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Companies » Sangamo Therapeutics regains rights to hemophilia gene therapy program

Sangamo Therapeutics regains rights to hemophilia gene therapy program

Sangamo resumes hemophilia A gene therapy program after Pfizer's decision to withdraw. AFFINE trial shows significant reduction in bleeding.

by Wendy Lazcano
January 3, 2025
Reading time: 2 mins read
Label on a genetic application form showing the word "Hemophilia" and a barcode, with test tubes in the background.

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Wendy Lazcano
Editor at Vitals Today
She is an experienced journalist who has worked in print media such as Diario de Cuba and Review Energy, as well as hosting radio (Wradio/Grupo PRISA) and television (Canal 33) programs. A specialist in politics and regulations, Wendy stands out for her ability to analyze and explain complex issues.
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Sangamo Therapeutics, a leader in genomic medicine, announced that it is taking back the rights to develop and market giroctocogene fitelparvovec. Pfizer decided not to continue with regulatory submissions or move forward with the commercialization of this treatment.

AFFINE trial confirms promising results for hemophilia

The phase 3 AFFINE trial, led by Pfizer, achieved both primary and secondary endpoints. The results demonstrated a significant reduction in annualized bleeding rate when compared to factor VIII replacement prophylaxis. These findings, presented at ASH 2024, were recognized as “Best of ASH.”

Sangamo seeks opportunities to commercialize the treatment

Sangamo is exploring alternatives to advance the program, including new strategic partners. The company highlights the potential of giroctocogene fitelparvovec as a transformative therapy for people with moderately severe to severe hemophilia A.

End of the agreement and future plans

The agreement with Pfizer will end on April 21, 2025, at which time Sangamo will resume full management of the program. The transition of the project will include continued monitoring of clinical trial participants.

Hemophilia A: challenges and needs

Hemophilia A, caused by a deficiency of coagulation factor VIII, causes prolonged bleeding and severe complications. This disorder affects 55-75% of patients with moderate or severe forms, requiring constant treatment.

Sangamo reaffirms its commitment to innovation

Sangamo is stepping up its work on innovative therapies for serious diseases. The company is advancing its Fabry disease program and other neurological projects, consolidating its leadership in genomic medicine.

Tags: FeaturedUSAInnovation
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