Discussion highlights advances in gene therapy and CAR-T, while underscoring challenges related to cost, scalability, and clinical implementation.
The session “Advanced Therapies across Disease Modalities: Viral Gene Delivery and CAR-T Implementation”, held during the 10th International Symposium on Immunobiologicals (ISI), brought together experts to discuss recent advances in advanced therapies — particularly gene therapy and CAR-T — and the challenges of scaling their implementation.
Chaired by Antônio Carlos Campos de Carvalho (Fiocruz), the session featured Marina Cavazzana (Assistance Publique – Hôpitaux de Paris), David Williams (Boston Children’s Hospital), and Nelson Hamerschlak (Hospital Israelita Albert Einstein).
Prior to the main session, two poster oral presentations provided relevant contributions. Luiza de Macedo Abdo presented a study on CAR-T cell engineering using hybrid transposons as a non-viral alternative, highlighting its potential to reduce production costs. Rafael de Freitas Azevedo Repossi presented research on AAV-mediated overexpression of the CB1 receptor in a mouse model of retinal degeneration, pointing to opportunities for nationally developed gene therapies.
Opening the session, Marina Cavazzana presented advances in gene therapy for immunodeficiencies and hematological diseases, with a focus on Severe Combined Immunodeficiencies (SCIDs). She highlighted the potential of these approaches to deliver curative treatments for rare genetic conditions.
David Williams then discussed curative strategies for sickle cell disease based on gene editing and cell therapy. While scientific progress has been significant, he emphasized key challenges related to regulation, financing, and large-scale implementation.
Closing the session, Nelson Hamerschlak brought a clinical perspective, addressing the implementation of CAR-T therapies in Brazil. He explained the process from cell collection and genetic modification to reinfusion, and highlighted the strong outcomes observed in international clinical studies.
Despite these advances, he pointed to a major gap in access: while thousands of treatments are performed annually in countries such as the United States, the number of procedures in Brazil remains limited. Expanding access, he noted, will depend on reducing costs, strengthening local production, and expanding clinical infrastructure.
Throughout the discussion, it became clear that while advanced therapies represent one of the most promising frontiers in medicine, their real-world impact still depends on overcoming structural barriers related to cost, production, and access.
The session reinforced that the future of these technologies lies not only in scientific innovation, but in making them viable and accessible across different health systems.
Text: Marcela Dobarro
Image: Monara Barreto
