Pfizer is a leading gene therapy developer, yet is facing setbacks in its studies. Unexpected findings in the study has lead Pfizer to delay their development plans, pausing patient screening and dosing in a Phase III study of its hemophilia A gene therapy. The unexpected findings included some participants experiencing higher-than-normal levels of the clotting factor that's missing in hemophilia. This gene therapy program is being developed with Sangamo Therapeutics.
You may be asking yourself, isn't increased levels of clotting factor good for hemophiliacs? And you'd probably be right, except for the fact that the increased levels observed in the Pfizer study was "higher-than-normal" and raised eyebrows to the risk of blood clots. According to Pfizer, no patient has experienced a blood clot, but some are actively being treated with oral blood thinners.
In hemophilia B, results of a study are expected in early 2023, and Pfizer is still assessing the impact on the testing timeline in hemophilia A. Even though there has been a setback, Pfizer will continue to proceed with there studies and work for a therapy.
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