Pfizer and Sangamo Therapeutics on Tuesday reported that two patients with severe haemophilia A administered the highest dose of the experimental gene therapy SB-525 reached normal Factor VIII levels. Interim results from the Phase I/II Alta study showed that at week six post infusion, the two patients reached 140 percent and 94 percent of normal FVIII levels, as measured by one-stage clotting assay, and 93 percent and 65 percent, as measured by chromogenic assay.

"The interim data from the first eight patients…are encouraging and demonstrate a dose-dependent relationship, evidence of sustained factor levels, and low variability, both within each patient and within each cohort," remarked Edward Conner, chief medical officer of Sangamo. Shares in Sangamo climbed as much as 23 percent on the news.

"We look forward to the opportunity to expanding the cohort administered [the highest] dose and subsequent planning for the pivotal study," added Seng Cheng, chief scientific officer of Pfizer's rare diseases research unit.

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