The Vall d’Hebron Barcelona Hospital Campus and Biocat held a new session of the ATMP Catalonia Series, titled ‘ATMPs: From Preclinical Discovery to First-in-Human Trials’. The event, held at the auditorium of the Central Building of the Vall d’Hebron Research Institute (VHIR), brought together professionals from academia, clinical practice and industry to address the main challenges associated with the development of advanced therapy medicinal products (ATMPs).

Throughout the session, key topics were addressed, including the translation of basic research into clinical practice, regulatory requirements, clinical trial design and the implementation of these innovative therapies within healthcare systems.

The event opened with a brief institutional welcome by Dr. Jasmina Zanoncello, from the Internal Strategy Department at VHIR, and Miquel Salas, from Biocat’s innovation area, who introduced the objectives of the session and the broader framework of the ATMP Catalonia Series.

Clinical research as a driver of healthcare

The first presentation was delivered by Dr. Xavier Cañas, Director of Commercial and Contract Clinical Research at VHIR, who positioned clinical research as a key element for improving healthcare delivery. He emphasized that hospitals with strong research activity are better prepared to provide high-quality care and advocated for a patient-centered model that considers not only clinical outcomes but also patient experience and quality of life.

Cañas also highlighted the importance of patient involvement throughout the entire process, as well as the need to move towards more decentralized research models. During his intervention, he warned about Europe’s declining role in global clinical trial leadership, in contrast to the growing presence of other regions such as China.

CAR-T: from scientific innovation to clinical practice

Dr. Núria Díez, Medical Advisor in Clinical Research at Novartis, then presented the session ‘Kymriah and the CAR-T revolution: Lessons from the first FDA approval and future challenges’. Her presentation highlighted the long development pathway of these therapies, built on close collaboration between academia and industry, as well as the ability to navigate uncertainty in an environment that initially lacked a clear regulatory framework.

Díez pointed out that the development of CAR-T therapies required close coordination with regulatory authorities and clinical centers to establish standards in key areas such as safety, biological material management and center qualification. This process has ultimately enabled the transformation of a scientific innovation into a real therapeutic option for patients with limited alternatives.

At the same time, she emphasized the human dimension of these therapies, noting that for patients they often represent a unique opportunity. She also underlined the clinical, logistical and organizational complexity of early CAR-T trials, which require a high level of multidisciplinary coordination across hospital units.

A complex and demanding regulatory pathway

Dr. Sílvia Martín Lluesma, Head of the Advanced Therapies Program at the Vall d’Hebron Institute of Oncology (VHIO), addressed the regulatory requirements for ATMP development. During her presentation, she stressed that bringing these therapies from the laboratory to the patient is a long, complex and costly process that involves overcoming several critical stages.

She highlighted the importance of preparing a comprehensive preclinical package, defining robust clinical protocols, establishing appropriate dosing strategies, and ensuring product safety and quality in compliance with regulatory agency requirements. She also emphasized the need for adequate funding, infrastructure and trained personnel to make this development feasible, particularly in the context of rare diseases.

The importance of collaboration in ATMP development

The first roundtable of the session, titled ‘From preclinical stage to first-in-human trial: ethical, regulatory and clinical trial design challenges in ATMP development’, was moderated by Dr. Miguel Chillón, ICREA researcher, director of the UAB-VHIR Joint Unit and coordinator of the Innovative and Advanced Therapies CORE at VHIR.

The discussion brought together experts in ATMP development, regulation and manufacturing. Participants agreed that the transition from preclinical research to first-in-human trials requires comprehensive planning from the earliest stages of development.

In this regard, they highlighted the need to design robust yet flexible preclinical programs adapted to the diversity of advanced therapies, as well as to define from the outset key aspects such as mechanism of action, initial dosing and product safety.

They also stressed the importance of early interaction with regulatory agencies to properly guide development and avoid obstacles in later phases. Furthermore, they agreed that a lack of alignment among stakeholders may compromise the viability of these therapies beyond early-stage trials.

In this context, the discussion emphasized the need to strengthen collaboration between research, clinical and manufacturing teams, as well as to move towards more harmonized models and shared platforms to optimize resources and accelerate development.

The academic model in CAR-T development

After a coffee break, the session resumed with a presentation by Dr. Hugo Calderón, focused on the academic development model for advanced therapies. During his talk, he explained the fundamentals of CAR-T therapies, which are based on the genetic modification of a patient’s own T lymphocytes to redirect their ability to recognize and eliminate tumor cells.

This approach allows overcoming tumor immune evasion mechanisms and opens new therapeutic possibilities. However, Calderón also highlighted the complexity of the manufacturing process, as these therapies are produced on an individualized basis for each patient and require significant technical expertise and resources.

Uncertainty as a central challenge in advanced therapies

The session concluded with a second roundtable focused on managing uncertainty in ATMPs, addressing issues such as high-risk administration, long-term durability and immunogenicity.

During the discussion, participants highlighted the inherent complexity of these therapies, which often involve highly variable biological mechanisms and clinical responses that are difficult to predict over time. In this sense, the need to move towards more personalized and adaptive models was emphasized, taking into account the specific biological characteristics of each patient.

This panel included Dr. Josefina Cortés, principal investigator of the Rheumatology Research Group at VHIR; Dr. Alena Gros, head of the Cancer Immunology and Immunotherapy Group at VHIO; Dr. Miguel Mulet, CEO of Terafront; and again Dr. Hugo Calderón, researcher of the Immunogenetics and Immunotherapy in Autoinflammatory and Immune Responses Group at the Hospital Clínic de Barcelona.

Speakers underlined that addressing this uncertainty requires a combination of scientific innovation, robust data generation and close collaboration among all stakeholders, with the ultimate goal of ensuring that these therapies can reach patients safely and effectively.