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UshTher SIGNED

Clinical trial of gene therapy with dual AAV vectors for retinitis pigmentosa in patients with Usher syndrome type IB

Total Cost €

EC-Contrib. €

Partnership

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Outcomes and
results

UshTher project word cloud

Explore the words cloud of the UshTher project. It provides you a very rough idea of what is the project "UshTher" about.

reconstitution laboratory previously transfer multicenter syndrome administrations caused rare expression cassette subretinal designation ushib model overcoming efficacy basis humans ush single capacity usher administration aav cargo combination retinal blindness untreatable grade smes manufacturing deafness tested vectors dual vivo agency gene bench leaders combined builds assembled cure drug envisages mouse performing performed twelve clinical implants data therapy competitive severe first halves orphan practices limited genes therapeutic trial biodistribution retina span medicines retinitis mutations vector bedside safe myo7a time pigmentosa received safety forms multinational innovative expertise good cochlear limitations patients frequent packaging ushther

Project "UshTher" data sheet

The following table provides information about the project.

Coordinator	FONDAZIONE TELETHON Organization address address: VIA VARESE 16/B city: ROMA postcode: 185 website: www.telethon.it contact info title: n.a. name: n.a. surname: n.a. function: n.a. email: n.a. telephone: n.a. fax: n.a.
Coordinator Country	Italy [IT]
Project website	http://www.ushther.eu/
Total cost	5˙998˙515 €
EC max contribution	5˙998˙515 € (100%)
Programme	1. H2020-EU.3.1.3. (Treating and managing disease)
Code Call	H2020-SC1-2017-Two-Stage-RTD
Funding Scheme	RIA
Starting year	2018
Duration (year-month-day)	from 2018-01-01 to 2022-12-31

Partnership

Take a look of project's partnership.

#	participants	country	role	EC contrib. [€]
1	FONDAZIONE TELETHON FONDAZIONE TELETHON Organization address address: VIA VARESE 16/B city: ROMA postcode: 185 website: www.telethon.it contact info title: n.a. name: n.a. surname: n.a. function: n.a. email: n.a. telephone: n.a. fax: n.a.	IT (ROMA)	coordinator	1˙783˙642.00
2	REITHERA SRL REITHERA SRL Organization address address: VIA DI CASTEL ROMANO 100 city: ROMA postcode: 128 website: www.okairos.com contact info title: n.a. name: n.a. surname: n.a. function: n.a. email: n.a. telephone: n.a. fax: n.a.	IT (ROMA)	participant	2˙566˙669.00
3	UNIVERSITA DEGLI STUDI DELLA CAMPANIA LUIGI VANVITELLI UNIVERSITA DEGLI STUDI DELLA CAMPANIA LUIGI VANVITELLI Organization address address: VIALE ABRAMO LINCOLN 5 city: CASERTA postcode: 81100 website: www.unina2.it contact info title: n.a. name: n.a. surname: n.a. function: n.a. email: n.a. telephone: n.a. fax: n.a.	IT (CASERTA)	participant	700˙567.00
4	GENOSAFE SAS GENOSAFE SAS Organization address address: RUE DE L'INTERNATIONALE 1 city: EVRY postcode: 91000 website: www.genosafe.com contact info title: n.a. name: n.a. surname: n.a. function: n.a. email: n.a. telephone: n.a. fax: n.a.	FR (EVRY)	participant	527˙952.00
5	HET ROTTERDAMS OOGHEELKUNDIG INSTITUUT BV HET ROTTERDAMS OOGHEELKUNDIG INSTITUUT BV Organization address address: SCHIEDAMSE VEST 160 D city: ROTTERDAM postcode: 3011 BH website: n.a. contact info title: n.a. name: n.a. surname: n.a. function: n.a. email: n.a. telephone: n.a. fax: n.a.	NL (ROTTERDAM)	participant	137˙445.00
6	INSTITUTO INVESTIGACION SANITARIA FUNDACION JIMENEZ DIAZ INSTITUTO INVESTIGACION SANITARIA FUNDACION JIMENEZ DIAZ Organization address address: AVENIDA DE LOS REYES CATOLICOS 2 city: MADRID postcode: 28040 website: http://www.fjd.es/instituto_investigacion contact info title: n.a. name: n.a. surname: n.a. function: n.a. email: n.a. telephone: n.a. fax: n.a.	ES (MADRID)	participant	136˙645.00
7	ASSOCIATION GENETHON ASSOCIATION GENETHON Organization address address: RUE DE L INTERNATIONALE 1 BIS city: EVRY postcode: 91002 website: www.genethon.fr contact info title: n.a. name: n.a. surname: n.a. function: n.a. email: n.a. telephone: n.a. fax: n.a.	FR (EVRY)	participant	91˙068.00
8	UNIVERSITA' DEGLI STUDI DI MILANO-BICOCCA UNIVERSITA' DEGLI STUDI DI MILANO-BICOCCA Organization address address: PIAZZA DELL'ATENEO NUOVO 1 city: MILANO postcode: 20126 website: www.unimib.it contact info title: n.a. name: n.a. surname: n.a. function: n.a. email: n.a. telephone: n.a. fax: n.a.	IT (MILANO)	participant	54˙525.00
9	STICHTING OOGZIEKENHUIS ROTTERDAM STICHTING OOGZIEKENHUIS ROTTERDAM Organization address address: SCHIEDAMSE VEST 180 city: ROTTERDAM postcode: 3011 BH website: www.eyeresearch-rotterdam.nl contact info title: n.a. name: n.a. surname: n.a. function: n.a. email: n.a. telephone: n.a. fax: n.a.	NL (ROTTERDAM)	participant	0.00

Map

Project objective

Usher syndrome (USH) is the most common combination of deafness and blindness due to retinitis pigmentosa. USHIB caused by mutations in the large MYO7A gene, is among the most severe and frequent forms of USH. While deafness can be improved with cochlear implants, blindness remains untreatable. Retinal gene therapy with AAV vectors is both safe and effective in humans, however it is limited by AAV cargo capacity which would not allow transfer of the large MYO7A expression cassette. UshTher objective is to test the safety and efficacy of a highly innovative gene therapy approach (dual AAV) in the retina of USHIB patients. The projects builds on data previously obtained by the coordinator that a single combined subretinal administration of dual AAV vectors, each packaging one of the two halves of a MYO7A expression cassette, results in MYO7A reconstitution and therapeutic efficacy in a mouse model of USHIB. This has received the Orphan Drug Designation from the European Medicines Agency. It would be the first time the dual AAV vector approach is tested in humans. Towards this ambitious objective, UshTher has assembled a very competitive consortium with leaders in the fields of retinal gene therapy from bench to bedside including SMEs with expertise in the development of gene therapy products. The planned activities span from manufacturing of clinical –grade dual AAV vectors to non clinical safety, biodistribution and expression studies performed under good laboratory practices up to performing a multicenter, multinational clinical trial which envisages subretinal administrations of dual AAV vectors in twelve USHIB patients. UshTher success will set the basis for a cure for USHIB retinitis pigmentosa, and for testing dual AAV vectors in other rare and more common conditions which require delivery of large genes, thus overcoming one of the major limitations of in vivo gene therapy.

Deliverables

List of deliverables.
Report Planned communication activity	Documents, reports	2020-04-07 22:46:13
Connection with patient organization	Documents, reports	2020-04-07 22:46:13
Website and intranet portal	Websites, patent fillings, videos etc.	2020-04-07 22:46:14
Official logo of UshTher	Documents, reports	2020-04-07 22:46:14
Flyer publication	Documents, reports	2020-04-07 22:46:14
Contact database of relevant representatives of stakeholder groups	Documents, reports	2020-04-07 22:46:13
Detailed dissemination plan	Documents, reports	2020-04-07 22:46:13
Social Network strategy plan	Websites, patent fillings, videos etc.	2020-04-07 22:46:14
Periodic report on project dissemination and exploitation	Documents, reports	2020-04-07 22:46:13

Take a look to the deliverables list in detail: detailed list of UshTher deliverables.

Are you the coordinator (or a participant) of this project? Plaese send me more information about the "USHTHER" project.

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Send me an email (fabio@fabiodisconzi.com) and I put them in your project's page as son as possible.

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The information about "USHTHER" are provided by the European Opendata Portal: CORDIS opendata.