Researchers from the companies VIVEbiotech, OneChain Immunotherapeutics and the UPF spin-off Integra Therapeutics, the Department of Medicine and Life Sciences of the Pompeu Fabra University (UPF), the Institute of Molecular Biology of Barcelona (IBMB-CSIC) and the Center for Energy, Environmental and Technological Research (CIEMAT) have joined forces to design using artificial intelligence and produce next-generation viral particles to be applied in advanced therapies. These particles can be used in immunotherapies, such as CAR-T therapy, to treat a variety of cancers and rare diseases including hereditary anemia due to pyruvate kinase deficiency.  

The project has been selected by the Ministry of Science, Innovation and Universities in collaboration with the Center for Technological Development and Innovation (CDTI) following an assessment of the impact it could have on health; it will be carried out between 2024 and 2027 and will receive €3.8 million in funding through the TransMisiones program. 

“We are pleased to announce this partnership between Spanish companies and research centers that have extensive knowledge and experience throughout the entire value chain of research, development, and manufacture of advanced therapies for treating rare diseases and oncology” said Dr. Gurutz Linazasoro, CEO of VIVEbiotech. VIVEbiotech, with headquarters in San Sebastián, is a company specializing in the development and manufacture of lentiviral vectors.

Dr. Avencia Sánchez-Mejías, CEO and co-founder of Integra Therapeutics, a company that develops gene writing tools in Barcelona, explains that incorporating AI into cell therapy holds great potential: “AI algorithms that are used for word processing, for example ChatGPT, can also be applied to biology. The tool we will create will allow us to design new DNA sequences and new protein functions that were not feasible before, in addition to obtain a universal therapeutic product for a specific disease more quickly.”

Cell therapy is an important technique for treating complex diseases or those with a poor prognosis. Cell therapy was traditionally carried out in cells extracted from patients that are readministered to the patient once they have been modified in the laboratory. 

“The development we are proposing will allow CAR-T therapy to be carried out directly within the patient by administering these new viral vectors, which has a significant advantage over ex vivo manufacturing in terms of time and cost savings and ease for the patient since they do not have to undergo apheresis, or in other words, cell extraction” explains Dr. Víctor Manuel Díaz, Scientific director of OneChain Immunotherapeutics. OneChain Immunotherapeutics is a spin-off of the Josep Carreras Leukaemia Research Institute, which develops CAR-T therapies for oncological diseases.

“Applying AI with directed evolution techniques means we can optimize the efficiency and accuracy of the identification, direction and delivery process of the new viral particles to specific cells of the patient’s organism in order for them to only act in the cells requiring treatment” Dr. Marc Güell states, who directs the Translational Synthetic Biology Laboratory at the UPF. 

As for Dr Noelia Ferruz’s group working on Artificial Intelligence for Protein Design at the IBMB-CSIC, it will focus on implementing language models to generate proteins with customizable properties. Ferruz notes that “these models will improve by being given feedback from the results obtained by the rest of the consortium members, thus becoming increasingly efficient.”

Harnessing gene therapy and gene editing tools is undoubtedly the most important challenge in the development of effective therapeutic alternatives when the target cell to be modified is the hematopoietic stem cell. “Generating effective vectors to genetically modify hematopoietic stem cells will allow us to tackle a large number of rare diseases that do not currently have an effective cure, and that are devastating for the affected patients and their friends and family” says Dr. José-Carlos Segovia, head of the Cellular Technology Department of the CIEMAT. 

Although this project focuses on making immunotherapies used to treat cancer and edit hematopoietic stem cells safer and more effective, which will allow rare diseases like hereditary anemia to be treated, in future, this technique could be applied to the treatment of autoimmune diseases and aging.

The Government’s TransMisiones Program

The TransMisiones program is a new public-private collaborative model, which is promoted by the Ministry of Science, Innovation and Universities to improve the impact that R&D&I has on society and steer research towards priority challenges.

The new program builds bridges between research centers and companies, promoting better coordination among the bids from the different agencies funding R&D (CDTI, AEI and ISCIII). The 2023 TransMisiones call for bids saw 40 projects approved, with a budget of €186.5 million.

Research Project PLEC2023-010243 funded by MICIU/AEI /10.13039/501100011033