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
Boehringer Ingelheim Intertiol 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 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.
Nov 27, 2018: Rocket Pharmaceuticals receives FDA Regenerative Medicine Advanced Therapy (RMAT) and Fast Track Desigtions 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 Figures


Number of Products under Development for Fanconi Anemia, H2 2020
Number of Products under Development by Companies, H2 2020
Number of Products under Development by Universities/Institutes, H2 2020
Number of Products by Top 10 Targets, H2 2020
Number of Products by Stage and Top 10 Targets, H2 2020
Number of Products by Top 10 Mechanism of Actions, H2 2020
Number of Products by Stage and Top 10 Mechanism of Actions, H2 2020
Number of Products by Top 10 Routes of Administration, H2 2020
Number of Products by Stage and Top 10 Routes of Administration, H2 2020
Number of Products by Top 10 Molecule Types, H2 2020
Number of Products by Stage and Top 10 Molecule Types, H2 2020


List of Tables


Number of Products under Development for Fanconi Anemia, H2 2020
Number of Products under Development by Companies, H2 2020
Number of Products under Development by Universities/Institutes, H2 2020
Products under Development by Companies, H2 2020
Products under Development by Universities/Institutes, H2 2020
Number of Products by Stage and Target, H2 2020
Number of Products by Stage and Mechanism of Action, H2 2020
Number of Products by Stage and Route of Administration, H2 2020
Number of Products by Stage and Molecule Type, H2 2020
Fanconi Anemia - Pipeline by Boehringer Ingelheim International GmbH, H2 2020
Fanconi Anemia - Pipeline by Elixirgen Therapeutics LLC, H2 2020
Fanconi Anemia - Pipeline by Foresee Pharmaceuticals Co Ltd, H2 2020
Fanconi Anemia - Pipeline by Novartis AG, H2 2020
Fanconi Anemia - Pipeline by Rimedion Inc, H2 2020
Fanconi Anemia - Pipeline by Rocket Pharmaceuticals Inc, H2 2020
Fanconi Anemia - Dormant Projects, H2 2020