SB-525 Gene Therapy Phase 1/2 Trial Begins for Hemophilia A Patients

SB-525 Gene Therapy Phase 1/2 Trial Begins for Hemophilia A Patients
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The first patient in a Phase 1/2 clinical trial (“the Alta trial”) designed to assess safety and tolerability of SB-525 gene therapy has received treatment. Sangamo, in a collaboration with Pfizer, developed  the SB-525 gene therapy to correct the Factor VIII (FVIII) defect in Hemophilia A patients.

The Alta trial ( Number: NCT03061201) is currently recruiting male patients, and intends to enroll 20 adult patients with severe Hemophilia A. This clinical study will assess SB-525 adverse events and the levels of circulating FVIII up to three years after SB-525 infusion.

Hemophilia A patients need to repeatedly receive FVIII blood infusions to replace defects in blood clotting Factor VIII and prevent bleeding. Now, a single SB-525 administration may enable patient’s own cells to constantly produce the same amino acid sequence used in replacement therapy (Refacto and Xyntha).

SB-525 relies on a genetically engineered virus, recombinant adeno-associated virus (rAAV), carrying a “healthy” FVIII gene construct. The rAAV is harmless for humans, but it still delivers the corrected gene to cells in tissues. SB-525 rAAV is designed to restrict FVIII gene expression to the liver, a strategy intended to increase FVIII long-term hepatic production in hemophilia A patients.

This new therapy results from a global collaboration and license agreement between Sangamo and Pfizer to develop and commercialize gene therapies for hemophilia.”We are excited to see progress in our partnership program for SB-525 with Sangamo,” said Greg LaRosa, Chief Scientific Officer, Pfizer Rare Disease in a press release issued by Sangamo . “By combining Sangamo’s scientific and technical expertise across multiple genomic platforms, with Pfizer’s global reach, resources and expertise, we are advancing the promise of gene therapy with the hope of making a meaningful impact for people living with Hemophilia A.”

Earlier this year, the U.S. Food and Drug Administration granted Orphan Drug and Fast Track designations to SB-525, which also received Orphan Medicinal Product designation from the European Medicines Agency.

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