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Our mission is to develop and produce advanced therapies, bringing innovation to clinical practice in a sustainable, efficient and equitable way for pediatric patients with rare diseases.
The Advanced Therapies Platform (ATP) at the SJD Barcelona Children's Hospital is a state-of-the-art, 1,280 m² facility designed to translate cutting-edge research in advanced therapies into clinical-grade treatments through the most rigorous and high-quality standards.
As described by Professor Alain Fischer, a pioneer in pediatric gene therapy, 'there are very few like this in the world', referring to our hospital's platform after his visit to the facilities in June 2025. These facilities are among the largest hospital infrastructures in the world for the manufacturing of advanced therapies.
What are advanced therapies?
Advanced therapies or Advanced Therapy Medicinal Products (ATMPs) represent a new paradigm in modern medicine, encompassing innovative genetic, cellular and tissue engineering therapies designed to treat patients with significant unmet medical needs. These treatments offer hope and new opportunities to children with rare and complex diseases for which no effective alternative treatments exist.
A prominent example is the manufacture of CAR-T cells, which has successfully provided treatment to patients with acute lymphoblastic leukemia who had not responded to conventional treatment.
To produce advanced therapy medicinal products, it is necessary to follow Good Manufacturing Practices (GMP) to ensure compliance with the manufacturing standards required at a national and international level. The Platform at the SJD Barcelona Children's Hospital holds GMP certification and authorisation from the Spanish Agency of Medicines and Medical Devices (ES/102I/24) to manufacture CAR-T cells for both pediatric and adult patients.
Production at the SJD Barcelona Children's Hospital
Since obtaining GMP certification in August 2024, the Advanced Therapies Platform has manufactured the CAR-T ARI0001 for pediatric and adult patients, validated additional advanced therapy medicines, and initiated in-house manufacturing of lentiviral vectors (LVVs) in compliance with GMP standards. Furthermore, the team has grown significantly, strengthening the multidisciplinary expertise needed to support the long-term development and sustainability of the facilities.
The manufacture of advanced therapy medicines for rare diseases represents a highly complex scientific, technological and economic challenge, due to low prevalence, underdiagnosis, the limited number of patients and scarce commercial incentives. Translating academic research into clinical applications requires multidisciplinary expertise, specific infrastructure and substantial financial investment, which jeopardises long-term sustainability.
Moreover, the pharmaceutical industry often overlooks rare diseases due to their poor commercial viability, meaning that affected patients do not receive potentially curative treatments.
Manufacturing and Import Authorisation (MIA)
The overall objective of the Advanced Therapies Platform, as an academic-hospital infrastructure, is to facilitate access to these medicines for pediatric patients with rare diseases. Its activity has expanded beyond academic manufacturing to establish strategic collaborations with biotechnology SMEs that require support in GMP-compliant manufacturing.
For this reason, the Platform has been granted Manufacturing and Import Authorisation (MIA). This accreditation authorises the facility to manufacture, carry out quality controls, certify and release advanced therapy medicinal products under GMP conditions for clinical use, as well as to import these medicinal products from countries both within and outside the European Union, as well as supporting exports for international clinical initiatives, in accordance with national and European regulatory requirements.
Facilities
The SJD Barcelona Children's Hospital's Advanced Therapies Platform spans 1,280 m² and comprises a GMP manufacturing area, a preclinical and pre-GMP R&D space, and a cryogenic area:
- The GMP manufacturing area, covering 400 m², features four cleanrooms (grade B/C) specifically designed for the manufacture of different types of advanced therapies (cellular, gene and tissue engineering) and lentiviral vectors (LVV). It also includes an in-process quality control laboratory, a microbiology laboratory, changing rooms and a storage area, all under controlled and certified environmental conditions.
- The R&D space spans 200 m² and features 25 laboratory benches, two cell culture rooms, two equipment rooms and a storage area. It is closely connected to the GMP area, providing a process development environment that enables seamless scale-up from preclinical/pre-GMP studies to clinical manufacturing. It also hosts researchers from the GEMDIP programme, a study on Immunodeficiency Diseases in Pediatrics at the SJD Research Institute. Furthermore, since 2024, the PTA hosts the biotechnology start-up Integra Therapeutics under a service agreement whereby the platform provides access to the R&D space, equipment and part of the administrative area.
- The Platform has a 40 m² cryogenic area for the storage of advanced therapy medicinal products, biological materials and sensitive therapies under GMP conditions, with restricted access. This cryogenic storage area also includes a space for the SJD Barcelona biobank.
The Advanced Therapy Platform has a dedicated administrative area with offices and co-working spaces.
Team
Our projects
Early-phase clinical trial to evaluate the safety of combining immunotherapy with tumor lysate-pulsed dendritic cells together with CAR-T cells targeting IL13Rα2, in patients with newly diagnosed diffuse intrinsic pontine glioma (DIPG).
Phase II clinical trial evaluating the efficacy and safety of var-cel (ARI-0001) in pediatric B-cell acute lymphoblastic leukemia in first relapse or refractory disease.
National, multicentre phase Ib clinical trial, designed as a prospective, open-label, non-randomised, exploratory basket study to evaluate the safety of varnimcabtagene autoleucel (ARI-0001) in pediatric systemic autoimmune diseases (LUp, MDJ, esJS). This project was awarded competitive grants from PICI (ISCIII) in 2025.
International phase II clinical trial in pediatric B-cell acute lymphoblastic leukemia (B-ALL) in first high-risk relapse. Pediatric Investigation Plan of the European Medicines Agency (EMA) for ARI-0001. The project is funded by the competitive ATTRACT grant and aims to treat 70 patients across five countries following a decentralised Point-of-Care model. The goal of ARI-chALL is to advance CAR-T therapy ARI-0001 in children with ALL in first relapse in order to obtain EMA approval and make an effective treatment that improves quality of life widely accessible.
Multicentre phase II clinical trial led by the Ospedale Pediatrico Bambino Gesù (IRCCS) in Rome (Italy). This study targets pediatric patients and young adults with very high-risk (VHR) CD19+ B-cell precursor acute lymphoblastic leukemia (ALL) in first relapse or refractory disease.
Development of a novel ex vivo gene therapy to treat a rare primary immunodeficiency of the major histocompatibility complex class II (MHCII-D). This project is supported by the following grants: FIS 2024 from the ISCIII (File No.: PI24/00424), PERIS (Pla Estratègic de Recerca i Innovació en Salut 2025) from the Generalitat de Catalunya, and the internal SJD Torrons Vicens-Rac1 fellowship.
'End-to-end digital coordination and traceability for cellular and gene therapy programmes, developed from within Hospital Sant Joan de Déu, one of Europe's leading academic pediatric hospitals.' This project has been selected as one of the five projects for the 2026 edition of the i4kids Acceleration Programme.
'With the creation of the Advanced Therapies Platform, we have the infrastructure to develop and manufacture innovative therapies and offer new therapeutic hope for patients with extremely complex diseases.'
'We are committed to transforming research into innovative therapies that offer new opportunities for patients, ensuring the highest quality and safety at every step of the process.'
Related departments
Our aim is to facilitate the work of researchers, laboratories and companies who need to assess the efficacy of treatments and products.
We focus on patients and their requirements, rather than just on the medicine itself.
We are a national and international reference centre in the provision of healthcare and conducting research into pediatric cancer.
We are referents in the integral treatment of pediatric rheumatic diseases.
Treatment for Allergies and Immunodeficiencies in children from 0 to 18 years of age, with patient follow-up to prevent admission to the A&E Department.
We are specialists in bone marrow translpants in children.
Alliances and networks
The Platform is part of several national and international advanced therapies networks. Within Catalonia and on a national level, it works with the Catalan advanced therapy network promoted by BIOCAT, and with the Spanish consortia CERTERA and RICORS/TERAV+, promoted respectively by the Carlos III Health Institute. It also has an alliance with CIEMAT for the ex vivo gene therapy project, as well as with the Fundación Jiménez Díaz (FJD).
On a European scale, it is part of a consortium for the production of CAR-T cells in pediatric oncohematology and of various ATMP initiatives, including PRECISEU (PeRsonalised medicine Empowerment Connecting Innovation ecoSystems across EUrope), a 23-million-Euro macro-initiative aimed at accelerating the adoption of personalised medicine and advanced therapies.
In addition, the Advanced Therapies Platform benefits from SJD Barcelona Children's Hospital's membership in 21 of the 24 ERN networks and in the pediatric hospital network ECHO. It also actively participates in multicentre clinical trials and cross-border initiatives, such as the European network of Centres of Excellence in Advanced Therapies, reinforcing its position as a European reference centre.
The construction of this infrastructure has only been possible thanks to the donors and collaborators of the Hospital's fundraising initiatives, as well as public funding and competitive grants from the Carlos III Institute and the Generalitat de Catalunya. These alliances allow us to remain at the forefront of the development, manufacture and clinical administration of advanced therapies.
Donors
Grants and public funding
