Freeline starts Gaucher study, Parkinson's research

 FLT201 is a highly differentiated AAV gene therapy candidate that delivers a longer-acting engineered variant of GCase, the enzyme missing in people with Gaucher disease

Company expects to report initial clinical data for FLT201 in third quarter of 2023

Parkinson’s disease program leverages same longer-acting GCase enzyme as FLT201, with aim of developing a gene therapy for genetically defined patient subset with GBA1 mutations

 Freeline Therapeutics Holdings plc (Nasdaq: FRLN) today announced that the first patient has been dosed in the Phase 1/2 GALILEO-1 clinical trial evaluating FLT201, its adeno-associated virus (AAV) gene therapy candidate, in Gaucher disease type 1. Gaucher disease is a debilitating genetic disorder in which a deficiency of the GCase enzyme leads to a buildup of fatty substances in the organs, causing symptoms including enlarged spleen and liver, low blood counts, bone pain and reduced lung function.

“We believe that FLT201 has life-changing potential for people with the most common type of Gaucher disease,” said Pamela Foulds, M.D., Chief Medical Officer at Freeline. “Existing therapies do not sufficiently penetrate deeper tissues, and enzyme levels wane in between infusions, giving harmful substrates the opportunity to build back up in all disease-affected organs. FLT201 is a highly differentiated gene therapy candidate for Gaucher disease type 1 that delivers a longer-acting, rationally engineered enzyme. Preclinically, FLT201 has been shown to produce sustained levels of enzyme and penetrate deeper tissues, including bone and lung, giving us reason to believe it has the opportunity to stop disease progression and improve outcomes with a one-time treatment.”

“The dosing of the first patient in the GALILEO-1 trial of FLT201 marks an important step forward for the Gaucher community,” said Dr. Ozlem Goker-Alpan, founder and CEO of the Lysosomal and Rare Disorder Research and Treatment Center (LDRTC) and an investigator in GALILEO-1. “There is a clear need for better treatment options. Existing therapies come with a heavy lifelong treatment burden, and many patients continue to experience serious symptoms. FLT201 represents a promising approach for addressing the underlying cause of Gaucher disease with a one-time gene therapy, potentially providing a more effective and less burdensome option for patients. I look forward to further evaluating FLT201 in this clinical trial.”

https://finance.yahoo.com/news/freeline-announces-first-patient-dosed-113000832.html