Basic Information

Gene symbol RPGR Synonyms COD1, CORDX1, CRD, PCDX, RP15, RP3, XLRP3, orf15 Type of gene protein-coding
Description retinitis pigmentosa GTPase regulator

GTO ID GTC2619
Trial ID NCT04794101
Disease Retinitis Pigmentosa
Altered gene RPGR
Therapeutic/Target gene Therapeutic gene
TherapyGene transfer
Treatment botaretigene sparoparvovec
PhasePhase3
Recruitment statusRecruiting
TitleFollow-up Phase 3 Randomized, Controlled Study of AAV5-RPGR for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene
Year2020
CountryBelgium|Canada|Denmark|France|Israel|Italy|Netherlands|Spain|Switzerland|United Kingdom|United States
Company sponsorMeiraGTx UK II Ltd
Other ID(s)MGT-RPGR-022|2020-002255-37
Vector information
Vectoradeno-associated virus
ConstructAAV5-hRKp.RPGR
Vector typeadeno-associated virus serotype 5 (AAV5) vector
Transgene/Inserted genea linear single strand of human RPGR-ORF15 deoxyribonucleic acid (DNA) with a 378-base pair (in frame) deletion in the purine-rich tract in the ORF15 region
Regulatory elementa fragment of the human RK promoter

Clinical Result

Cohort1: dose level 1
Administration route subretinal injection
Dosage 4E11vg/kg
Age Child, Adult, Older_Adult
References PMID: 38871269
Cohort2: dose level 2
Administration route subretinal injection
Dosage 2E11vg/kg
Age Child, Adult, Older_Adult
References PMID: 38871269

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