Freeline Therapeutics Holdings plc

Freeline Therapeutics Holdings plc is a United Kingdom-based clinical-stage biotechnology company. The Company is developing transformative adeno-associated virus (AAV) vector-mediated gene therapies. It is focused on improving patient lives through one-time treatments for chronic debilitating diseases. It uses its rationally designed AAV vector and capsid (AAVS3), along with promoters and transgenes, to deliver a functional copy of a therapeutic gene into human liver cells, thereby expressing a persistent functional level of the missing or dysfunctional protein into the patient's bloodstream. Therapeutic AAV vectors consist of two main components, the capsid and the expression cassette (that contains the gene and the promoter). The Company is advancing clinical programs in Fabry disease and Gaucher disease Type 1. The Company's pipeline includes Fabry Disease FLT190 for the treatment of Fabry disease and Gaucher Disease FLT201 for the treatment of type 1 Gaucher disease.