Establishing Freeline Commercial Scale Viral Gene Therapy Manufacture for Fabry Disease

The rapid development of novel treatments called Advanced Therapies, such as gene therapies that provide the correct proteins, have required new, manufacturing facilities worldwide. Freeline Therapeutics Limited (Freeline), a UK company that makes gene therapies to treat bleeding and metabolic disorders, will work with the Cell and Gene Therapy Catapult (CGTC) to set up Freeline's UK manufacturing site at the CGTC, at the same time establishing an operating model for virus processes applicable to other gene therapies companies joining the CGTC. This has many advantages: 1. It will help Freeline to make new treatments more quickly so that patients can be treated sooner, the first of these will be for Fabry disease, a rare disease affecting 2.56 in 10,000 people, with diagnosed prevalent cases expected to double in the 7 major markets (GlobalData epidemiologist). 2. Removing Freeline’s reliance on availability of severely restricted European CMO capacity (few capable of clinical & commercial supply), gaining control of its manufacture to support the full product lifecycle. 3. Freeline will be able to make its own approved new therapies in the UK for delivery to patients worldwide, the first of these is anticipated to be for haemophila B a rare bleeding disorder affecting 1 in 30,000 males worldwide. 4. Establishing CGTC as a centre of excellence for large scale manufacture of viral vectors and cell therapies, for UK & International companies to its quality managed, self-contained units, creating employment opportunities for the skilled UK workforce.