Fanconi Anemia - Pipeline Review, H1 2020
Fanconi Anemia - Pipeline Review, H1 2020
SUMMARY
Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Fanconi Anemia – Pipeline Review, H1 2020, provides an overview of the Fanconi Anemia (Hematological Disorders) pipeline landscape.
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/?L.
REPORT HIGHLIGHTS
Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Fanconi Anemia – Pipeline Review, H1 2020, provides comprehensive information on the therapeutics under development for Fanconi Anemia (Hematological Disorders), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The guide covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases.
The Fanconi Anemia (Hematological Disorders) pipeline guide also reviews of key players involved in therapeutic development for Fanconi Anemia and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies/Universities/Institutes, the molecules developed by Companies in Phase II, Phase I, Preclinical and Discovery stages are 2, 1, 3 and 2 respectively. Similarly, the Universities portfolio in Phase II and Preclinical stages comprises 3 and 2 molecules, respectively.
Fanconi Anemia (Hematological Disorders) pipeline guide helps in identifying and tracking emerging players in the market and their portfolios, enhances decision making capabilities and helps to create effective counter strategies to gain competitive advantage. The guide is built using data and information sourced from Global Markets Direct’s proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.
Note: Certain content/sections in the pipeline guide may be removed or altered based on the availability and relevance of data.
SCOPE
SUMMARY
Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Fanconi Anemia – Pipeline Review, H1 2020, provides an overview of the Fanconi Anemia (Hematological Disorders) pipeline landscape.
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/?L.
REPORT HIGHLIGHTS
Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Fanconi Anemia – Pipeline Review, H1 2020, provides comprehensive information on the therapeutics under development for Fanconi Anemia (Hematological Disorders), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The guide covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases.
The Fanconi Anemia (Hematological Disorders) pipeline guide also reviews of key players involved in therapeutic development for Fanconi Anemia and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies/Universities/Institutes, the molecules developed by Companies in Phase II, Phase I, Preclinical and Discovery stages are 2, 1, 3 and 2 respectively. Similarly, the Universities portfolio in Phase II and Preclinical stages comprises 3 and 2 molecules, respectively.
Fanconi Anemia (Hematological Disorders) pipeline guide helps in identifying and tracking emerging players in the market and their portfolios, enhances decision making capabilities and helps to create effective counter strategies to gain competitive advantage. The guide is built using data and information sourced from Global Markets Direct’s proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.
Note: Certain content/sections in the pipeline guide may be removed or altered based on the availability and relevance of data.
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)
- 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.
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
Boehringer Ingelheim International GmbH
Elixirgen Therapeutics LLC
Foresee Pharmaceuticals Co Ltd
Novartis AG
Rimedion Inc
Rocket Pharmaceuticals Inc
Fanconi Anemia - Drug Profiles
afatinib dimaleate - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
eltrombopag olamine - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
EXG-34217 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Fancalen - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
FP-045 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapies to Activate FANCC and FANCG for Fanconi Anemia - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy 1 for Fanconi Anemia - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy to Activate FANC-A for Fanconi Anemia - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy to Activate FANCA for Fanconi Anemia - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy to Activate FANCA Protein for Fanconi Anemia - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
JP-4039 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
RPL-102 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Small Molecules for Fanconi Anemia - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Fanconi Anemia - Dormant Projects
Fanconi Anemia - Product Development Milestones
Featured News & Press Releases
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
Mar 12, 2019: Rocket Pharmaceuticals announces first patient dosed in phase 1 clinical trial of RP-L102 in the U.S.
Nov 27, 2018: Rocket Pharmaceuticals receives FDA Regenerative Medicine Advanced Therapy (RMAT) and Fast Track Designations for RP-L102 Gene Therapy for Fanconi Anemia
Nov 07, 2018: Rocket Pharmaceuticals announces FDA clearance of IND application for RP-L102 gene therapy for fanconi anemia
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer
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
Boehringer Ingelheim International GmbH
Elixirgen Therapeutics LLC
Foresee Pharmaceuticals Co Ltd
Novartis AG
Rimedion Inc
Rocket Pharmaceuticals Inc
Fanconi Anemia - Drug Profiles
afatinib dimaleate - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
eltrombopag olamine - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
EXG-34217 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Fancalen - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
FP-045 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapies to Activate FANCC and FANCG for Fanconi Anemia - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy 1 for Fanconi Anemia - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy to Activate FANC-A for Fanconi Anemia - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy to Activate FANCA for Fanconi Anemia - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy to Activate FANCA Protein for Fanconi Anemia - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
JP-4039 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
RPL-102 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Small Molecules for Fanconi Anemia - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Fanconi Anemia - Dormant Projects
Fanconi Anemia - Product Development Milestones
Featured News & Press Releases
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
Mar 12, 2019: Rocket Pharmaceuticals announces first patient dosed in phase 1 clinical trial of RP-L102 in the U.S.
Nov 27, 2018: Rocket Pharmaceuticals receives FDA Regenerative Medicine Advanced Therapy (RMAT) and Fast Track Designations for RP-L102 Gene Therapy for Fanconi Anemia
Nov 07, 2018: Rocket Pharmaceuticals announces FDA clearance of IND application for RP-L102 gene therapy for fanconi anemia
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer
LIST OF TABLES
Number of Products under Development for Fanconi Anemia, H1 2020
Number of Products under Development by Companies, H1 2020
Number of Products under Development by Universities/Institutes, H1 2020
Products under Development by Companies, H1 2020
Products under Development by Universities/Institutes, H1 2020
Number of Products by Stage and Target, H1 2020
Number of Products by Stage and Mechanism of Action, H1 2020
Number of Products by Stage and Route of Administration, H1 2020
Number of Products by Stage and Molecule Type, H1 2020
Fanconi Anemia - Pipeline by Boehringer Ingelheim International GmbH, H1 2020
Fanconi Anemia - Pipeline by Elixirgen Therapeutics LLC, H1 2020
Fanconi Anemia - Pipeline by Foresee Pharmaceuticals Co Ltd, H1 2020
Fanconi Anemia - Pipeline by Novartis AG, H1 2020
Fanconi Anemia - Pipeline by Rimedion Inc, H1 2020
Fanconi Anemia - Pipeline by Rocket Pharmaceuticals Inc, H1 2020
Fanconi Anemia - Dormant Projects, H1 2020
Number of Products under Development for Fanconi Anemia, H1 2020
Number of Products under Development by Companies, H1 2020
Number of Products under Development by Universities/Institutes, H1 2020
Products under Development by Companies, H1 2020
Products under Development by Universities/Institutes, H1 2020
Number of Products by Stage and Target, H1 2020
Number of Products by Stage and Mechanism of Action, H1 2020
Number of Products by Stage and Route of Administration, H1 2020
Number of Products by Stage and Molecule Type, H1 2020
Fanconi Anemia - Pipeline by Boehringer Ingelheim International GmbH, H1 2020
Fanconi Anemia - Pipeline by Elixirgen Therapeutics LLC, H1 2020
Fanconi Anemia - Pipeline by Foresee Pharmaceuticals Co Ltd, H1 2020
Fanconi Anemia - Pipeline by Novartis AG, H1 2020
Fanconi Anemia - Pipeline by Rimedion Inc, H1 2020
Fanconi Anemia - Pipeline by Rocket Pharmaceuticals Inc, H1 2020
Fanconi Anemia - Dormant Projects, H1 2020
LIST OF FIGURES
Number of Products under Development for Fanconi Anemia, H1 2020
Number of Products under Development by Companies, H1 2020
Number of Products under Development by Universities/Institutes, H1 2020
Number of Products by Targets, H1 2020
Number of Products by Stage and Targets, H1 2020
Number of Products by Mechanism of Actions, H1 2020
Number of Products by Stage and Mechanism of Actions, H1 2020
Number of Products by Routes of Administration, H1 2020
Number of Products by Stage and Routes of Administration, H1 2020
Number of Products by Molecule Types, H1 2020
Number of Products by Stage and Molecule Types, H1 2020
COMPANIES MENTIONED
Boehringer Ingelheim International GmbH
Elixirgen Therapeutics LLC
Foresee Pharmaceuticals Co Ltd
Novartis AG
Rimedion Inc
Rocket Pharmaceuticals Inc
Number of Products under Development for Fanconi Anemia, H1 2020
Number of Products under Development by Companies, H1 2020
Number of Products under Development by Universities/Institutes, H1 2020
Number of Products by Targets, H1 2020
Number of Products by Stage and Targets, H1 2020
Number of Products by Mechanism of Actions, H1 2020
Number of Products by Stage and Mechanism of Actions, H1 2020
Number of Products by Routes of Administration, H1 2020
Number of Products by Stage and Routes of Administration, H1 2020
Number of Products by Molecule Types, H1 2020
Number of Products by Stage and Molecule Types, H1 2020
COMPANIES MENTIONED
Boehringer Ingelheim International GmbH
Elixirgen Therapeutics LLC
Foresee Pharmaceuticals Co Ltd
Novartis AG
Rimedion Inc
Rocket Pharmaceuticals Inc
