EU/3/17/1969

Table of contents

About

On 17 January 2018, orphan designation (EU/3/17/1969) was granted by the European Commission to the University of Cambridge, United Kingdom, for recombinant adeno-associated viral vector serotype 2/1 encoding human beta-hexosaminidase alpha and beta subunits (also known as CAM-GM201) for the treatment of GM2 gangliosidosis.

Key facts

Active substance
Recombinant adeno-associated viral vector serotype 2/1 encoding human beta-hexosaminidase alpha and beta subunits
Disease / condition
Treatment of GM2 gangliosidosis
Date of decision
17/01/2018
Outcome
Positive
Orphan decision number
EU/3/17/1969

Sponsor's contact details

University of Cambridge
Department of Medicine
Addenbroke's Hospital
Hills Road
Cambridge CB2 0QQ
United Kingdom
Tel. +44 (0)1223 336844
E-mail: mjl98@cam.ac.uk

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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