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On 22 February 2018, orphan designation (EU/3/18/1975) was granted by the European Commission to Nightstar Therapeutics plc, the United Kingdom, for adenovirus-associated viral vector serotype 8 containing the human RPGR gene for the treatment of retinitis pigmentosa.

The sponsor’s address was updated in September 2018.

Key facts

Active substance
Adenovirus-associated viral vector serotype 8 containing the human RPGR gene
Disease / condition
Treatment of retinitis pigmentosa
Date of decision
Orphan decision number

Sponsor's contact details

Nightstar Therapeutics plc
9-10 Midford Place
London W1T 5BJ
United Kingdom
Tel. 44 (0)207 7062 2777

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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