First Patient Announced in New Hemophilia A Clinical Trial

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According to a late-summer press release sent out by the companies, two pharma players are joining forces and making waves in gene therapy for hemophilia A.

Sangamo and Pfizer announce that the first patient—ever—has received treatment in the Phase 1/2 clinical trial evaluating SB-525, an investigational gene therapy for patients with hemophilia A.

Chief Executive Officer of Sangamo Theraputics Dr. Sandy Macrae says, “We are excited to announce the first patient infusion in this initial clinical trial of SB-525. Gene therapy holds promise as a potential one-time treatment to control hemophilia A and prevent spontaneous bleeding.”

The clinical trial is also known as “the Alta trial.” Individuals will be administered a range of doses; both researchers and participants will know which treatment is being directed. The study is aiming to assess the tolerability and safety of SB-525 investigational gene therapy in up to 20 adults living with severe hemophilia A. Chief Scientific Officer, Pfizer Rare Disease Greg LaRosa says, “We are excited to see progress in our partnership program for SB-525 with Sangamo.” Hey, we are excited too!

Orphan Drug and Fast Track designations have been granted by the FDA. Right now, there are about 16,000 people living with hemophilia A in the United States and more than 150,000 across the globe.

Want to learn more about SB-525, Sangamo, or Pfizer? Click here for the official release.


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