- Global Pharma News & Resources

Hereditary Hematological Disorders - Global Drug Development Pipeline Review 2018 -

The "Hereditary Hematological Disorders Drug Development Pipeline Review, 2018" report has been added to's offering.
This report provides an overview of the pipeline landscape for hereditary hematological disorders. It provides comprehensive information on the therapeutics under development and key players involved in therapeutic development for thalassemia, hemophilia B and sickle cell anemia, and features dormant and discontinued products.
  • Thalassemia - There are 40 products in development for this indication.
  • Hemophilia B - There are 42 products in development for this indication.
  • Sickle Cell Anemia - There are 68 products in development for this indication.

Molecular targets acted on by products in development for hereditary hematological disorders include coagulation factors, histone deacetylases and protein kinases. Companies operating in this pipeline space include Sangamo Therapeutics, Gamida Cell and Bluebird Bio.


  • Which companies are the most active within each pipeline?
  • Which pharmaceutical approaches are the most prominent at each stage of the pipeline and within each indication?
  • To what extent do universities and institutions play a role within this pipeline, compared to pharmaceutical companies?
  • What are the most important R&D milestones and data publications to have happened in this disease area?

Key Topics Covered

1 Table of Contents

2 Introduction

3 Therapeutics Development

4 Therapeutics Assessment

5 Companies Involved in Therapeutics Development

6 Dormant Projects

7 Discontinued Products

8 Product Development Milestones

Companies Featured

  • Acceleron Pharma Inc
  • Annexin Pharmaceuticals AB
  • ArQule Inc
  • Bayer AG
  • Bio Products Laboratory Ltd
  • Bioverativ Inc
  • Bluebird bio Inc
  • Bristol-Myers Squibb Co
  • CRISPR Therapeutics
  • CSL Ltd
  • Cadila Healthcare Ltd
  • Cell Source Inc
  • China Biologic Products Inc
  • Editas Medicine Inc
  • Expression Therapeutics LLC
  • Kiadis Pharma NV
  • LFB SA
  • MaxCyte Inc
  • Merck & Co Inc
  • MimeTech Srl
  • Morphogenesis Inc
  • NKT Therapeutics Inc
  • Novartis AG
  • Novo Nordisk AS
  • OPKO Biologics Ltd
  • Orphagen Pharmaceuticals Inc
  • Oryzon Genomics SA
  • Pharming Group NV
  • Sangamo Therapeutics Inc
  • Sanofi
  • Shire Plc
  • Silence Therapeutics Plc
  • UniQure NV
  • Vifor Pharma AG
  • and many more...

For more information about this report visit

View source version on

Editor Details

Last Updated: 06-Dec-2018