Trial Profile

An Open Label Dose Escalation Phase 1 Clinical Trial of Retinal Gene Therapy for Choroideraemia Using an Adeno-associated Viral Vector (AAV2) Encoding Rab-escort Protein 1 (REP1)

Status: Completed

Phase of Trial: Phase I/II

Latest Information Update: 22 Nov 2018

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At a glance

08 Nov 2018 According to a Nightstar Therapeutics media release, results were published in Nature Medicine in 2018.
03 Apr 2018 According to a Nightstar Therapeutics media release, two years of follow-up data from this trial will be presented at the The Association for Research in Vision and Ophthalmology (ARVO) meeting (2018).
03 Apr 2018 Results of two years of follow-up of patients in this trial presented in the Nightstar Therapeutics media release.

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