism of Actions, 2022

Number of Products by Stage and Mechanism of Actions, 2022

Number of Products by Routes of Administration, 2022

Number of Products by Stage and Routes of Administration, 2022

Number of Products by Top 10 Molecule Types, 2022

Number of Products by Stage and Top 10 Molecule Types, 2022

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