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.

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

Sangamo Therapeutics UK LTD
C/O Jag Shaw Baker
5th Floor Berners House
47-48 Berners Street
London W1T 3NF
United Kingdom
Tel. +44 (0)7768 237 340
E-mail: communications@sangamo.com

Patients' organisations

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

  • Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe;
  • European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.

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