Hemophilia Gene Therapy - A Pipeline Analysis Report

This pipeline analysis report identifies uniQure, Alnylam Pharmaceuticals, BioMarin Pharmaceutical, Spark Therapeutics, Shire, and Sangamo Therapeutics as some of the companies that are actively involved in the development of gene therapy molecules for hemophilia patients. This report presents a comprehensive analysis of the therapeutic assessment by therapy (monotherapy and combination therapy), by ROA (intravenous, subcutaneous, and unspecified), and by stage and ROA (phase III, phase I/II, phase I and IND, and pre-clinical). This pipeline analysis report also provides a detailed analysis of hemophilia gene therapy based on indications such as hemophilia A and hemophilia B.

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.

The Tier 1 companies covered in this report are –

• uniQure
• Alnylam Pharmaceuticals
• BioMarin Pharmaceutical
• Spark Therapeutics
• Shire

Segmentation of hemophilia gene therapy molecules by indication

• Hemophilia A
• Hemophilia B

It has been observed that the prevalence of hemophilia A is more in the US and the countries in EU5 such as France, Germany, Italy, Spain, and United Kingdom. According to this pipeline analysis report, most of the companies that are involved in the development of gene therapy for hemophilia patients are currently focusing on developing drugs for hemophilia A.

Therapeutic assessment of the hemophilia gene therapy molecules based on therapy

• Monotherapy
• Combination therapy

Monotherapy is the utilization of a single drug to treat a disease. Our research analysts have identified that all the hemophilia pipeline drugs that are currently in the pipeline are being developed only as a monotherapy agent and most of these molecules are still in the pre-clinical phase.

Key questions answered in the report include

• What are the different stages of the pipeline molecules in investigation?
• What are the molecules in the various development stages in the hemophilia gene therapy pipeline?
• What are the key companies that are currently involved in the development of gene therapy drugs for hemophilia?
• What are the molecules discontinued from the hemophilia gene therapy pipeline?
• What are the major regulatory authorities approving drugs in various regions?

Technavio also offers customization on reports based on specific client requirement.