KU Leuven Spin-Off Pioneers More Targeted Approaches in Gene Therapy
Gene therapy uses safe viruses to repair or replace defective genes, making it possible to correct the genetic cause of a disease – something that was previously impossible. While such new drugs have great potential, there are still some challenges. One drawback is the need for high doses, which can lead to unwanted side effects. Tavira Therapeutics, a spin-off founded last year from KU Leuven, aims to tackle that problem by developing an innovative technology that can improve the effectiveness of current gene therapies.
Gene therapy is helping to improve the lives of people with incurable genetic diseases around the world. The therapy involves reprogramming naturally occurring, harmless viruses to deliver therapeutic genes into a patient’s cells. The new medicines can provide long-lasting health benefits from a single dose, offering therapeutic options for patients who would otherwise have no treatment. Gene therapy medicines are now available across Europe, for example for the treatment of haemophilia, demonstrating their potential on a large scale.
“In the most common form of gene therapy, therapeutic genes are delivered 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 nonpathogenic – in other words, harmless – and have a unique set of properties that make them ideal vehicles for gene delivery. But they don’t always efficiently target the tissues and cells that need to receive the therapeutic genes packaged inside them. That means a lot of virus has to be used in one dose to ensure that enough of it reaches the target cells. Sometimes this can cause unwanted side effects, which is not ideal.”
Professor Els Henckaerts
More efficient and safer gene therapies
Ensuring that AAV viruses efficiently deliver their precious cargo to the right cells has been a challenge for gene therapy for some time. Several years ago, the Trellis research group developed an innovative process that equips the viruses with molecules that precisely target them to the right locations. This advance enables more efficient delivery of the genes, and may help future AAV gene therapies have fewer side effects.
In collaboration with her colleagues Benjamien Moeyaert, Veronick Benoy, Inge Van Hove and Vibha Tamboli, Prof. Henckaerts founded the spin-off Tavira Therapeutics in 2023 together with the company General Inception, with the aim of bringing the new technology to patients. The start-up has so far attracted 1.1 million euros in financing, including additional investments from General Inception (via their industrial holding ‘Igniter Europe’) and from the Gemma Frisius Fund of KU Leuven.
“We will now use that investment to further develop our unique AAV capsid technology,” says Henckaerts. “In collaboration with our partners, we aim to create novel gene therapies that offer breakthrough treatments for genetic disorders that are currently incurable. At the moment, we focus on diseases of the central nervous system, but we hope to be able to treat other indications in the near future.”
During the founding of Tavira Therapeutics, Professor Henckaerts and her team were closely guided by the technology transfer experts at KU Leuven Research & Development (LRD), which has a strong track record in supporting scientists who want to translate their research into impactful innovations. “LRD helped us to valorize our research results through a spin-off, giving us the opportunity to bring our technology to the patient,” she says. “Having access to this kind of business-related expertise at our university is incredibly valuable!”