Hunting a cure, Spark reports steady progress on hemophilia B gene therapy as Pfizer preps for pivotal handoff
After some fine tuning on the manufacturing side, Spark Therapeutics $ONCE says it’s racked up a new round of promising results for its Phase I/II study of a groundbreaking new gene therapy that aims to cure hemophilia B. And the biotech is prepping a handoff of SPK-9001 to its Big Pharma allies at Pfizer $PFE this summer, who are planning to start a Phase III pivotal study.
Researchers are now reporting on 15 patients in the early-stage study getting a replacement factor IX gene to correct the mutation that requires constant care and factor replacement therapy to prevent bleeds. The annualized bleed rate for all of them was cut by 97% and researchers were careful to highlight three patients who are doing fine — with factor IX levels ranging from 38.1% to 54.5% beyond the 12-week mark— after being treated with an enhanced therapy produced by a new manufacturing approach.
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