Gene therapy uses safe viruses to fix or replace faulty genes, making it possible to correct the genetic cause of a disease - something that was not possible before. Although these new medicines have shown great potential, there are still some challenges. One drawback is the need for high doses, which can lead to undesirable side effects. Tavira Therapeutics, a startup launched at KU Leuven last year, is working to tackle this problem by developing an innovative technology to enhance the effectiveness of current gene therapies.
Gene therapy is helping to change the lives of individuals affected by incurable genetic conditions across the globe. It works by reprogramming naturally occurring, harmless viruses so that they can deliver therapeutic genes to the cells of a patient. These new drugs can offer long-lasting benefits from a single dose and are providing therapeutic options to patients that would otherwise have no treatments available to them at all. As gene therapy-based drugs are already available throughout Europe today, for example to treat hemophilia, the potential of this type of treatment has been widely demonstrated.
"In the most common form of gene therapy, therapeutic genes are introduced into the patient's body via a virus known as the adeno-associated virus (AAV)," explains Professor Els Henckaerts of the Trellis Research Group, which is part of the KU Leuven Departments of Cellular and Molecular Medicine, and Microbiology, Immunology and Transplantation.
"AAV viruses are non-pathogenic - in other words harmless - and have a unique set of characteristics that make them ideal vehicles for gene delivery. However, they are not always very efficient at targeting the tissues and cells that need to receive the therapeutic genes that are packaged inside them. This means that a lot of virus has to be used in a single dose to make sure enough reaches the target cells. Sometimes this can cause unwanted side effects which is obviously not ideal."
Professor Els Henckaerts
More efficient and safer gene therapies
Making sure that AAV viruses are more efficient at delivering their precious cargo to the right cells has been a challenge for gene therapy for some time. However, a few years ago, the Trellis Research Group developed an innovative process that equips AAV viruses with molecules that accurately target them towards their intended locations. This advancement enables more efficient delivery and can help future AAV gene therapies have fewer side effects.
Working with her colleagues Benjamien Moeyaert, Veronick Benoy, Inge Van Hove and Vibha Tamboli, Prof. Henckaerts co-founded the spin-off "Tavira Therapeutics" together with the company General Inception in 2023, with the intention of bringing this new technology to patients. The start-up has so far attracted 1.1 million euros in financing, including additional investment from General Inception (via 'Igniter Europe', an affiliated industrial holding) and from the Gemma Frisius Fund of KU Leuven.
"These new investments in our company will be used to further develop our unique AAV capsid technology," said Professor Henckaerts. "In collaboration with our partners, we strive to create new gene therapies that will provide breakthrough treatments for genetic disorders that are currently incurable. At the moment, we are focusing on diseases of the central nervous system, but we are hoping to move into other indications in the near future."
During the creation of Tavira Therapeutics, Professor Henckaerts and her team were closely guided by the technology transfer experts at KU Leuven Research & Development (LRD) who have a strong track record of helping academic researchers turn their ideas into impactful innovations. "LRD helped us transition our research into a spin-off, giving us the opportunity to bring our technology to patients," she said. "Having access to this kind of business-related expertise at our university is incredibly valuable!"
