Advanced therapies, adjuvants, and artificial intelligence (friday complete)

Advanced therapies, adjuvants, and artificial intelligence mark the third day of ISI 2026

The third day of ISI 2026 brought together national and international experts to discuss advanced therapies, vaccine platforms, adjuvants, and artificial intelligence applications in the development and production of immunobiologicals. Thursday’s program (May 8) was marked by debates on technological innovation, access to high-cost treatments, preparedness for future pandemics, and digital transformation in healthcare.

Gene therapies and CAR-T advance, but access remains a challenge

The day’s first panel addressed advances in advanced therapies for different diseases, focusing on gene therapy and the implementation of CAR-T therapies. Coordinated by Antônio Carlos Campos de Carvalho, from Fiocruz, the session brought together international specialists and representatives from Brazilian institutions involved in the clinical and translational development of these technologies.

Before the main presentations, two scientific studies were presented in oral poster format. Luiza de Macedo presented a study on CAR-T cell engineering using hybrid transposons as a non-viral alternative for genetic modification. The researcher highlighted that CAR-T therapy production remains highly complex, time-consuming, and expensive, reaching costs of approximately US$500,000 per patient. The study seeks to evaluate strategies capable of reducing costs and expanding the feasibility of these therapies. Next, Rafael de Freitas Azevedo Repossi presented research on CB1 receptor overexpression mediated by an AAV vector in an experimental model of retinal degeneration, discussing perspectives for the national development of gene therapies.

Participating remotely, Marina Cavazzana, from Assistance Publique Hôpitaux de Paris, presented experiences involving gene therapy for immunodeficiencies and hematological diseases. The researcher highlighted studies related to Severe Combined Immunodeficiencies (SCIDs), a rare group of genetic disorders characterized by the body’s inability to produce functional T and B cells, severely compromising patients’ immune systems. Marina also mentioned ongoing scientific collaborations with the Fernandes Figueira Institute (IFF/Fiocruz).

David Williams, from Boston Children’s Hospital, addressed advances and challenges in gene therapies targeting sickle cell disease. The presentation discussed curative strategies based on gene editing and cell therapy, as well as the regulatory, financial, and manufacturing challenges associated with implementing these technologies on a large scale.

Closing the panel, Nelson Hamerschlak, from Hospital Israelita Albert Einstein, presented experiences with the clinical implementation of CAR-T therapies in Brazil. The hematologist explained that T cells are collected from patients through leukapheresis and later genetically modified before being reinfused in a single dose. Hamerschlak highlighted that international clinical studies have demonstrated robust and long-lasting results for different approved CAR-T products, especially in pediatric patients. However, he emphasized that real-world clinical practice involves more complex patients, including elderly individuals, people with comorbidities, and patients with more aggressive tumors.

According to him, while the United States performs between 6,000 and 8,000 CAR-T infusions annually, Brazil currently records around 250 cases per year. The specialist stated that the future of the therapy depends on optimizing manufacturing processes, expanding therapeutic indications, developing new technologies, and, above all, increasing access for the Brazilian population.

During the final discussion, Antônio Carlos Campos de Carvalho asked David Williams about prospects for continuing a study published in 2022. The researcher replied that he intends to advance toward clinical trials but highlighted funding-related difficulties. Addressing Nelson Hamerschlak, the chair asked about strategies to expand national production of these therapies. The hematologist emphasized the importance of collaboration among Brazilian institutions, stating that the country still faces structural limitations for GMP-standard manufacturing, although preclinical initiatives involving vector development and new cellular platforms are already underway.

Adjuvants gain prominence in vaccine development

The day’s second panel discussed the strategic role of adjuvants in the development of new immunobiologicals and next-generation vaccines. Coordinated by Hugo Defendi, Deputy Director of Innovation at Bio-Manguinhos/Fiocruz, the session gathered international experts who presented new approaches involving delivery systems, lipid nanostructures, and glycobiology applied to immunization.

Opening the session, Barbara Araujo Nogueira presented a comparative study on immunization against Acinetobacter baumannii using OMV and OmpA, analyzing the relationship between humoral response and functional protection.

Next, Francisco Ibanez presented results related to a Chagas disease vaccine adjuvanted with QS-21 GH, discussing immunological efficacy and therapeutic perspectives.

Dennis Christensen, from Croda, delivered a lecture on the rational design of adjuvant systems for next-generation vaccines. The researcher discussed how different combinations of adjuvants can modulate specific immune responses and enhance the effectiveness of vaccines targeting different infectious diseases.

Aneesh Thakur, from VIDO/University of Saskatchewan, addressed the use of lipid nanostructures as a new platform for vaccine adjuvants. According to him, these technologies represent a new paradigm for global vaccines, enabling greater stability, immune targeting, and scalability in production.

Pi-Hui Liang, from ImmunAdd, presented research involving glycobiology applied to the development of new vaccine adjuvants. The lecture highlighted how carbohydrates and glycobiological interactions can influence immune responses and open new possibilities for vaccine design.

Closing the panel, Milena Akamatsu, from the Butantan Institute, presented the development of IB160, a squalene-based emulsion designed for influenza pandemic response. The researcher discussed the platform’s advances and its strategic potential for pandemic preparedness.

Artificial intelligence transforms research, industry, and pandemic preparedness

The next panel discussed the impact of artificial intelligence on technological development, industrial production, and preparedness for health emergencies. Coordinated by Eduardo Krempser, from Bio-Manguinhos/Fiocruz, the session brought together researchers, digital transformation specialists, and representatives of international initiatives.

Opening the scientific presentations, Caio Velloso presented a study on the application of artificial intelligence and Retrieval-Augmented Generation (RAG) for regulatory analysis in the pharmaceutical industry. The researcher explained that much of today’s regulatory documentation still depends heavily on human experts and that the study evaluated the performance of AI systems in supporting this process. According to him, the results indicate that hybrid models combining human expertise and AI agility show the greatest potential for efficiency.

Charlotte Deane, from St Anne’s College, opened the main panel discussing applications of bioinformatics, statistics, and artificial intelligence in drug discovery. The researcher emphasized that data are the main driver of AI and stated that, although artificial intelligence has transformative potential for drug development, it is not a universal solution. According to Charlotte, AI outperforms traditional methods in some scenarios, while conventional approaches still deliver better results in others.

Next, Caetano Penna, from the Center for Management and Strategic Studies (CGEE/MCTI), presented perspectives on the Brazilian Artificial Intelligence Plan (PBIA). The economist highlighted that Brazil already possesses important structural capacities in AI, especially in healthcare, chemistry, and biotechnology — sectors that have concentrated public investment and knowledge production.

Representing the Ministry of Health, Tiago Bahia Fontana presented initiatives related to digital transformation in Brazil’s Unified Health System (SUS) and strategies for health data integration within the PBIA framework.

Nestor Ayala discussed the impacts of artificial intelligence on industrial transformation and the transition from Industry 4.0 to Industry 5.0. According to him, the current focus is no longer solely on automation but increasingly on collaboration between humans and intelligent systems. The researcher stated that the main challenge today is organizational rather than technological and highlighted the importance of gradual training and AI literacy processes within companies.

Closing the program, Newton Wahome, from CEPI, presented the concept of the “Pandemic Preparedness Engine,” a global AI-based platform aimed at preparing for future pandemics. The initiative seeks to accelerate vaccine development for emerging health threats within 100 days after the identification of a new pathogen.

According to Wahome, the platform would function as a kind of “ChatGPT for vaccine developers,” integrating genomic surveillance, epidemiological, and molecular modeling data to predict antigenic structures capable of generating effective immune responses. The project involves partnerships with institutions such as Houston Methodist Research Institute, the University of Leipzig, and UC Davis.

The researcher also highlighted that the platform incorporates biosafety mechanisms to prevent misuse of the technology and includes plans for the creation of “AI factories” — distributed computational centers in different regions of the world aimed at expanding global access to AI capabilities applied to healthcare.

During the final debate, Eduardo Krempser raised questions regarding data quality and data sharing for training AI systems, as well as the challenges of digital transformation in the healthcare sector. The speakers emphasized the importance of gradual technological implementation processes and international collaboration to ensure the safe and equitable development of these tools.

Extracellular matrix engineering and translational innovation close ISI

Closing the scientific program of ISI 2026, the symposium’s final panel addressed Brazilian perspectives on extracellular matrix engineering for neural regeneration, focusing on the development of polylaminin technology. Coordinated by Rosane Cuber, Director of Bio-Manguinhos/Fiocruz, the session brought together Tatiana Coelho de Sampaio, from UFRJ, and Rogério Almeida, Vice President of Research and Development at Cristália Laboratories.

Opening the panel, Rosane Cuber emphasized the importance of encouraging not only basic research but also strategies capable of transforming scientific knowledge into concrete solutions for the population. According to her, translational science represents a fundamental link between universities, innovation, and access to healthcare.

Tatiana Sampaio began her presentation by explaining the role of laminin, a protein present in the extracellular matrix and essential for the structural organization of tissues. The researcher described how different types of extracellular matrix function within the body, including filling structures and membrane-like layers responsible for tissue compartmentalization.

According to Tatiana, laminin is present in virtually all tissues of the body and plays an especially important role in peripheral nerves, which have regenerative capacity. In the central nervous system, including the brain and spinal cord, however, this regeneration does not occur naturally. Based on this observation, the researcher presented the central hypothesis of her work: whether the presence of laminin could favor regenerative processes in spinal cord injuries.

Next, Rogério Almeida presented the collaborative trajectory between Cristália and UFRJ in the development of polylaminin technology. The executive highlighted that the partnership represents a still uncommon example in Brazil of translating academic research into industrial application and therapeutic development.

Presenting Cristália’s trajectory, Rogério emphasized the company’s history of national pharmaceutical innovation, including more than one hundred granted patents and projects developed in partnership with public institutions. He cited as an example the development of somatropin in partnership with Bio-Manguinhos, a process that took 14 years before reaching Brazil’s public healthcare system (SUS).

According to the researcher, one of the main challenges for health innovation in Brazil remains the limited connection between universities and industry, causing many promising projects to remain restricted to academia without advancing toward therapeutic applications.

Rogério explained that the invention process related to polylaminin began in 1999, while formal cooperation between UFRJ and Cristália started a few years later. The patent was granted in 2025, and clinical studies are expected to begin soon. He emphasized that the project represents a radical innovation, requiring unprecedented evaluations by Anvisa due to the lack of previous international references regarding the safety of intracellular application of the technology.

Closing the symposium, Rosane Cuber stated that the panel symbolically represented the event’s objectives by bringing together basic science, innovation, and practical application. The director also highlighted structural challenges in Brazilian academic training, pointing out that biomedical fields still provide limited education focused on translating scientific knowledge into products and therapies.

Rosane further emphasized that cultural barriers between universities and industry persist, both among researchers and within the productive sector, hindering strategic partnerships for health innovation. According to her, strengthening these connections is essential for scientific discoveries to effectively reach the population through new medicines and therapies.

Text: Marcela Dobarro
Imagem: André Rocha