Basic Information

Gene symbol CD59 Synonyms 16.3A5, 1F5, EJ16, EJ30, EL32, G344, HRF-20, HRF20, MAC-IP, MACIF, MEM43, MIC11, MIN1, MIN2, MIN3, MIRL, MSK21, p18-20 Type of gene protein-coding
Description CD59 molecule (CD59 blood group)

GTO ID GTC1922
Trial ID NCT03585556
Disease Age-Related Macular Degeneration
Altered gene CD59
Therapeutic/Target gene Therapeutic gene
TherapyGene transfer
Treatment HMR59
Co-treatment Oral prednisolone
PhasePhase1
Recruitment statusCompleted
TitleA Phase 1 Proof of Concept Study Evaluating Intravitreal AAVCAGsCD59 for the Treatment of Wet Age-Related Macular Degeneration (AMD)
Year2018
CountryUnited States
Company sponsorJanssen Research & Development, LLC
Other ID(s)81201887MDG1002|HMR-1002|81201887MDG1002
Vector information
Vectoradeno-associated virus
ConstructAAV.CAG-sCD59
Vector typeadeno-associated virus (AAV) vector
Transgene/Inserted genesoluble CD59
Regulatory elementthe CAG promoter

Clinical Result

Cohort1: dose level 1
Administration route intravitreal injection
Dosage 3.56E11vg
Pts 2
Age Adult, Older_Adult
References PMID: 35017696
Cohort2: dose level 2
Administration route intravitreal injection
Dosage 1.071E12 vg
Pts 3
Age Adult, Older_Adult
References PMID: 35017696

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