Freeline Therapeutics Holdings plc

freeline.life

Freeline Therapeutics Holdings Plc engages in the business of developing and commercializing gene therapies. The company is headquartered in Stevenage, Hertfordshire and currently employs 152 full-time employees. The company went IPO on 2020-08-07. The firm is developing transformative adeno-associated virus (AAV) vector-mediated gene therapies for patients suffering from chronic debilitating diseases. The company uses its rationally designed AAV vector and capsid (AAVS3), along with novel 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 a patient’s bloodstream. The company is advancing FLT201, a differentiated gene therapy candidate that delivers a novel transgene, in a Phase 1/2 clinical trial in people with Gaucher disease type 1. Therapeutic AAV vectors consist of two main components: the capsid and the expression cassette (that contains the gene and the promoter). The company has additional programs in research, including one focused on GBA1-linked Parkinson’s disease that leverages the same novel transgene as FLT201.