Research
23-04-2024
X-PAND: a new multicenter project on gene therapy
Publication date: 13-12-2022
Updated on: 28-04-2023
Topic: Research
Estimated reading time: 1 min
Article Author
Sofia Erica RossiMedical Editor
Luigi NaldiniEditor and Translator
Viktoryia LuhakovaOspedale San Raffaele announces the launch of the four-year multicenter international project X-PAND (GA 101070950), funded with approximately 4 million euros by the European Union under the Horizon Europe - European Innovation Council program.
The project aims to improve current hematopoietic stem cell gene therapy protocols by using the support of the most innovative genetic and epigenetic analysis techniques. The goal of the Ospedale San Raffaele' researchers is to move stem cell gene therapy toward new, efficient, and increasingly safe clinical applications.
Members of the X-PAND project
The X-PAND consortium is coordinated by Luigi Naldini, director of the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy, and Bernhard Gentner, associate professor at the University of Lausanne and group leader at the SR-Tiget. The project also has scientific input from the laboratory of Raffaella Di Micco, group leader at SR-Tiget.
Other international partners of the consortium are:
- University of Navarre (Pamplona, Spain);
- University Hospital of Freiburg (Germany);
- Moncloa Center for Energy, Environmental and Technological Research (Madrid, Spain);
- Miguel Servet Public Foundation (Pamplona, Spain);
- University Hospital of Lausanne (Switzerland).
Successes of gene therapy
Hematopoietic stem cells and their ability to generate all types of blood cells while maintaining a high potential for self-renewal are the basis of many cutting-edge cell therapies.
Because of these characteristics, they represent an effective tool for cell and gene therapy approaches to treat various diseases, including genetic diseases that directly affect hematopoiesis (i.e., blood cell formation and maturation), immunity, systemic disorders, and cancer.
In recent years, hematopoietic stem cell-based gene therapy treatments have already proven effective in the treatment of serious genetic diseases: for example, Strimvelis for the treatment of ADA-SCID and Libmeldy for metachromatic leukodystrophy (both developed at SR-Tiget).
Intent of the X-PAND project
However, a substantial bottleneck in the field of gene therapy is the development of protocols for the growth and differentiation of hematopoietic stem cells in ex vivo cultures and for effective genetic manipulation that does not compromise their stemness, that is, their ability to differentiate into many different cell types. In addition, some potential effects can only be detected in the laboratory, retrospectively as a result of time-consuming transplantation experiments.
Therefore, unraveling the genetic and biological identity of these cells, including through the support of multi-omics analysis techniques, would lead to extraordinary improvements for their future therapeutic use in the clinic.
Importance of multidisciplinary team
Following this logic, the X-PAND consortium decided to bring together a multidisciplinary team of international scientists with extensive experience in gene transfer and in vitro editing, stem cell characterization and expansion technologies, multi-omic data analysis, and clinical application in the field of monogenic diseases and cancer.
This integrated vision will enable the 'big leap' in the correction of defective genes and the development of new cell and gene therapies with hematopoietic stem cells for increasingly safe and effective clinical applications.