SPK-8016, also known as RG6358, is an experimental gene therapy that was being investigated for hemophilia A patients who had developed neutralizing antibodies (inhibitors) against clotting factor VIII (FVIII).
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While there is no cure for hemophilia A, treatments like Wilate, Jivi, and DDAVP help prevent and control bleeding. These therapies work by replacing or increasing factor VIII. Experimental gene therapies, such as the discontinued SPK-8016, have also been investigated.
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Wilate (human von Willebrand factor/coagulation factor VIII complex) is a plasma-derived replacement therapy that's approved to prevent and control bleeds in adults and adolescents with hemophilia A. It is given via an into-the-vein, or intravenous, injection.
Jivi (damoctocog alfa pegol) is an approved lab-made replacement therapy designed to prevent or treat bleeding episodes in previously treated children and adults with hemophilia A. It is administered via into-the-vein (intravenous) infusions.
There is no cure for hemophilia A, but several treatments are available to help prevent and control bleeds, reduce the risk of complications, and improve quality of life. People with hemophilia A are susceptible to excessive and prolonged bleeding episodes because their bodies don’t make enough functional factor…
Hemophilia A is a rare genetic disorder that affects the blood’s ability to clot properly. It is the most common form of hemophilia, responsible for 80% of all cases.
Hemophilia is a disorder wherein the blood is not able to clot properly. As a result, patients bleed easily, and it's unusually prolonged and heavy.
Hympavzi (marstacimab-hncq) is an antibody-based therapy that is approved to prevent or reduce the frequency of bleeds in patients with hemophilia A or B, with or without inhibitors. It is given as weekly subcutaneous, or under-the-skin, injections that can be self-administered.
FLT180a, also known as verbrinacogene setparvovec, is an investigational gene therapy that was investigated as a treatment for people with hemophilia B.