Developed at the HUN-REN Research Centre for Natural Sciences and recently published in Frontiers in Immunology, this method is freely available to the international scientific community. By helping researchers detect antibody-related risks more effectively, coreTIA has the potential to accelerate the development of gene therapies and bring life-saving treatments to patients sooner.
The new diagnostic method was developed under the leadership of Dr. Dániel Hillier, lecturer at the Faculty of Information Technology and Bionics of Pázmány Péter Catholic University (PPKE ITK), in collaboration with the Systems Neurobiology of Vision Research Group at the HUN-REN Research Centre for Natural Sciences.
Approved gene therapies such as Luxturna (for certain types of blindness) or Zolgensma (for spinal muscular atrophy) can dramatically improve patients’ quality of life. These therapies are delivered into the human body using adeno-associated viruses (AAVs) as vectors.
The human immune system is not encountering these viral particles for the first time. In everyday life, the body may be exposed both to AAVs and to other viruses, such as adenoviruses used in Covid-19 vaccines (including Vaxzevria and Sputnik V). In both cases, the immune system produces antibodies - regardless of whether the exposure occurred through natural infection or for therapeutic purposes. If a patient has previously (and often unknowingly) been infected with the same type of AAV that a gene therapy relies on, pre-existing antibodies may drastically reduce or even completely block the therapy’s effectiveness.
A Hungarian method for successful gene therapies
Before gene therapies can be applied, it is essential to measure the level of AAV-specific antibodies in the body. Until now, every research laboratory and pharmaceutical company has had to develop and maintain its own method for this purpose. This is not only costly and inefficient, but the wide variety of approaches also makes it difficult to reproduce research results across laboratories - leading to a suboptimal use of scientific resources.
The new method developed by Hungarian researchers, coreTIA, measures the level of neutralizing antibodies against therapeutic viruses from a simple blood sample. Compared to earlier techniques, it is far more reliable, requires fewer samples, and - through statistical analysis - provides each result with a confidence score.
Thanks to the collaboration of several leading institutions (including the Department of Ophthalmology at Semmelweis University, the Grastyán Endre Translational Research Centre at the University of Pécs, the Institute of Neuroscience at KU Leuven, the HUN-REN Institute of Experimental Medicine, Femtonics Ltd., and the Research Centre for Natural Sciences), the applicability of the new diagnostic method was tested on blood samples from humans, non-human primates, cats, and mice. This multi-species validation is particularly important, as animal models continue to play a key role in developing novel gene therapies and in understanding diseases that remain difficult to treat today. A standardized methodology such as coreTIA enables preclinical and clinical trials of new therapies to be conducted consistently at every stage of development.
Beatrix Kovács, PhD candidate, and Dr. Dániel Hillier, research group leader
Sharing knowledge: a faster path to healing
A single gene therapy treatment can bring significant improvement to a patient’s life, but its cost may reach hundreds of thousands of euros per person. To make such life-changing therapies accessible to more patients, it is crucial to reduce both development and diagnostic expenses. One way to achieve this is through the introduction of unified, validated diagnostic standards -rather than every institution developing and maintaining its own separate method.
Researchers at the HUN-REN Research Centre for Natural Sciences have taken a major step in this direction. By making their method openly available to the international scientific community — complete with a step-by-step protocol and open-source code, much like in the software industry — they aim to ensure that medicine, too, can build on transparent, verifiable, and universally accessible methods. Such openness can form the foundation of safe and cost-effective therapies.
“The reproducibility crisis in science wastes billions of euros and slows the progress of medicine. Many research results cannot be replicated in other laboratories, delaying the arrival of new treatments to patients. Open science offers an effective solution to this challenge. By sharing our coreTIA method freely, we make it possible for laboratories anywhere in the world to use the same reliable standard for measuring immune responses to gene therapy—and to build upon each other’s work. Accelerating scientific breakthroughs requires exactly this kind of open collaboration, which can help gene therapies reach patients suffering from serious diseases more quickly and safely,” said Dr. Dániel Hillier, lead researcher, head of the Systems Neurobiology of Vision Research Group at the HUN-REN Research Centre for Natural Sciences, and lecturer at the Faculty of Information Technology and Bionics of Pázmány Péter Catholic University.
Among the co-authors of the study are Dr. Balázs Rózsa and Dr. István Ulbert. The full article is available in Frontiers in Immunology HERE.
Further information:
Dr. Dániel Hillier
Lecturer, Faculty of Information Technology and Bionics, Pázmány Péter Catholic University, Head of the Systems Neurobiology of Vision Group, HUN-REN Research Centre for Natural Sciences
hillier.daniel@ppke.hu
