B/BE/21/BVW8 - Information sheet for the public (prepared by the notifier)Public information according to the provisions of Directive 2001/18/EC
Long title
Phase 3 Randomized, Controlled Study of AAV5-hRKp.RPGR for the Treatment of X-linked Retinitis Pigmentosa Associated with Variants in the RPGR gene
Date receipt dossier
EU record number
B/BE/21/BVW8
EudraCT number
2020-002873-88
Company / Sponsor
Meira GTx UK II Limited
Phase
III
Treated organism
Humans
Indication category
Genetic disorders
Disease
X-linked Retinitis Pigmentosa associated with Variants in the RPGR gene
Therapeutic approach
Gene therapy
Genetic modification
Non-replicating recombinant vector derived from adeno-associated virus AAV 5 carrying the human RPGR gene
Method of transfer of nucleic acid of interest
Non-replicating recombinant vector derived from adeno-associated virus AAV 5
Administered biological material
Genetically modified virus
Route of administration
Subretinal
Locations in Belgium
UZ Gent
Nr of subjects
Up to 60 patients overall and 10 patients in Belgium
Foreseen duration
Start April 2022 with long-term safety follow-up of patients
Type of procedure
Contained use and Deliberate release
Current status
Authorized