Hemophilia Gene Therapy - A Pipeline Analysis Report

Overview of the pipeline analysis report on hemophilia gene therapy

Hemophilia is an ideal genetic disorder and the drawbacks with the majority of the standard protein substitution therapy drugs resulted in the shift in interest towards the development of alternative treatments by gene therapy. Though the risk of developing antibodies to the viral vectors that are used to deliver the genetic material to the cells poses as a major drawback to gene therapy, the rising commercial importance of the gene therapy approach for the treatment of hemophilia resulted in a rise in the number of collaboration agreements between industries and research institutes.  For instance, the acquisition of Baxalta helped the specialty pharmaceutical company become the largest hemophilia player and the company has announced that it has generated a revenue of more than USD 870 million in 2016.

According to Technavio’s pipeline analysis report on hemophilia gene therapy, the pipeline molecules in hemophilia gene therapy are currently in the pre-clinical development stage.  SHP648, a high-performing next-generation FIX gene therapy candidate from one of the major Tier I companies Shire, is currently in the pre-clinical stage and the development of this therapeutic candidate is based on the AAV8-based gene therapy technology.

Key companies

Technavio’s analysts have identified thirteen companies that are actively involved in the development of gene therapy for hemophilia patients. Based on the companies’ revenue, this report has segregated the players into Tier 1, Tier 2, and Tier 3 companies. One of the major Tier 1 companies Alnylam Pharmaceuticals, is a leading player in the biotechnology industry and has registered an income of more than USD 410 million.