Gene therapy is the introduction of genetic material into cells to replace missing or defective genes. If a gene doesn’t function properly, the introduction of a normal copy of a gene may be able to treat or prevent disease. It is a technique that has the potential to deliver long-term and potentially curative benefits to patients with genetic disorders.

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.

The Syncona team has built companies targeting genetic diseases in the key tissue compartments where gene therapy has shown dramatic patient impact. These include: the liver, the central nervous system and kidney. Our ambition is ultimately to address the most prevalent diseases in each tissue compartment.

Key facts*


Value of gene therapy portfolio


Number of clinical trials


Portfolio company Board seats

Our gene therapy portfolio companies

The gene therapy pipeline

Our domain areas

Syncona has deep domain expertise across four key therapeutic areas.

*Unless stated all financials at 30 September 2022