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Hemophilia Gene Therapy Market - Coding For Improved Quality of Life.

Hemophilia is a genetic bleeding disorder in which an individual lacks or has low levels of proteins called clotting factors. There are around 13 types of clotting factors that work with blood platelets, which are necessary for clotting process to initiate. There are three forms of hemophilia - A, B, and C. Hemophilia A is the most common form and is caused due to deficiency in clotting factor VIII. Hemophilia B occurs due to deficiency of clotting factor IX and Hemophilia C is caused due to clotting factor XI deficiency. Hemophilia is incurable with current therapeutic options, which only reduces symptoms such as spontaneous bleeding in muscles and joints as well as increased risk for intracranial hemorrhage. These treatment options lasts only for a day and is costly. As against conventional clotting factor replacement therapy, gene therapy is expected to offer sustainable cure for hemophilia by correcting defective gene sequence (F8 or F9 gene) that codes for clotting factor VIII or IX in the patient’s body.

In this therapy, recombinant Adeno - associated virus is most commonly utilized to deliver a codon optimized version of the clotting factors genes (VIII or IX) to patients affected by hemophilia. This gene is shortened by deleting a discrete portion of the gene to better fit the coding sequence into the viral vector. There is dose dependent gene expression level observed which means genetic expression for clotting factor increases with increased dose of gene therapy.

The global hemophilia gene therapy market was valued at US$ 0.00 million in 2017 and is expected to witness a CAGR of 56.0% over the forecast period (2018 – 2026).

Figure 1. Global Hemophilia Gene Therapy Market Value (US$ Mn), by Region, 2017

Source – Coherent Market Insights Report

Increasing hemophilia patient base worldwide especially in developed countries is expected to drive growth of the hemophilia gene therapy market

High prevalence of hemophilia is increasing demand for sustainable treatment of the disease, which is projected to drive the market growth over the forecast period. According to the National Hemophilia Foundation factsheet 2018, hemophilia affected over 400,000 individuals worldwide. Moreover, according to same source, an estimated 75% of patients suffering from hemophilia have no access to treatment or receive inadequate treatment. According to the Centers for Disease Control and Prevention (CDC) report, Hemophilia A occurs in 1 out of 5,000 live male births. It is around four times more common than Hemophilia B. Few effective treatments available for hemophilia require lifetime infusion of expensive drugs manufactured through recombinant biotechnology or from human plasma.

Gene therapy may prove to be panacea in this scenario of increasing prevalence rate of hemophilia worldwide.

Figure 2. Global Hemophilia Gene Therapy Market, Product Pipeline, 2017

Company Product Indication Clinical Stage BioMarin Pharmaceuticals Valoctocogene roxaparvovec Hemophilia A Phase III Pfizer Fidanacogene elaparvovec Hemophilia B Phase III uniQure AMT-061 Hemophilia B Phase III Sangamo Therapeutics SB-525 Hemophilia A Phase I/II Spark Therapeutics SPK-8011 Hemophilia A Phase I/II Shire PLC SHP 654 Hemophilia A Phase I

Source – Clinicaltrials.gov, Secondary Analysis by Coherent Market Insights

Strong gene-based product pipeline for hemophilia treatment is expected to drive the hemophilia gene therapy market growth

Gene therapy for hemophilia has strong product pipeline, which is expected to enter the market in the near future. This is expected to drive the market growth, as it would result in availability of new treatment options for management of hemophilia. By Januarys 2019, over 15 investigational gene therapy are under the clinical development stage. In December 2018, Spark Therapeutics announced updated preliminary data for the ongoing phase 1/2 clinical trial of investigational SPK-8011 in hemophilia A.

Current therapies for treating hemophilia are only symptomatic management of the disease by administering clotting factors XIII or IX. Furthermore, there are serious adverse effects associated with plasma derived products, and limited access to treatment. Gene therapy may address these issues to certain level. BioMarin Pharmaceutical’s gene therapy product Valoctocogene roxaparvovec for hemophilia A is in phase III of clinical development in 2019.

Regulatory bodies such as the U.S. Food and Drug Administration (FDA) are supporting the product development for gene therapy by allotting breakthrough designation status to investigational gene therapy. For instance, in 2017, the U.S. FDA granted breakthrough therapy designation to valoctocogene roxaparvovec, which is an Adeno -associated virus 5 factor VIII vector designed to restore factor VIII plasma concentrations in patients with hemophilia A.

Key players operating in the global hemophilia gene therapy market include BioMarin Pharmaceuticals, Inc., Spark Therapeutics, Pfizer, Inc., UniQure NV, Ultragenyx Pharmaceutical, Shire PLC Sangamo Therapeutics, Inc., and Freeline Therapeutics.