An Open-Label Dose-Escalation Study to Assess the Safety and Tolerability of a Single Intravitreal Injection of SPVN20 Gene Therapy in Subjects with No Light Perception Due to End-Stage Rod-Cone Dystrophy, and Who Retain Dormant Foveal Cone Photoreceptors

Study title: 
An Open-Label Dose-Escalation Study to Assess the Safety and Tolerability of a Single Intravitreal Injection of SPVN20 Gene Therapy in Subjects with No Light Perception Due to End-Stage Rod-Cone Dystrophy, and Who Retain Dormant Foveal Cone Photoreceptors
Long title: 
An Open-Label Dose-Escalation Study to Assess the Safety and Tolerability of a Single Intravitreal Injection of SPVN20 Gene Therapy in Subjects with No Light Perception Due to End-Stage Rod-Cone Dystrophy, and Who Retain Dormant Foveal Cone Photoreceptors (NYRVANA)
Date receipt dossier: 
14 May 2025
EU record number: 
B/BE/25/BVW4
EudraCT number: 
2025-520665-47-00
Company / Sponsor: 
SparingVision
Phase: 
I
Treated organism: 
Humans
Indication category: 
Eye degenerative disease
Disease: 
End-stage rod-cone dystrophy
Therapeutic approach: 
Immunotherapy
Genetic modification: 
Non-replicating recombinant vector derived from adeno-associated virus AAV2 carrying the G protein-gated inwardly rectifying potassium (GIRK) channel 1, mutated for an F137S amino-acid substitution (GIRK1(F137S)) gene
Method of transfer of nucleic acid of interest: 
Non-replicating recombinant vector derived from adeno-associated virus AAV2
Administered biological material: 
Genetically modified virus
Route of administration: 
Intravitreal
Locations in Belgium: 
UZ Gent
Nr of subjects: 
Up to 9 patients overall and 4 patients in Belgium
Foreseen duration: 
From July 2025 to July 2031 in Belgium
Type of procedure: 
Contained use and Deliberate release
Current status: 
Under evaluation

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