Fanconi Anemia – Pipeline Review, H2 2020

Global Markets Direct’s, ‘Fanconi Anemia – Pipeline Review, H2 2020’, provides an overview of the Fanconi Anemia pipeline landscape.

The report provides comprehensive information on the therapeutics under development for Fanconi Anemia, 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 Fanconi Anemia and features dormant and discontinued projects.

Global Markets Direct’s report features investigational drugs from across globe covering over 20 therapy areas and nearly 3,000 indications. The report 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. Drug profiles featured in the report undergoes periodic review following a stringent set of processes to ensure that all the profiles are updated with the latest set of information. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.

The report 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.

Note*: Certain sections in the report may be removed or altered based on the availability and relevance of data.

Scope

– The report provides a snapshot of the global therapeutic landscape of Fanconi Anemia

– The report reviews pipeline therapeutics for Fanconi Anemia by companies and universities/research institutes based on information derived from company and industry-specific sources

– The report covers pipeline products based on various stages of development ranging from pre-registration till discovery and undisclosed stages

– The report features descriptive drug profiles for the pipeline products which includes, product description, descriptive MoA, R&D brief, licensing and collaboration details & other developmental activities

– The report reviews key players involved Fanconi Anemia therapeutics and enlists all their major and minor projects

– The report assesses Fanconi Anemia therapeutics based on drug target, mechanism of action (MoA), route of administration (RoA) and molecule type

– The report summarizes all the dormant and discontinued pipeline projects

– The report reviews latest news related to pipeline therapeutics for Fanconi Anemia

Reasons to buy

- Gain strategically significant competitor information, analysis, and insights to formulate effective R&D strategies

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

- Identify and understand important and diverse types of therapeutics under development for Fanconi Anemia

- Identify potential new clients or partners in the target demographic

- Develop strategic initiatives by understanding the focus areas of leading companies

- Plan mergers and acquisitions effectively by identifying key players and it’s most promising pipeline therapeutics

- Devise corrective measures for pipeline projects by understanding Fanconi Anemia 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

- Modify the therapeutic portfolio by identifying discontinued projects and understanding the factors that drove them from pipeline

Companies mentioned

Boehringer Ingelheim International GmbH

Elixirgen Therapeutics LLC

Foresee Pharmaceuticals Co Ltd

Novartis AG

Rimedion Inc

Rocket Pharmaceuticals Inc

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

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

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

List of Figures

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

    Pricing

Discounts available for multiple purchases.

reportstore@globalmarketsdirect.com
+44 20 7947 2745

Saved reports