Following disappointing results in its gene therapy trial for haemophilia, Philadelphia-based Spark Therapeutics's shares have fallen by 25%. The company said it was "disappointed" with the results, which were announced on Tuesday. haemophilia A reduces the need for infusions by 97% in a trial of 12 patients. However, in the higher dosage trial intended for later stage testing, the treatment had an negative reaction in two of seven patients, with one admitted to hospital with a serious adverse effect.
Following disappointing results in its gene therapy trial for haemophilia, Philadelphia-based Spark Therapeutics's shares have fallen by 25%. Spark claims that the experimental treatment for haemophilia A reduces the need for infusions by 97% in a trial of 12 patients. However, in the higher dosage trial intended for later stage testing, the treatment had an negative reaction in two of seven patients, with one admitted to hospital with a serious adverse effect.