Latest Data Release From Clinical Trial Suggests That Hemophilia A Gene Therapy Isn’t Far Off

According to a story from biopharmadive.com, the race to a hemophilia A gene therapy continues to tighten following a recent release of early data from Sangamo Therapeutics and Pfizer. The company presented findings last Saturday at the American Society of Hematology’s annual meeting. Sangamo’s gene therapy for hemophilia is currently known as SB-525 and the latest results indicate that it could be an effective method for treating the disorder.

About Hemophilia

Hemophilia is a genetic disorder which affects the ability of the blood to form clots, a process that is vital for stopping bleeding after a wound is sustained. The severity of symptoms can vary widely. The disorder is caused by a mutation found on the X chromosome. Symptoms include bleeding for a long time after an injury, risk of bleeding in the brain and joints, and easy bruising. Bleeding in the joints can cause permanent damage and brain bleeding can lead to headaches, decreased consciousness, and seizures. There are multiple types of hemophilia, with the most common types being type A and type B, which are distinguished by having deficiencies in different clotting factors. Treatment involves replacing the missing clotting factor. Drugs that thin the blood should be avoided. To learn more about hemophilia, click here.

Encouraging Results so Far

The data release was in regard to five hemophilia A patients that took the strongest formulated dose of SB-525. The treatment was able to trigger a major boost in factor VIII that even exceeded what is typically considered the normal range. In four of these patients, that level was maintained for more than 12 weeks. For the first patient dosed, the levels have remained elevated for 44 weeks.

During this time, all of the patients were able to halt their typical factor replacement treatment and haven’t experienced any episodes of bleeding. Sangamo hopes that SB-525 will display superior long-term effectiveness when compared to BioMarin’s gene therapy, which is currently at the greatest progression of development. BioMarin’s gene therapy was found to lose effectiveness after 12 to 18 months.

Only time will tell how SB-525 measures up, but it is important to note that the drug did not act the same way in all of the patients. One patient saw factor VIII levels fluctuate up and down and the researchers aren’t sure exactly why.

At any rate, hemophilia A patients should have something to look forward to in 2020.


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