Positive Results in Trial for Gene Therapy

American depositary receipts of Freeline Therapeutics Holdings experienced a 14% increase, reaching $4.29, following encouraging data from an ongoing trial evaluating FLT201, the company's adeno-associated virus gene therapy candidate.

Despite this recent boost, the stock is still down by 40% for the year.

Freeline, a clinical-stage biotechnology company, announced that data from the trial's initial two patients indicated that a single infusion of the gene therapy candidate resulted in increased GCase activity in plasma. Additionally, it led to the normalization of GCase activity in leukocytes, all while maintaining a favorable safety and tolerability profile.

Pamela Foulds, Freeline's Chief Medical Officer, expressed her confidence in the therapeutic potential of FLT201, which was further solidified by the latest data. She also revealed that, based on the positive response and excellent safety record thus far, the company has decided to treat a third patient at the same dosage levels initially planned, rather than opting for a higher dose as originally intended.

Gaucher disease, a genetic disorder causing symptoms such as an enlarged spleen and liver due to a deficiency of the GCase enzyme, is the target condition for FLT201.