EU/3/16/1753: Orphan designation for the treatment of leukocyte adhesion deficiency type I

Haematopoietic stem cells modified with a lentiviral vector containing the CD18 gene

Table of contents

Overview

On 14 October 2016, orphan designation (EU/3/16/1753) was granted by the European Commission to Centro de Investigación Biomédica en Red, Spain, for haematopoietic stem cells modified with a lentiviral vector containing the CD18 gene for the treatment of leukocyte adhesion deficiency type I.

In October 2017 Centro de Investigación Biomédica en Red (CIBER) changed name to Consorcio Centro de Investigación Biomédica en Red, M.P.

The sponsor's name was updated in November 2022.

Key facts

Active substance
Haematopoietic stem cells modified with a lentiviral vector containing the CD18 gene
Intended use
Treatment of leukocyte adhesion deficiency type I
Orphan designation status
Positive
EU designation number
EU/3/16/1753
Date of designation
20/09/2016
Sponsor

Consorcio Centro de Investigación Biomédica en Red
Instituto de Salud Carlos III
Pabellón 11, Planta 0
C/ Monforte de Lemos 3-5
28029 Madrid
Spain
Tel. +34 911 718 114
E-mail: gestores@ciberer.es

Update history

Date Update
October 2017 Centro de Investigación Biomédica en Red (CIBER) changed name to Consorcio Centro de Investigación Biomédica en Red, M.P.
November 2022 The sponsor's name was updated.

Documents related to this orphan designation evaluation

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.

EU register of orphan medicines

The list of medicines that have received an orphan designation in the EU is available on the European Commission's website:

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