Experimental Treatments for Hemophilia

Aminocaproic acid, also known as 6-aminocaproic acid or epsilon-aminocaproic acid, is an antifibrinolytic medication that can help to stabilize blood clots. The medication is not formally approved as a treatment for hemophilia, but it may be used off-label in some situations to help control bleeding in hemophilia patients. It specifically has been used to manage bleeding in the mouth, such as during tooth extractions.

Genome editing using the experimental CRISPR/Cas9 (clustered regularly interspaced short palindromic repeats) method is a potentially faster, easier, cheaper, more accurate, and more flexible therapeutic approach than other DNA editing techniques, and may offer a promising new technology for treating genetic diseases such as hemophilia. Preclinical studies in mice have shown that this therapeutic strategy might benefit people with hemophilia.

SB-525 is composed of a small portion of genetic material with the code necessary for the production of factor VIII. Once inside cells, it is hoped that this genetic material will bind to the patient’s own genetic material and allow the production of factor VIII by the cell’s protein-making machinery. A Phase 3 trial is currently recruiting hemophilia A and B patients.

SPK-8011 is an experimental gene therapy being developed for the treatment of hemophilia A. It contains a modified virus that carries the human F8 gene under the control of a specific DNA sequence, which ensures its protein product, factor VIII, is only made in the liver. A Phase 1/2 trial is still enrolling hemophilia A patients.

SPK-9001 is a gene therapy candidate designed to deliver a healthy copy of the gene encoding for factor IX to the patient’s liver cells where the clotting factors are made. Once delivered to the liver cells using a modified harmless adeno-associated virus, SPK-9001 helps maintain a constant and sustained level of factor IX in the blood. A Phase 3 trial is currently recruiting hemophilia B patients.

TRM-201 is a non-steroidal anti-inflammatory drug (NSAID) that is being developed to treat severe joint disease in patients with hemophilia. TRM-201 binds to and inhibits COX-2. As a result, it is intended to reduce inflammation, pain, and fever, as well as prevent the clotting of blood (anti-platelet effect), and reduce gastrointestinal bleeding, compared with other NSAIDs. A Phase 3 trial is being planned.