Gene Therapy SB-525 Controls Bleedings in Men for at Least 2 Years

Gene Therapy SB-525 Controls Bleedings in Men for at Least 2 Years

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Gene Therapy SB-525 Controls Bleedings in Men for at Least 2 Years

A single dose of the investigational gene therapy SB-525 (giroctocogene fitelparvovec) continues to prevent bleeds and the need for prophylaxis, or preventive treatment, for at least two years in men with severe hemophilia A. These are the latest results from the five men given the highest dose of the therapy — 3×10e13 (30 trillion) vector genomes (vg)/kg — in the ongoing Alta Phase 1/2 clinical trial (NCT03061201). All of them also showed a fast and sustained…

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