Curing hereditary diseases with gene therapy
Today, it is possible to cure hereditary blind children, paralysed children with muscular atrophy and haemophilia patients using gene therapy. But the price tag is high, millions of kronor per treatment. At KTH, Johan Rockberg and his research team are working to ensure that more diseases can be cured with gene therapy and at a lower cost. KTH is a world leader in key research areas crucial to the successful development of effective gene therapy, such as antibody technology, directed evolution, synthetic biology, mathematics and machine learning.
Replacing missing or damaged genes
Revolutionary medicines that cure hereditary blind children, paralysed children with muscular atrophy and haemophiliac patients have been approved in the last five years. After a single treatment, blind people regain their sight, paralysed people can control their muscles and learn to walk, and haemophiliacs can start to heal their wounds naturally. The treatments are based on gene therapy, which uses a harmless virus to deliver DNA to replace missing or damaged genes in our cells. Gene therapy can potentially treat more and larger groups of patients, but unfortunately, these drugs are very expensive to produce. A single injection for haemophilia can cost up to €35 million.
Safer, cheaper and more effective gene therapy
The lack of efficient production methods is a major reason for the high manufacturing cost. One of the cost drivers is that cells do not like to make viruses, even harmless ones. Another challenge is that the viruses are not good enough at finding their way into the body and sometimes enter the wrong cells to deliver DNA, posing a safety risk. Some organs are also difficult for the virus to reach.
Adeno-associated virus (AAV)-based therapies can make new or functional copies of missing or non-functional genes in human cells. Simulations and computer modelling to improve the precision of drug treatments will also reduce the need for costly and time-consuming experiments. Solving the lack of efficient AAV production would increase gene therapy medicines' quality, productivity, efficiency and safety.
