Syncona has one of the largest and highest quality gene therapy platforms globally with the potential to deliver dramatic efficacy across the tens of thousands of known genetic diseases.
We have built companies targeting genetic diseases in the key tissue compartments where there is in the tissue compartments where gene therapy has shown dramatic patient impact that gene therapy can be effective. These include: the retina, the liver and the central nervous system. Our ambition is ultimately to address the most prevalent diseases in each tissue compartment. One of Syncona’s first gene therapy companies, Nightstar, was acquired by Biogen in June 2019. Read more about Nightstar in our previous portfolio companies.