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Freeline Therapeutics Holdings plc (NASDAQ:FRLN) reported initial data from the ongoing Phase 1/2 GALILEO-1 trial of FLT201, its adeno-associated virus (AAV) gene therapy candidate, in Gaucher disease.
Gaucher disease is a genetic disorder in which a deficiency of the GCase enzyme leads to a buildup of harmful substrates, causing symptoms including enlarged spleen and liver, low blood counts, bone pain, and reduced lung function.
Data from the first two patients in GALILEO-1 show that a single infusion of FLT201 led to several hundred-fold increases in GCase activity in plasma and normalization of GCase activity in leukocytes.
Both patients were treated with a 4.5x1011 vg/kg dose and have successfully come off their prior therapies.
As of the September 27 data cutoff, the data demonstrated:
Price Action: FRLN shares are up 5.05% at $3.95 on the last check Wednesday.
Posted In: FRLN