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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.

Field of science

  • /social sciences/economics and business/business and management/commerce
  • /medical and health sciences/basic medicine/pharmacology and pharmacy/pharmaceutical drug
  • /medical and health sciences/medical biotechnology/genetic engineering/gene therapy
  • /natural sciences/biological sciences/genetics and heredity/genome
  • /medical and health sciences/clinical medicine/ophthalmology

Call for proposal

H2020-SC1-2018-Single-Stage-RTD
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Funding Scheme

RIA - Research and Innovation action

Coordinator

FONDAZIONE TELETHON
Address
Via Varese 16/B
00185 Roma
Italy
Activity type
Research Organisations
EU contribution
€ 5 027 968,75

Participants (7)

UNIVERSIDAD POMPEU FABRA
Spain
EU contribution
€ 2 767 377,50
Address
Placa De La Merce, 10-12
08002 Barcelona
Activity type
Higher or Secondary Education Establishments
UNIVERSITA DEGLI STUDI DI TRENTO
Italy
EU contribution
€ 1 333 798,75
Address
Via Calepina 14
38122 Trento
Activity type
Higher or Secondary Education Establishments
TECHNISCHE UNIVERSITAET DRESDEN
Germany
EU contribution
€ 1 107 000
Address
Helmholtzstrasse 10
01069 Dresden
Activity type
Higher or Secondary Education Establishments
VRIJE UNIVERSITEIT BRUSSEL
Belgium
EU contribution
€ 1 328 412,50
Address
Pleinlaan 2
1050 Brussel
Activity type
Higher or Secondary Education Establishments
UNIVERSITE DE NANTES
France
EU contribution
€ 1 107 500
Address
Quai De Tourville 1
44035 Nantes Cedex 1
Activity type
Higher or Secondary Education Establishments
LUDWIG-MAXIMILIANS-UNIVERSITAET MUENCHEN
Germany
EU contribution
€ 1 106 062,50
Address
Geschwister Scholl Platz 1
80539 Muenchen
Activity type
Higher or Secondary Education Establishments
THE GENERAL HOSPITAL CORPORATION
United States
EU contribution
€ 1 217 710
Address
Fruit Street 55
02114 Boston Ma
Activity type
Other