Research

Our mission is to revolutionise healthcare by developing advanced therapies, such as gene therapies, for some of the world’s most challenging diseases.

Our initial aim is to develop to safe and effective gene therapies. Undesirable and severe side effects are currently a major barrier to gene therapies reaching the clinic and the patients who need them.

We harness the power of engineering biology to help make treatments safe, effective and targeted, with minimal side effects.

 

What is gene therapy?

Gene therapy is an innovative medical technology that offers ground-breaking opportunities to treat, prevent or potentially cure diseases or injuries by:

  • Replacing a faulty gene with a healthy one
  • Inactivating a defective gene
  • Adding a new gene

 

Our Focus

Advanced therapeutics, including gene therapies, could treat a wide range of diseases. We will initially focus on reaching the clinic with gene therapies for: 

Cancer cells

Cancer

While cancer survival rates have improved, it remains a leading cause of death worldwide. It is estimated there will be 28 million new cases of cancer globally each year by 2040. 

Heart disease

Heart Disease

Heart disease is the leading cause of death globally. It includes a range of conditions that affect the heart and blood vessels, such as coronary artery disease, heart attacks and heart failure.

Rare diseases

Rare Diseases

There are over 7,000 rare diseases - 95% lack an effective treatment. Our initial focus is on lysosomal storage diseases – metabolic disorders that can lead to organ damage and premature death.

 

How we are improving gene therapies

To minimise side effects and ensure that gene therapies are only active in the right place in the body, at the right level, and for the right amount of time we are developing a series of new tools: engineered genetic control systems.

We use our engineering biology expertise to design, build, and test these genetic control systems to make treatments more specific and targeted, preventing undesirable side effects.

Graphic of human body with a target symbol

Right location

Gene therapy will be delivered and activated only where it is needed in the patient body. Not in other tissues where they may be harmful. Our genetic control tools will ensure the treatment is accurately targeted towards the right organ, tissue and cell type. 

Graphic of DNA with levels symbol

Right level

Dial-like genetic controls will regulate therapeutic dosages to safe and effective levels. Like a thermostat, it will hold the dosage at the right level for it to be effective, whilst preventing levels from becoming unsafe, potentially enabling personalisation.

Graphic of DNA and clock symbol

Right time

Once delivered into a patient, we will precisely control when and how long the therapeutic is active for – such as switching it on only when it is needed, for as long as it is needed, and switching off the treatment if there are ever undesired side effects.  

 

Our Responsible Innovation commitment

Our research focuses on developing innovative solutions that are not only technologically feasible but that are also equitable, just, and in the public interest. 

A key feature of our Hub is the integration of social scientific expertise and research into its programme. 

Our team of social scientists play a crucial role in examining the social, political, and ethical dimensions of an engineering biology approach to advanced therapeutics, and developing the Hub’s capacity for responsible research and innovation. 

Edinburgh Hub for Responsible Innovation

 

Graphic of a person thinking with lightbulbs around their head

 

Introductory webinar

Dive into an overview of our engineering biology research program and activities, including examples of clinical applications of our research  - part of a UKRI series

Summary of webinar : Engineering Biology for Advanced Therapeutics

UKRI Webinar recording (1h) : Engineering Biology for Advanced Therapeutics

 

Collaborate with us

We want to work with clinicians, industry and relevant stakeholders who can help our tools to reach the clinic.