Freeline announces first patient dosed with its novel gene therapy candidate for Gaucher Disease

Freeline has 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 build up of fatty substances in the organs, causing symptoms including enlarged spleen and liver, low blood counts, bone pain and reduced lung function.

Freeline has also announced that it will be extending innovation into GBA1-linked Parkinson’s Disease. The research program builds on its work with Gaucher disease, leveraging its rationally engineered longer-acting GCase variant to extend the impact of its innovation to develop a gene therapy candidate for a subset of Parkinson’s disease patients with mutations in the GBA1 gene, which encodes for GCase.

Read the full news update on the Freeline website.