The University of Coimbra (UC) is leading a new research consortium - GeneH: Excellence Hub for Advancing Innovation in Gene Therapy - which aims to accelerate gene therapy in Europe. In particular, GeneH wants to speed up the application of innovative therapies for genetic diseases that currently have no cure, boosting the application of scientific knowledge in the clinical field, which is fundamental for the advancement of treatments.
The new funding, of around 5 million euros (more precisely 4,885,375.00 euros), will make it possible to create, by the end of 2028, close collaborations between academia, industry, government entities and society, through a quadruple helix approach - which is essential to overcome the current barriers in translating research into the clinic.
“Despite the scientific advances in gene therapy, there are significant challenges that we need to overcome to ensure that these innovations actually reach patients. Clinical trials in this area are still limited due to regulatory complexity, the lack of specialized infrastructures and the limited funding available for clinical development,” says Luís Pereira de Almeida, a professor at the UC Faculty of Pharmacy, president of the UC Center for Neuroscience and Cell Biology and coordinator of the Center for Innovation in Biomedicine and Biotechnology.
“In addition, there is great potential in the interaction between academia and industry that we can and must exploit better, to facilitate the transfer of technology and allow scientific discoveries to be translated into innovative and accessible therapies. Another major challenge is the involvement of regulatory bodies and political decision-makers, whose active participation is key to speeding up approval and funding processes. Finally, specialized training in this area is still insufficient, both for researchers and health professionals, and it is essential that we invest in training to ensure the sustainability and growth of this sector,” adds the GeneH coordinator.
Gene therapy is an innovative therapeutic approach, which is capable of developing new treatments for various diseases, especially rare pathologies, and which allows a curative approach in many cases with just a single administration of the treatment. It has revolutionized the treatment and progression of, for example, neuromuscular or ophthalmological diseases such as Spinal Muscular Atrophy or Leber's Congenital Amaurosis. There are currently around 20 gene therapy products approved for the treatment of diseases in Europe, although there are around 7,000 rare diseases in the world, 95% of which have no effective therapy.
Given this context, Gene H, through its multidisciplinary research and innovation network, aims to “accelerate research and development of new gene therapies, bring scientific advances closer to clinical trials, promote collaboration between different sectors to overcome regulatory and funding barriers, while promoting training in gene therapy and strengthening the involvement of society”, says Luís Pereira de Almeida.
The GeneH consortium will work closely with GeneT: Centro de Excelência em Terapia Génica em Portugal, Portugal's first gene therapy research and innovation center. “GeneH will expand the GeneT network, strengthening international collaboration and promoting additional synergies between research, industry, government and society, to accelerate innovation and clinical trials,” explains Luís Pereira de Almeida, who also leads GeneT. It should be remembered that the UC also coordinates the ERA Chair GCure - From Gene to Cure, a project funded with 2.5 million euros to strengthen the UC's capacity and visibility at the forefront of gene therapy research and innovation.
GeneH will involve entities from two countries, Portugal and Slovenia, and 13 partners, including teaching and research institutions, health authorities, government entities, industry and civil society. In addition to the University of Coimbra, the consortium in Portugal includes the Portuguese Association of Hereditary Ataxias, Biocant, Bluepharma, the Centro Regional Coordination and Development Commission, the Pedro Nunes Institute and the Coimbra Local Health Unit. In Slovenia, GeneH includes the Regional Development Agency of the Ljubljana Urban Region, the Slovenian Association of People with Rare Diseases, BioSistemika (a biotechnology company), the Slovenian Innovation Hub, the National Chemical Institute and JAFRAL (a biotechnology company).
“Our team wants to ensure that innovations reach people with genetic diseases that can be treated with gene therapy as quickly as possible in a safe and effective way, as well as ensuring that patients' needs are heard,” stresses Luís Pereira de Almeida. “This consortium is also very important for strengthening Europe's role as a global leader in the field of gene therapy,” he adds.
The consortium is funded by the European Commission, within the scope of Horizon Europe and through the Excellence Hubs competition, which aims to support projects focused on innovation, particularly in promoting and creating synergies between innovation ecosystems and entities such as universities, companies, government bodies and society, to broaden the participation of certain countries in research. The call for this competition in which GeneH was selected was highly competitive, with 205 applications submitted and only 11 projects selected for funding.
GeneH Public Presentation Event
The GeneH launch event - in English - will take place next Friday, the 21st, between 2pm and 6pm, in the Amphitheater of the Science Museum of the University of Coimbra.
The GeneH coordinator, Luís Pereira de Almeida, and the coordinator of the Center for Gene and Cell Therapy Technologies at Slovenia's National Institute of Chemistry, Mojca Bencina, will speak about the consortium. The Minister for Education, Science and Innovation, Fernando Alexandre, will close the session at 5.30.
Also speaking will be the Slovenian Ambassador to Portugal, Mojca Nemec van Gorp, the gene therapy researcher and co-founder of Vivet Therapeutics, Gloria González Aseguinolaza, and the Scientific Program Manager of the European Infrastructure for Translational Medicine, David Morrow.
The session will also feature a panel on the importance of the quadruple helix model for research, development and clinical translation, with representatives from Health Cluster Portugal (Guy Villax), IAPMEI - Agency for Competitiveness and Innovation (Nuno Gonçalves), Infarmed - National Authority for Medicines and Health Products (Carlos Alves), Portugal Ventures (Miguel Botto), and the Union of Rare Disease Associations of Portugal (Raquel Marques).
More information about the GeneH launch event is available here.
