Fanconi Anemia Drugs in Development by Stages, Target, MoA, RoA, Molecule Type and Key Players, 2022 Update

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Fanconi Anemia Pipeline Drugs Market Report Overview

Fanconi anemia (fan-KO-nee uh-NEE-me-uh), or FA, is a rare, inherited blood disorder that leads to bone marrow failure. The disorder also is called Fanconi’s anemia. FA is a type of aplastic anemia. In aplastic anemia, the bone marrow stops making or doesn’t make enough of all three types of blood cells. Low levels of the three types of blood cells can harm many of the body’s organs, tissues, and systems. Treatment is recommended for significant cytopenias, such as hemoglobin less than 8 g/dL, platelets fewer than 500/μL.

The Fanconi Anemia pipeline drugs market research report outlays comprehensive information on the Fanconi Anemia targeted therapeutics, complete with analysis by indications, stage of development, mechanism of action (MoA), route of administration (RoA), and molecule type. The report also reviews key players involved in Fanconi Anemia targeted therapeutics development with respective active and dormant or discontinued projects.

Fanconi Anemia Pipeline Drugs Market Segmentation by Targets

The key targets in the Fanconi Anemia pipeline drugs market are Fanconi Anemia Group A Protein, Aldehyde Dehydrogenase Mitochondrial, Epidermal Growth Factor Receptor, Fanconi Anemia Group C, Fanconi Anemia Group G Protein, Receptor Tyrosine Protein Kinase ERBB 2, Receptor Tyrosine Protein Kinase ERBB 4, and Thrombopoietin Receptor.

Fanconi Anemia Pipeline Drugs Market Analysis, by Targets

Fanconi Anemia Pipeline Drugs Market Analysis, by Targets

For more insights on Fanconi Anemia pipeline drugs targets, download a free report sample

Fanconi Anemia Pipeline Drugs Market Segmentation by Mechanism of Actions

The key mechanism of actions in the Fanconi Anemia pipeline drugs market are Fanconi Anemia Group A Protein Activator, Aldehyde Dehydrogenase Mitochondrial Activator, Epidermal Growth Factor Receptor Inhibitor, Fanconi Anemia Group C Activator, Fanconi Anemia Group G Protein Activator, Receptor Tyrosine Protein Kinase ERBB 2 Inhibitor, Receptor Tyrosine Protein Kinase ERBB 4 Inhibitor, and Thrombopoietin Receptor Agonist.

Fanconi Anemia Pipeline Drugs Market Analysis, by Mechanism of Actions

Fanconi Anemia Pipeline Drugs Market Analysis, by Mechanism of Actions

For more insights on Fanconi Anemia pipeline drugs mechanism of actions, download a free report sample

Fanconi Anemia Pipeline Drugs Market Segmentation by Routes of Administration

The key routes of administration in the Fanconi Anemia pipeline drugs market are intravenous, oral, and parenteral.

Fanconi Anemia Pipeline Drugs Market Analysis, by Routes of Administration

Fanconi Anemia Pipeline Drugs Market Analysis, by Routes of Administration

For more insights on Fanconi Anemia pipeline drugs routes of administration, download a free report sample

Fanconi Anemia Pipeline Drugs Market Segmentation by Molecule Types

The key molecule types in the Fanconi Anemia pipeline drugs market are gene-modified cell therapy, small molecule, and gene therapy.

Fanconi Anemia Pipeline Drugs Market Analysis, by Molecule Types

Fanconi Anemia Pipeline Drugs Market Analysis, by Molecule Types

For more insights on Fanconi Anemia pipeline drugs molecule type, download a free report sample

Competitive Landscape

Some of the leading companies in the Fanconi Anemia pipeline drugs market are Rocket Pharmaceuticals Inc, Bellicum Pharmaceuticals Inc, Boehringer Ingelheim International GmbH, Foresee Pharmaceuticals Co Ltd, Novartis AG, and Rimedion Inc.

Fanconi Anemia Pipeline Drugs Market Analysis, by Companies

Fanconi Anemia Pipeline Drugs Market Analysis, by Companies

To know more about the leading players in the Fanconi Anemia pipeline drugs market, download a free report sample

Fanconi Anemia Pipeline Drugs Market Overview

Key Target Fanconi Anemia Group A Protein, Aldehyde Dehydrogenase Mitochondrial, Epidermal Growth Factor Receptor, Fanconi Anemia Group C, Fanconi Anemia Group G Protein, Receptor Tyrosine Protein Kinase ERBB 2, Receptor Tyrosine Protein Kinase ERBB 4, and Thrombopoietin Receptor
Key Mechanism of Actions Fanconi Anemia Group A Protein Activator, Aldehyde Dehydrogenase Mitochondrial Activator, Epidermal Growth Factor Receptor Inhibitor, Fanconi Anemia Group C Activator, Fanconi Anemia Group G Protein Activator, Receptor Tyrosine Protein Kinase ERBB 2 Inhibitor, Receptor Tyrosine Protein Kinase ERBB 4 Inhibitor, and Thrombopoietin Receptor Agonist
Key Routes of Administration Intravenous, Oral, and Parenteral
Key Molecule Types Gene-Modified Cell Therapy, Small Molecule, and Gene Therapy
Leading Companies Rocket Pharmaceuticals Inc, Bellicum Pharmaceuticals Inc, Boehringer Ingelheim International GmbH, Foresee Pharmaceuticals Co Ltd, Novartis AG, and Rimedion Inc

Scope 

  • The pipeline guide provides a snapshot of the global therapeutic landscape of Fanconi Anemia (Hematological Disorders).
  • The pipeline guide reviews pipeline therapeutics for Fanconi Anemia (Hematological 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 Fanconi Anemia (Hematological Disorders) therapeutics and enlists all their major and minor projects.
  • The pipeline guide evaluates Fanconi Anemia (Hematological 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 Fanconi Anemia (Hematological 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 Fanconi Anemia (Hematological 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 Fanconi Anemia (Hematological 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.

Table of Contents

Table of Contents

List of Tables

List of Figures

Introduction

Global Markets Direct Report Coverage

Fanconi Anemia – Overview

Fanconi Anemia – Therapeutics Development

Pipeline Overview

Pipeline by Companies

Pipeline by Universities/Institutes

Products under Development by Companies

Products under Development by Universities/Institutes

Fanconi Anemia – Therapeutics Assessment

Assessment by Target

Assessment by Mechanism of Action

Assessment by Route of Administration

Assessment by Molecule Type

Fanconi Anemia – Companies Involved in Therapeutics Development

Fanconi Anemia – Drug Profiles

Fanconi Anemia – Dormant Projects

Fanconi Anemia – Product Development Milestones

Featured News & Press Releases

Nov 04, 2021: Rocket Pharmaceuticals announces presentations highlighting lentiviral gene therapy, L102 at the 63rd American Society of Hematology (ASH) Annual Meeting

May 13, 2021: Rocket Pharmaceuticals presents positive clinical data of mozafancogene autotemcel at 24th Annual Meeting of the American Society of Gene and Cell Therapy

Dec 07, 2020: Rocket Pharmaceuticals presents positive clinical data from its Fanconi Anemia Program at the 62nd American Society of Hematology annual meeting

May 12, 2020: Rocket Pharmaceuticals presents positive updates on RP-L102 program at the 23rd annual meeting of the American Society of Gene and Cell Therapy

Feb 24, 2020: Rocket Pharmaceuticals announces publication of manuscript evaluating mosaicism in Fanconi anemia

Dec 16, 2019: Rocket Pharmaceuticals receives the European Medicines Agency PRIME eligibility for RP-L102 gene therapy for Fanconi anemia

Dec 08, 2019: Rocket Pharmaceuticals presents promising preliminary results from phase 1 trial of commercial-grade RP-L102 “Process B” for Fanconi Anemia at 61st American Society of Hematology Annual Meeting

Dec 06, 2019: Rocket pharmaceuticals announces first patient treated in global registrational phase 2 study of RP-L102 “process B” for fanconi anemia

Nov 06, 2019: Rocket Pharmaceuticals to present preliminary phase 1 data of RP-L102 “Process B” for Fanconi Anemia at the 61st American Society of Hematology Annual Meeting

Oct 24, 2019: Rocket Pharmaceuticals presents first evidence of long-term improvement and stabilization in blood counts and durable mosaicism in RP-L102 “Process A” for Fanconi Anemia

Oct 15, 2019: Rocket Pharmaceuticals announces upcoming presentation on Gene Therapy 1 for Fanconi Anemia at the European Society of Gene and Cell Therapy Annual Congress

Sep 10, 2019: Rocket Pharmaceuticals announces publication of data from phase 1/2 trial of first-generation RP-L102 for Fanconi Anemia in Nature Medicine

Sep 04, 2019: Rocket Pharmaceuticals announces registration-enabling phase 2 plans for RP-L102 gene therapy for fanconi anemia following a supportive end-of-phase 1 FDA meeting

Aug 22, 2019: Rocket Pharmaceuticals announces clearance from the Spanish Agency for Medicines and Health Products for the phase 2 registration-enabling FANCOLEN-II study of RP-L102 for Fanconi Anemia

Apr 15, 2019: Rocket Pharmaceuticals announces upcoming presentations at the American Society of Gene and Cell Therapy 2019 Annual Meeting

Appendix

Methodology

Coverage

Secondary Research

Primary Research

Expert Panel Validation

Contact Us

Disclaimer

Table

List of Tables

Number of Products under Development for Fanconi Anemia, 2022

Number of Products under Development by Companies, 2022

Number of Products under Development by Universities/Institutes, 2022

Products under Development by Companies, 2022

Products under Development by Universities/Institutes, 2022

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

Fanconi Anemia – Pipeline by Bellicum Pharmaceuticals Inc, 2022

Fanconi Anemia – Pipeline by Boehringer Ingelheim International GmbH, 2022

Fanconi Anemia – Pipeline by Foresee Pharmaceuticals Co Ltd, 2022

Fanconi Anemia – Pipeline by Novartis AG, 2022

Fanconi Anemia – Pipeline by Rimedion Inc, 2022

Fanconi Anemia – Pipeline by Rocket Pharmaceuticals Inc, 2022

Fanconi Anemia – Dormant Projects, 2022

Figures

List of Figures

Number of Products under Development for Fanconi Anemia, 2022

Number of Products under Development by Companies, 2022

Number of Products under Development by Universities/Institutes, 2022

Number of Products by Top 10 Targets, 2022

Number of Products by Stage and Top 10 Targets, 2022

Number of Products by Top 10 Mechanism of Actions, 2022

Number of Products by Stage and Top 10 Mechanism of Actions, 2022

Number of Products by Top 10 Routes of Administration, 2022

Number of Products by Stage and Top 10 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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