Table of Content


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
Bellicum Pharmaceuticals Inc
Boehringer Ingelheim Intertiol GmbH
Elixirgen Therapeutics Inc
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
mozafancogene autotemcel - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
rivogenlecleucel - 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 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 prelimiry 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 registratiol phase 2 study of RP-L102 “process B” for Fanconi anemia
Nov 06, 2019: Rocket Pharmaceuticals to present prelimiry 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 ture Medicine
Sep 04, 2019: Rocket Pharmaceuticals announces registration-ebling 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-ebling 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.
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer



List of Figures

List of Figures
Number of Products under Development for Fanconi Anemia, 2021
Number of Products under Development by Companies, 2021
Number of Products under Development by Universities/Institutes, 2021
Number of Products by Top 10 Targets, 2021
Number of Products by Stage and Top 10 Targets, 2021
Number of Products by Top 10 Mechanism of Actions, 2021
Number of Products by Stage and Top 10 Mechanism of Actions, 2021
Number of Products by Routes of Administration, 2021
Number of Products by Stage and Routes of Administration, 2021
Number of Products by Molecule Types, 2021
Number of Products by Stage and Molecule Types, 2021

List of Tables

List of Tables
Number of Products under Development for Fanconi Anemia, 2021
Number of Products under Development by Companies, 2021
Number of Products under Development by Universities/Institutes, 2021
Products under Development by Companies, 2021
Products under Development by Universities/Institutes, 2021
Number of Products by Stage and Target, 2021
Number of Products by Stage and Mechanism of Action, 2021
Number of Products by Stage and Route of Administration, 2021
Number of Products by Stage and Molecule Type, 2021
Fanconi Anemia - Pipeline by Bellicum Pharmaceuticals Inc, 2021
Fanconi Anemia - Pipeline by Boehringer Ingelheim International GmbH, 2021
Fanconi Anemia - Pipeline by Elixirgen Therapeutics Inc, 2021
Fanconi Anemia - Pipeline by Foresee Pharmaceuticals Co Ltd, 2021
Fanconi Anemia - Pipeline by Novartis AG, 2021
Fanconi Anemia - Pipeline by Rimedion Inc, 2021
Fanconi Anemia - Pipeline by Rocket Pharmaceuticals Inc, 2021
Fanconi Anemia - Dormant Projects, 2021