Gene Therapy

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.

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.

Freeline is a clinical-stage gene therapy company based in the UK and Germany. Freeline’s vision is to create better lives for people suffering from inherited, systemic debilitating diseases.
Gyroscope, a clinical stage ophthalmology company, was founded to explore the convergence of advancements made in the understanding of the complement system’s impact on eye disease, the genetic basis of age-related macular degeneration (AMD) and gene therapy as a mode of sustained treatment delivery.
SwanBio Therapeutics is a gene therapy company developing leading-edge medicines to deliver dramatic clinical efficacy for the treatment of neurological diseases. Founded by proven industry leaders with a commitment to positively impacting the lives of people with neurological diseases, SwanBio merges clinical, scientific and drug commercialization expertise.

Our leading gene therapy portfolio today

We actively build out capabilities and technology to build companies with industrial scale

  • Strategy
  • Commercial model
  • Leadership and Team
  • Delivery
  • Manufacturing
Developing a strategy and plan to build a company which can take products to market

We build companies which target the key tissue compartments in gene therapy


Diseases of blindness or blinding conditions.

Central nervous system

Neurological disorders impacting the function of brain and spinal cord.


Diseases impacting a number of tissues and organs or the body as a whole.

Syncona gene therapy platform

One of the largest and highest quality unencumbered gene therapy platforms globally, with the potential to deliver dramatic efficacy across the tens of thousands of known genetic disease