2025 AWARDS WINNERS

Outstanding Achievement Award

• Juan Bueren, Ciemat, Madrid

Professor Juan A. Bueren is a leading global expert in gene therapy and hematopoietic stem cell research, with pioneering contributions to treatments for rare diseases such as Fanconi Anemia (FA) and Leukocyte Adhesion Deficiency (LAD).

He developed the first successful gene therapy for FA without prior conditioning—avoiding the toxicity of traditional transplantation—now under EMA and FDA review. He also led preclinical and clinical development of gene therapy for LAD, achieving full cures in treated infants, with FDA evaluation underway.

Juan has an extensive scientific record, with over 500 publications (183 in PubMed) and more than 7,800 citations in top-tier journals including The Lancet, Nature Medicine, Cell Stem Cell, and NEJM. He has served as President of the European Society of Gene and Cell Therapy.

His work has led to 12 patent applications, 6 orphan drug designations, and successful technology transfer, including licensing two gene therapies to Rocket Pharmaceuticals. He co-founded Kijy Therapeutics, advancing genetically modified mesenchymal stem cell therapies. Beyond research, he founded the Fanconi Anemia Foundation in Spain and has been recognized with the FARF Distinguished Service Award for his contributions to patients and families affected by 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.