SUPERSIST

Towards clinical translation of new gene targeting technologies for correcting inherited mutations and empowering adoptive immunotherapy of cancer

Coordinatore	UNIVERSITA VITA-SALUTE SAN RAFFAELE    more  Organization address address: Via Olgettina 58 city: MILANO postcode: 20132 contact info Titolo: Dr. Nome: Maria Rosa Cognome: Pedrazzi Email: send email Telefono: +39 02 2643 4845 Fax: +39 02 2643 4717
Nazionalità Coordinatore	Italy [IT]
Totale costo	7˙877˙054 €
EC contributo	5˙999˙997 €
Programma	FP7-HEALTH Specific Programme "Cooperation": Health
Code Call	FP7-HEALTH-2013-INNOVATION-2
Funding Scheme	CP-FP
Anno di inizio	2013
Periodo (anno-mese-giorno)	2013-05-01   -   2016-04-30

 Partecipanti

#	participant	country	role	EC contrib. [€]
1	UNIVERSITA VITA-SALUTE SAN RAFFAELE  Organization address address: Via Olgettina 58 city: MILANO postcode: 20132 contact info Titolo: Dr. Nome: Maria Rosa Cognome: Pedrazzi Email: send email Telefono: +39 02 2643 4845 Fax: +39 02 2643 4717	IT (MILANO)	coordinator	1˙470˙000.00
2	MOLECULAR MEDICINE SPA  Organization address address: VIA OLGETTINA 58 city: MILANO postcode: 20132 contact info Titolo: Dr. Nome: Enrico Cognome: Cappelli Email: send email Telefono: +39 02 212 77302 Fax: +39 02 212 77 404	IT (MILANO)	participant	3˙000˙002.00
3	STICHTING HET NEDERLANDS KANKER INSTITUUT  Organization address address: PLESMANLAAN 121 city: AMSTERDAM postcode: 1066 CX contact info Titolo: Dr. Nome: Henri Cognome: Van Luenen Email: send email Telefono: +31 20512209	NL (AMSTERDAM)	participant	573˙744.00
4	DEUTSCHES KREBSFORSCHUNGSZENTRUM  Organization address address: Im Neuenheimer Feld 280 city: HEIDELBERG postcode: 69120 contact info Titolo: Dr. Nome: Ina Cognome: Krischek Email: send email Telefono: +49 6221 422700 Fax: +49 6221 422708	DE (HEIDELBERG)	participant	478˙126.00
5	UNIVERSITY COLLEGE LONDON  Organization address address: GOWER STREET city: LONDON postcode: WC1E 6BT contact info Titolo: Ms. Nome: Malgorzata Cognome: Kielbasa Email: send email Telefono: +44 2031083064 Fax: +44 20 78132849	UK (LONDON)	participant	478˙125.00

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 Obiettivo del progetto (Objective)

'Substantial evidence supports the therapeutic potential of ex vivo gene therapy based on Hematopoietic Stem Cell (HSC) or T lymphocytes to treat inherited diseases or cancer. Yet, the intrinsic limitations of current gene replacement approaches based on semi-randomly integrating vectors, and the occurrence in some clinical trials of severe adverse events related to gene transfer, prevent safe deployment and broad application of gene therapy. This project aims to overcome these limits by exploiting the results of an earlier highly successful integrated project (PERSIST), which demonstrated the potential of gene targeting technologies based on engineered nucleases to provide radical new solutions to these hurdles. By homology-driven repair of a nuclease-targeted dysfunctional gene, we will insert a functional copy downstream its own endogenous promoter in HSC, thus restoring both function and physiological expression control. For adoptive T-cell therapy, we will combine nuclease-mediated disruption of the endogenous TCR genes with transfer of tumor-specific TCR, thus editing T-cell specificity at the genetic level. We will develop these innovative approaches into robust, scalable and clinically ready processes for safe ex vivo genetic modification of hematopoietic cells, and apply them to the treatment of paradigmatic diseases that provide a favourable risk-benefit ratio for clinical testing. Because both HSC and T-cell based gene therapy strategies share methodological aspects and technological challenges, we will address them systematically by involving a SME which holds unique know-how and top-level expertise in vector manufacturing and HSC and T-cell processing, as already established for our previous and ongoing gene therapy trials. Our findings will lead to the design of new clinical trials to provide durable benefits or even a cure to patients suffering from severe and otherwise fatal diseases, and representing a template for broaden application in medicine.'