Patient portal
We research the genetic origins of rare diseases to better understand them and develop new therapies, giving another chance to children who, unfortunately, have no effective treatment options at this moment in time.
Advanced Therapies are a new treatment option offering hope and new opportunities to children with rare diseases that have no effective treatment at this time. The SJD Barcelona Children’s Hospital boasts a dedicated platform to develop and manufacture new therapies for pediatric patients.
Of particular note is the project for the development and production of CAR-T cells, which are used in cases of acute lymphoblastic leukemia that has not responded to conventional treatment.
The Advanced Therapies Platform offers another chance at treatment for patients with complex disease. The platform not only allows for the internal production of treatment options, but it is also open to collaboration with other pharmaceutical institutions and companies, thereby extending the reach and impact of these innovative treatments.
What are advanced therapies?
Advanced therapies are a new category of medication. They are based on genes (gene therapy), cells (cell therapy) or tissues (tissue engineering), and use autologous (from the patient), allogenic (from a donor) or xenogeneic (from another species) components.
An example is CAR-T cell therapy, which genetically modifies the patient's T-lymphocytes to be able to recognise and destroy cancer cells.
The creation of these medications involves substantial manipulation, meaning their biological characteristics, their physiological functions or even their structural properties are intricately modified. That is why they must be manufactured according to Good Manufacturing Practices (GMP).
Manufacturing certification
In August 2024, the Advanced Therapies Platform at the SJD Barcelona Children's Hospital received certification of Good Manufacturing Practices (GMP) compliance, and received authorisation to produce CAR-T cell therapies for both adult and pediatric patients.
The European Medicines Agency (EMA) requires all manufacturers of drugs destined for the EU to comply with strict quality assurance standards, regardless of their location in the world. These Good Manufacturing Practices ensure that all medications:
- Are of consistent, uniform quality.
- Are suitable for their intended use.
- Comply with all established requirements regarding commercial authorisation or regarding clinical trials.
We are specialists in transforming scientific knowledge into real therapeutic solutions, combining our experience in cellular and molecular biology with the development of innovative medications. From preclinical studies through to clinical production, we follow the highest regulatory standards to guarantee safe and effective treatments.
Our focus on advanced therapies, including cell therapy, gene therapy and tissue engineering, allows us to bridge the gap between research and application in patients. Through a rigorous and multidisciplinary process, we promote the development of new treatments that revolutionise medicine and improve quality of life for patients.
Our projects
'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 specialists in bone marrow translpants in children.
Facilities
The Advanced Therapies Platform spans 1,280 square metres and houses four specially designed cleanrooms where different therapies are produced (cell, gene and tissue engineering). These facilities comply with Good Manufacturing Practice (GMP) guidelines to ensure maximum safety and product quality.
In addition, the platform boasts research and development areas, a cryogenics area and storage facilities, allowing researchers from SJD and elsewhere to work dynamically towards the development of new advanced therapies.
The cleanrooms are equipped with cutting-edge technology and are operated by highly trained professionals, ensuring a safe, controlled environment in which to produce these therapies.
Team
Partnerships
The center is part of the joint immunotherapy platform with Hospital Clinic, and is also a member of the Catalan Advanced Therapy Network, promoted by Biocat, and the Spanish state consortiums CERTERA and TERAV, promoted by the Carlos III Health Institute. It is also a member of a European consortium for the production of CAR-T cells in pediatric oncohematology.
These facilities are only possible thanks to fundraising efforts by donors and grants such as AGAUR from the Generalitat de Catalunya, also allowing for the purchase of specialist equipment.
These close partnerships allow SJD to be at the vanguard of advanced therapy development, benefitting from both the collaboration and the exchange of knowledge with leading institutions in the field.
Donors
Grants and public funding
