EU/3/17/1955

Table of contents

About

On 17 January 2018, orphan designation (EU/3/17/1955) was granted by the European Commission to Quintiles Ireland Limited, Ireland, for adeno-associated viral vector serotype 2/6 encoding zinc-finger nucleases and the human alpha L-iduronidase gene (also known as SB-318) for the treatment of mucopolysaccharidosis type I.

In May 2018 the sponsor, Quintiles Ireland Limited changed name to IQVIA RDS Ireland Limited.

The sponsor's address was updated in June 2018.

The sponsorship was transferred to TxCell S.A., France, in February 2019.

Key facts

Active substance
Adeno-associated viral vector serotype 2/6 encoding zinc-finger nucleases and the human alpha L-iduronidase gene
Disease / condition
Treatment of mucopolysaccharidosis type I
Date of decision
17/01/2018
Outcome
Positive
Orphan decision number
EU/3/17/1955

Sponsor's contact details

TxCell S.A.
Les Cardoulines Ht1
Allee De La Nertiere
06560 Valbonne
France
Tel. +33 49 72 18 30 1
E-mail: communications@sangamo.com

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;

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