Experimental Treatments for Hemophilia

Overview

There is no cure for hemophilia, but there are treatments available that can help manage the disease and maintain quality of life. In addition to existing approved treatments, several experimental therapies are being evaluated to identify a more long-term solution for patients with hemophilia. These include gene therapy, extended half-life treatments, and other approaches.

CRISPR/Cas9

CRISPR/Cas9 is a gene-editing technology that can be used to alter the genetic sequence of a specific gene inside a cell. Treatments using this approach have shown promise in preclinical models of both hemophilia A and hemophilia B, and several companies are working to develop therapies for hemophilia based on CRISPR/Cas9.

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Giroctocogene fitelparvovec

Giroctocogene fitelparvovec is an experimental gene therapy currently being developed by Pfizer for the treatment of hemophilia A. Given as a one-time intravenous or into-the-vein, infusion, it aims to reduce the risk of bleeds in hemophilia patients.

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Mim8

Mim8 (denecimig) is an investigational therapy being developed by NovoNordisk to prevent or reduce the frequency of bleeding episodes in hemophilia A patients with or without inhibitors. It is given as an under-the-skin, or subcutaneous, injection.

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