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 GTC0386
Trial ID NCT03252847
Disease Retinitis Pigmentosa
Altered gene RPGR
Therapeutic/Target gene Therapeutic gene
TherapyGene transfer
Treatment botaretigene sparoparvovec
PhasePhase1|Phase2
Recruitment statusCompleted
TitleAn Open Label, Multi-centre, Phase I/II Dose Escalation Trial of a Recombinant Adeno-associated Virus Vector (AAV2-.RPGR) for Gene Therapy of Adults and Children With X-linked Retinitis Pigmentosa Owing to Defects in Retinitis Pigmentosa GTPase Regulator (RPGR)
Year2017
CountryUnited Kingdom|United States
Company sponsorMeiraGTx UK II Ltd
Other ID(s)MGT009|2016-003967-21
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

Cohort 1
Administration route subretinal injection
Dosage 0.3~0.8 ml
Pts 45
Age Child, Adult, Older_Adult
Outcome At 12 mo compared with baseline, V30 in total in the treated eye improved in Pt 005 (7.56 dB-sr vs 9.50 dB-sr, +25.7% improvement) and in Pt 007 (9.54 dB-sr vs 10.18 dB-sr, +6.7% improvement).
Adverse reactions 2/45(Eye disorders)
References PMID: 38871269

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