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 GTC0368
Trial ID NCT03116113
Disease Retinitis Pigmentosa
Altered gene RPGR
Therapeutic/Target gene Therapeutic gene
TherapyGene transfer
Treatment BIIB112
PhasePhase1|Phase2
Recruitment statusCompleted
TitleA Dose Escalation (Phase 1), and Dose Expansion (Phase 2/3) Clinical Trial of Retinal Gene Therapy for X-linked Retinitis Pigmentosa Using an Adeno-Associated Viral Vector (AAV8) Encoding Retinitis Pigmentosa GTPase Regulator (RPGR)
Year2017
CountryUnited Kingdom|United States
Company sponsorNightstaRx Ltd, a Biogen Company
Other ID(s)274RP101|NSR-RPGR-01|2016-003852-60
Vector information
Vectoradeno-associated virus
ConstructAAV8.coRPGR
Vector typeadeno-associated virus serotype 8 (AAV8) vector
Transgene/Inserted genea codon-optimized human RPGR (hRPGRco) cDNA

Clinical Result

Cohort1: dose level 1
Administration route subretinal injection
Dosage 5E10 gp/ml
Pts 3
Age Child, Adult, Older_Adult
Adverse reactions 3/3(Sub-conjunctival haemorrhage)
References PMID: 32094925
Cohort2: dose level 2
Administration route subretinal injection
Dosage 1E11 gp/ml
Pts 3
Age Child, Adult, Older_Adult
Adverse reactions 1/3(Anterior uveitis)
References PMID: 32094925
Cohort3: dose level 3
Administration route subretinal injection
Dosage 5E11 gp/ml
Pts 3
Age Child, Adult, Older_Adult
Adverse reactions 3/3(Persistent subretinal fluid; Anterior uveitis; Suture related inflammation; Corneal abrasion)
References PMID: 32094925
Cohort4: dose level 4
Administration route subretinal injection
Dosage 1E12 gp/ml
Pts 3
Age Child, Adult, Older_Adult
Adverse reactions 3/3(Anterior uveitis; subretinal inflammation)
References PMID: 32094925
Cohort5: dose level 5
Administration route subretinal injection
Dosage 2.5E12 gp/ml
Pts 3
Age Child, Adult, Older_Adult
Adverse reactions 2/3(subretinal inflammation)
References PMID: 32094925
Cohort6: dose level 6
Administration route subretinal injection
Dosage 5E12 gp/ml
Pts 3
Age Child, Adult, Older_Adult
Adverse reactions 3/3(subretinal inflammation; Ocular pain; Suture granuloma; Anterior uveitis)
References PMID: 32094925

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