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.

Industry: Biological Products, (No Diagnostic Substances) Peers: Aptorum Group Ltd NuCana PLC Orchard Therapeutics plc