AN INNOVATIVE APPLICATION
OF HEMATOPOIETIC STEM CELL EXPANSION FOR GENE THERAPY

IN A NUTSHELL

The technological breakthrough of X-PAND is to clarify the identity of ex-vivo expanded hematopoietic stem cells (HSC) through iterative cell sorting, multiomics single cell profiling and to apply this knowledge to a state-of-the-art toolbox of genetic engineering technologies.
This innovative multidisciplinary procedure would maximize the chances of delivering a safer next-generation hematopoietic stem cell-based gene therapy product, extending its application to new disease contexts.

WORKPLAN

WP1

Enhancing the scope of gene addition and gene editing with translatable and sustainable ex vivo HSC expansion

WP2

Novel in vitro multiomic approaches to replace in vivo animal models 

WP3

Genome and epigenome integrity and HSC response to editing

WP4

Optimized genetic correction of HSCs for cancer-prone monogenic diseases and novel “gain-of-function” engineering of HSC against cancer

WP5

Multiomics HSC readouts: bioinformatic data integration & storage

WP6

Exploitation, dissemination & communication

WP7

Management and scientific coordination

WP8

Portfolio Activities

CONSORTIUM

Ospedale San Raffaele

Milan, Italy

Universitätsklinikum Freiburg

Freiburg, Germany

Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas

Madrid, Spain

Fundación Miguel Servet

Pamplona, Spain

Universidad de Navarra

Pamplona, Spain

Lausanne University Hospital

Lausanne, Switzerland

Università Vita-Salute San Raffaele

Milan, Italy

NEWS