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 GTC2333
Trial ID NCT04312672
Disease Retinitis Pigmentosa
Altered gene RPGR
Therapeutic/Target gene Therapeutic gene
TherapyGene transfer
Treatment botaretigene sparoparvovec
Recruitment statusActive, Not Recruiting
TitleLong Term Follow-Up Study of Participants Following an 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)
Year2019
CountryUnited Kingdom|United States
Company sponsorMeiraGTx UK II Ltd
Other ID(s)MGT010
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 None
Age Child, Adult, Older_Adult
References PMID: 38871269

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