Why Is Gene Therapy Player Freeline Therapeutics Stock Trading Higher Today?

Freeline Therapeutics Holdings plc 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:

• Favorable safety and tolerability, with no infusion reactions and no serious adverse events as of 13 weeks post-dosing for patient 1 and six weeks post-dosing for patient 2. 
• All treatment-related adverse events were Grade 1 and resolved without intervention.
• No elevations in liver enzymes.
• Patient 1 showed a nearly 700-fold increase over baseline to more than 70 μmol/L/h as of 12 weeks post-dosing. 
• Patient 2 showed a similarly robust response, with a greater than 300-fold increase over baseline to approximately 30 μmol/L/h as of four weeks post-dosing. 
• Normalization of leukocyte GCase in both patients demonstrates cellular uptake from plasma.

Price Action: FRLN shares are up 5.05% at $3.95 on the last check Wednesday.