EU/03/14/1278

Table of contents

About

On 4 June 2014, orphan designation (EU/03/14/1278) was granted by the European Commission to Alan Boyd Consultants Ltd, United Kingdom, for adeno-associated viral vector serotype 2 containing the human CHM gene encoding human Rab escort protein 1 for the treatment of choroideremia.

The sponsorship was transferred to Spark Therapeutics Ireland Ltd, Ireland, in October 2017.

The sponsor’s address was updated in April 2019.

Key facts

Active substance
Adeno-associated viral vector serotype 2 containing the human CHM gene encoding human Rab escort protein 1
Disease / condition
Treatment of choroideraemia
Date of first decision
04/06/2014
Outcome
Positive
EU designation number
EU/03/14/1278

Sponsor's contact details

Spark Therapeutics Ireland Limited
6th Floor
2 Grand Canal Quay
Dublin 2
Co. Dublin
Ireland
Tel: +353 87 2865356
E-mail: info@sparktx.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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