EU/3/20/2254

Table of contents

About

On 28 February 2020, orphan designation EU/3/20/2254 was granted by the European Commission to Fondazione Telethon, Italy, for combination of three adeno-associated viral vectors of serotype 8 containing the 5'-, the body- and the 3'- coding sequences of human CEP290 fused to inteins for the treatment of inherited retinal dystrophies.

Key facts

Active substance
Combination of three adeno-associated viral vectors of serotype 8 containing the 5'-, the body- and the 3'- coding sequences of human CEP290 fused to inteins
Disease / condition
Treatment of inherited retinal dystrophies
Date of first decision
28/02/2020
Outcome
Positive
EU designation number
EU/3/20/2254

Sponsor's contact details

Fondazione Telethon
Via Varese 16 B
00185 Rome
Italy
Tel. +39 08 119 230 622
E-mail: fontanel@tigem.it

Patients' organisations

For contact details of patients’ organisations whose activities are targeted at rare diseases, see:

  • European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.

  • Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.

How useful was this page?

Add your rating