CORDIS
EU research results

CORDIS

English EN

Unlocking Precision Gene Therapy

Objective

Gene therapy has recently shown remarkable potential to offer definitive treatments for otherwise incurable diseases. Currently, seven gene therapy products have reached the market and many more are entering clinical testing for selected indications. Moreover, emerging technologies for targeted gene editing are complementing the scope of conventional gene transfer, opening the way to precise gene correction and making possible to silence, activate or recode any sequence of interest in the genome. However, in order to realize the full potential of these strategies and broaden application of gene therapy, the field has to solve several major hurdles, including: risk of insertional mutagenesis by gene transfer vectors, limited efficiency and durability of some gene correction strategies, off target effects of editing tools, poor tissue targeting and immune response to editing components and delivery vehicles. UPGRADE will offer radical new solutions to overcome these hurdles. We will exploit and further develop disruptive new technologies for precision gene and epigenome editing and for site-specific transgene insertion, and stringently characterize their specificity and cellular responses. We will combine these improved technologies with advanced viral and non-viral vectors enabling cell/tissue targeting and immune evasion, to generate prototypes of advanced medicinal products (AMP). The safety and efficacy profile of each AMP will be stringently validated in tissues (hematopoiesis, heart and skeletal muscle, liver, retina) and disease models (muscle wasting, storage and blood disorders, hypercholesterolemia) paradigmatic for unmet medical need and potential long-term cure, if the limitations of current gene therapy strategies are overcome. These AMPs represent versatile products portable to the treatment of several other diseases because of related pathogenesis or correction strategies, thus providing the basis for tackling diseases affecting large patient groups.
Leaflet | Map data © OpenStreetMap contributors, Credit: EC-GISCO, © EuroGeographics for the administrative boundaries

Coordinator

FONDAZIONE TELETHON

Address

Via Varese 16/B
00185 Roma

Italy

Activity type

Research Organisations

EU Contribution

€ 5 027 968,75

Participants (7)

Sort alphabetically

Sort by EU Contribution

Expand all

UNIVERSIDAD POMPEU FABRA

Spain

EU Contribution

€ 2 767 377,50

UNIVERSITA DEGLI STUDI DI TRENTO

Italy

EU Contribution

€ 1 333 798,75

TECHNISCHE UNIVERSITAET DRESDEN

Germany

EU Contribution

€ 1 107 000

VRIJE UNIVERSITEIT BRUSSEL

Belgium

EU Contribution

€ 1 328 412,50

UNIVERSITE DE NANTES

France

EU Contribution

€ 1 107 500

LUDWIG-MAXIMILIANS-UNIVERSITAET MUENCHEN

Germany

EU Contribution

€ 1 106 062,50

THE GENERAL HOSPITAL CORPORATION

United States

EU Contribution

€ 1 217 710

Project information

Grant agreement ID: 825825

Status

Ongoing project

  • Start date

    1 January 2019

  • End date

    31 December 2023

Funded under:

H2020-EU.3.1.3.

  • Overall budget:

    € 14 996 955

  • EU contribution

    € 14 995 830

Coordinated by:

FONDAZIONE TELETHON

Italy