2025 AWARDS WINNERS

Outstanding Achievement Award

• Juan Bueren, Ciemat, Madrid

Supported by:

Professor Juan A. Bueren is a world leader in the field of gene therapy for rare diseases and hematopoietic stem cell research. His work has been fundamental in the development of innovative treatments that have transformed the clinical management of severe diseases such as Fanconi Anemia and Leukocyte Adhesion Deficiency (LAD). Among his major contributions is the development of the first successful gene therapy treatment for patients with Fanconi Anemia, without the need for prior conditioning, eliminating the high toxicity and long-term side effects associated with conventional transplantation. This milestone has changed the paradigm in FA treatment and is currently under revision by the EMA and FDA as a novel treatment for FA patients with unmet clinical need. He also led the preclinical studies and clinical trial of gene therapy for Leukocyte Adhesion Deficiency (LAD), a devastating disease affecting infants, which resulted in the cure of all the patients treated and is currently under review by the FDA as a novel treatment for patients without a suitable donor

Globally, he has an outstanding scientific production, with more than 500 indexed publications, 183 of them in PubMed, and more than 7,800 citations. His research has been published in high-impact journals such as The Lancet, Nature Medicine, Cell Stem Cell, New England Journal of Medicine, consolidating his leadership in the field of gene therapy. His contribution to the scientific community was also reflected by serving as President of the European Society of Gene and Cell therapy.

The different contributions have resulted in patent applications (12) and orphan drugs designations (6). His achievements have been translated into a high technology transfer activity with the licensing of two gene therapies to Rocket Pharmaceuticals, now under review by the EMA and FDA for their commercialization approval. He co-foundered Kijy Therapeutics, a company dedicated to the application of genetically modified mesenchymal stem cells for the treatment of inflammatory diseases. His collaboration with industry has been fundamental to the clinical translation of advanced therapies into the real world, with tremendous impact on global health.

His commitment to patients has extended beyond academic research, leading the creation of the Fanconi Anemia Foundation in Spain and fostering collaboration between researchers, clinicians and families. For this reason, he has been honored with the Distinguished Service Award from the Fanconi Anemia Research Foundation (FARF), highlighting his real impact in improving the quality of life for patients with genetic diseases.

Career Progression Awards

• Michela Milani,San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), Milan

Ph.D. in molecular medicine at Vita-Salute San Raffaele University of Milan, Michela contributed to the advancement of gene therapy for hemophilia and to the understanding of the biology of liver stem cells at San Raffaele Telethon Institute for Gene Therapy. She developed new lentiviral vectors tailored for in vivo use to achieve efficient and safe gene transfer in non-human primates. Recently, she broadened her interests in in vivo lentiviral vector-mediated gene therapy by showing effective and stable gene transfer into hematopoietic stem and progenitor cells in newborn mice. Currently, she is a Project Leader at the San Raffaele Telethon Institute for Gene Therapy, where she will continue developing new strategies for improved in vivo genetic engineering."

• Ivana Trapani, Telethon Institute of Genetics and Medicine (TIGEM)

Dr. Ivana Trapani is Group Leader at the Telethon Institute of Genetics and Medicine (TIGEM) and Assistant Professor at the University of Naples “Federico II.” She earned her PhD in Molecular Medicine from the European School of Molecular Medicine and continued her research thereafter at TIGEM. In 2021, she began her independent career focusing on development of both gene-dependent and gene-independent strategies, using adeno-associated viral (AAV) vectors, to treat diseases caused by mutations in large genes.

Early Career Investigator Awards

• Gabriele Casirati, Boston Children's Hospital

Dr. Gabriele Casirati is a hematologist and instructor at Boston Children’s Hospital and Dana-Farber Cancer Institute. He specializes in immune engineering and novel gene editing strategies, with a focus on enhancing targeted immunotherapies, hematopoietic stem cell transplantation and gene therapy. Dr. Casirati’s research has contributed to the development of epitope editing approaches aimed at making bone marrow transplants and CAR-T cell therapies safer and more effective.