Gene therapy for blood disorders
Fully diluted ownership
Value of Syncona holding
% of nav
Freeline Therapeutics is a biopharmaceutical company based at the UCLB Royal Free London Hospital focused on the development of liver-directed gene therapies.
Gene therapy is based on technology that delivers a working copy of a faulty gene directly to the target organ. Once delivered, the gene produces a correct form of the missing protein and has the potential to cure the disease.
Freeline’s most advanced program is a treatment for haemophilia B, a rare disorder that causes painful, crippling bleeding in patients’ joints.
There are several genetic diseases that are treated chronically with enzyme replacement therapy (ERT).
Freeline’s technology could replace ERT in a number of these diseases, with a single injection providing the possibility of long-term disease modification.
- Chris Hollowood
- Dominic Schmidt
- Prof. Amit Nathwani