Researcher wins UKRI - Artificial Intelligence for Health award
Dr Giovanni Stracquadanio, Senior Lecturer (Associate Professor) in Synthetic Biology, and Co-director of the Edinburgh Genome Foundry, along with Prof Susan Rosser and Dr Eve Miller-Hodges, is the recipient of a UKRI - Artificial Intelligence Innovation to Accelerate Health Research Award.
Giovanni has been awarded £599,000 for his project, Intelligent Deimmunisation for Enzyme Replacement Therapies (IDERT), from the UK Research and Innovation’s (UKRI) Technology Missions Fund. This funding is meant to support the responsible development and deployment of AI technologies in society.
Enzyme Replacement Therapies
Lysosomal storage diseases (LSDs) are rare inherited diseases caused by the deficiency of lysosomal enzymes.
The disease causes the accumulation of chemicals in a part of the cell called the lysosome. This can ultimately lead to organ damage and premature death. LSDs are caused by inherited genetic mutations, meaning they cannot be cured but can be treated using Enzyme Replacement Therapies (ERTs).
This involves restoring the body’s enzyme levels through the intravenous (IV) infusion of a corrected version of the defective enzyme.
The response to ERT is variable, because the corrected enzymes are less active than the normal enzyme, are unstable in blood and are not properly absorbed by cells.
Repeated treatments usually trigger an undesirable immune response and the formation of anti-drug antibodies, limiting the ERTs’ activity and preventing long-term treatment in many patients.
Using AI to design new therapies for rare inherited diseases
One of the most common LSDs is Fabry Disease (FD), which causes irreversible damage to the heart, vasculature, and kidney.
Giovanni and his team use Artificial Intelligence (AI) to design enzymes that possess desired functions, but at the same time lack specific surface molecules that trigger undesirable immune reactions, a process called deimmunisation. They anticipate their research will deliver better therapies for FD patients.
They will manufacture these enzymes using mammalian cell systems, to ensure the ERTs have human-like biochemical properties.
Importantly, since AI has the potential to impact the life of millions of patients and their families, the team will engage with patients through open meetings to explain the potential of this new technology. The project builds on the team's unique expertise in AI, the production of large molecules in living cells, and medicine. It is one of the largest ERT deimmunisation studies to date, with the potential to provide effective treatment options for FD and other LSD patients in the future.
Dr Stracquadanio and Prof Rosser are also the founders of ZYTHERA, a new startup company of the University of Edinburgh, using AI and engineering biology to engineer new, more effective enzyme replacement therapies.
Giovanni and his team get advice on matters of IP, business development and fundraising from Edinburgh Innovations, the University of Edinburgh’s commercialisation service.
We are building a next generation of enzyme replacement therapies suitable for lifelong treatment, which will improve the life of many patients who are currently left with no treatment option due to strong immune response. Importantly, we are doing it working with and for the patients, and we are excited to show them the potential of AI for treating rare diseases.