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 GTC2665
Trial ID NCT04850118
Disease Retinitis Pigmentosa
Altered gene RPGR
Therapeutic/Target gene Therapeutic gene
TherapyGene transfer
Treatment AGTC-501
PhasePhase2|Phase3
Recruitment statusNot Recruiting
TitleA Phase 2/3, Randomized, Controlled, Masked, Multi-center Study to Evaluate the Efficacy, Safety and Tolerability of Two Doses of AGTC-501, a Recombinant Adeno-associated Virus Vector Expressing RPGR (rAAV2tYF-GRK1-RPGR), Compared to an Untreated Control Group in Male Subjects With X-linked Retinitis Pigmentosa Confirmed by a Pathogenic Variant in the RPGR Gene
Year2021
CountryUnited Kingdom
Company sponsorApplied Genetic Technologies Corp
Other ID(s)AGTC-RPGR-002
Vector information
Vectoradeno-associated virus
ConstructrAAV2tYF-GRK1-hRPGRco
Vector typerecombinant adeno-associated virus serotype 2 (rAAV2) vector
Transgene/Inserted genea codon-optimized human RPGR (hRPGRco) cDNA
Regulatory elementa photoreceptor-specific promoter (G protein-coupled receptor kinase 1 [GRK1])
Viral genome modificationan AAV2 capsid variant with three surface tyrosine residues changed to phenylalanine (AAV2tYF).
Vector production methodThe vector AGTC-501 was produced using a recombinant herpes simplex virus (rHSV)-assisted vector expansion (HAVE) system in suspension-cultured baby hamster kidney (sBHK) cells.

Clinical Result

Cohort 1
Administration route subretinal injection
Age Child, Adult
References PMID: 31910043

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