Freeline Presents Positive New Data from Phase 1/2 GALILEO-1 Trial of FLT201, Its Novel Gene Therapy Candidate, in Gaucher Disease at ESGCT 30th Annual Congress

Substantial reduction in lyso-Gb1 substrate levels, an established biomarker of clinical response, observed in first patient treated with FLT201

Increases of several hundredfold over baseline in plasma GCase activity and normalization of cellular GCase levels continue to be observed in first two patients treated with FLT201

FLT201 continues to be well-tolerated, with no serious adverse events

Third patient dosed this month in ongoing Phase 1/2 GALILEO-1 trial

LONDON, Oct. 25, 2023 (GLOBE NEWSWIRE) -- Freeline Therapeutics Holdings plc (Nasdaq: FRLN) today announced that new clinical data from its ongoing Phase 1/2 GALILEO-1 trial of FLT201, its adeno-associated virus (AAV) gene therapy candidate for Gaucher disease, show a substantial reduction of glucosylsphingosine (lyso-Gb1) levels in the blood of the first patient treated with FLT201. Lyso-Gb1 is a well-established biomarker of clinical response in Gaucher disease, with reductions in lyso-Gb1 correlating with positive clinical outcomes. These data are being highlighted in an oral presentation at the European Society of Gene & Cell Therapy (ESGCT) 30^th Annual Congress held in Brussels, Belgium.

The oral presentation at ESGCT builds on recently reported initial clinical data from the first two patients treated with FLT201, which demonstrated a favorable safety and tolerability profile, robust increases in plasma levels of glucocerebrosidase (GCase), the enzyme that is deficient in people with Gaucher disease, and normalization of cellular GCase activity. The GCase deficiency in Gaucher disease leads to a buildup of harmful substrates glucosylceramide (Gb-1) and lyso-Gb1, causing symptoms including enlarged spleen and liver, low blood counts, bone pain and reduced lung function.

“Gaucher disease is a debilitating condition if not treated properly. A gene therapy that can deliver the same or better efficacy than currently available treatment modalities, while possibly freeing people from an ongoing treatment burden would mark a significant advance for patients with Gaucher disease,” said Ozlem Goker-Alpan, M.D., founder and CEO of the Lysosomal and Rare Disorder Research and Treatment Center (LDRTC) and an investigator in GALILEO-1. “The emerging data from the first two patients treated with FLT201 are very encouraging. The most recent evidence from the first patient that FLT201 is causing a reduction in Gaucher disease biomarkers with a persistent increase in peripheral GCase levels is particularly compelling.”