MEUSIX

Clinical trial of gene therapy for MPS VI - a severe lysosomal storage disorder

Coordinatore	FONDAZIONE TELETHON    more  Organization address address: VIA VARESE 16/B city: ROMA postcode: 185 contact info Titolo: Mrs. Nome: Irene Cognome: Mearelli Email: send email Telefono: +39 06 44015308
Nazionalità Coordinatore	Italy [IT]
Totale costo	7˙877˙621 €
EC contributo	5˙995˙041 €
Programma	FP7-HEALTH Specific Programme "Cooperation": Health
Code Call	FP7-HEALTH-2012-INNOVATION-1
Funding Scheme	CP-FP
Anno di inizio	2012
Periodo (anno-mese-giorno)	2012-12-01   -   2017-11-30

 Partecipanti

#	participant	country	role	EC contrib. [€]
1	FONDAZIONE TELETHON  Organization address address: VIA VARESE 16/B city: ROMA postcode: 185 contact info Titolo: Mrs. Nome: Irene Cognome: Mearelli Email: send email Telefono: +39 06 44015308	IT (ROMA)	coordinator	1˙639˙600.00
2	REGENX BIOSCIENCES LLC  Organization address address: 17 TH STREET NW 750 SUITE 1100 city: WASHINGTON DC postcode: 2006 contact info Titolo: Dr. Nome: Karen Cognome: Kozarsky Email: send email Telefono: +01 202 778 2366	US (WASHINGTON DC)	participant	1˙709˙809.00
3	UNIVERSITA DEGLI STUDI DI NAPOLI FEDERICO II.  Organization address address: Corso Umberto I 40 city: NAPOLI postcode: 80138 contact info Titolo: Mrs. Nome: Gaetana Cognome: Errico Email: send email Telefono: +39 0817463365 Fax: +39 0817463116	IT (NAPOLI)	participant	1˙060˙080.00
4	GENOSAFE SAS  Organization address address: RUE DE L'INTERNATIONALE 1 city: EVRY postcode: 91000 contact info Titolo: Dr. Nome: Severine Cognome: Pouillot Email: send email Telefono: +33 1 69 74770 Fax: +33 1 69471161	FR (EVRY)	participant	687˙274.00
5	INFORMA SRL  Organization address address: VIA DEL COMMERCIO 36 city: ROMA postcode: 154 contact info Titolo: Dr. Nome: Francesca Cognome: Incardona Email: send email Telefono: +39 06 57589250	IT (ROMA)	participant	300˙000.00
6	ERASMUS UNIVERSITAIR MEDISCH CENTRUM ROTTERDAM  Organization address address: 's Gravendijkwal 230 city: ROTTERDAM postcode: 3015CE contact info Titolo: Prof. Nome: Ans Cognome: Van Der Ploeg Email: send email Telefono: +31 10 7037254	NL (ROTTERDAM)	participant	241˙278.00
7	HACETTEPE UNIVERSITESI  Organization address address: HACETTEPE UNIVERSITESI BEYTEPE KAMPUSU REKTORLUK BINASI city: CANKAYA ANKARA postcode: 6800 contact info Titolo: Prof. Nome: Hatice Serap Cognome: Sivri Email: send email Telefono: +90 312 3051141 Fax: +90 312 3100863	TR (CANKAYA ANKARA)	participant	240˙000.00
8	UNIVERSITA' DEGLI STUDI DI MILANO-BICOCCA  Organization address address: PIAZZA DELL'ATENEO NUOVO 1 city: MILANO postcode: IT-20126 contact info Titolo: Dr. Nome: Paola Cognome: Di Rienzo Email: send email Telefono: +39 02 64488351 Fax: +39 02 64486012	IT (MILANO)	participant	117˙000.00

Mappa

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 Obiettivo del progetto (Objective)

'Mucopolysaccharidosis VI (MPS VI, or Maroteaux-Lamy syndrome; OMIM #253200) is a rare lysosomal storage disease caused by deficient activity of arylsulfatase B (ARSB). MPS VI is characterized by growth retardation, corneal clouding, cardiac valve disease, organomegaly, skeletal dysplasia, without central nervous system involvement. Thus, systemic therapies targeting peripheral organs have the potential to fully correct the MPS VI phenotype. Enzyme replacement therapy, the current treatment for MPS VI, requires weekly infusions of a costly enzyme and has limited efficacy on bone and corneal disease. Based on the encouraging preclinical results generated by our group, gene therapy based on a single intravascular administration of adeno-associated viral (AAV) vectors targeting liver has the potential to provide a lifelong source of ARSB. The MeuSIX consortium plans to conduct a multicenter phase 1/2 clinical trial to investigate the safety and efficacy of AAV-mediated gene therapy in patients with MPS VI. An orphan drug designation (ODD) has been obtained from both the European Medicinal Agency and the US Food and Drug Administration for the MPS VI therapeutic AAV vector. The results from this clinical trial proposed by the MeuSIX consortium has the potential to have a tremendous impact on the natural history of MPSVI and to significantly improve the quality of life of the affected patients. Moreover, the approach developed may facilitate the development of similar approaches for other inborn errors of metabolism.'