Basic Information

Gene symbol CCR5 Synonyms CC-CKR-5, CCCKR5, CCR-5, CD195, CKR-5, CKR5, CMKBR5, IDDM22 Type of gene protein-coding
Gene perturbation-related omics dataset PertOrg
Description C-C motif chemokine receptor 5

GTO ID GTC0075
Trial ID NCT00842634
Disease HIV Infection
Altered gene CCR5
Therapeutic/Target gene Target gene
TherapyGene editing
Treatment ZFN modified T cells
PhasePhase1
Recruitment statusCompleted
TitleA Phase I Study of Autologous T-Cells Genetically Modified at the CCR5 Gene by Zinc Finger Nucleases SB-728 in HIV-Infected Patients
Year2009
CountryUnited States
Company sponsorUniversity of Pennsylvania
Other ID(s)806383
Vector information
Vectoradenovirus
Vector typeadenoviral vector
Transgene/Inserted geneZinc Finger Nuclease(ZFNs)

Clinical Result

Cohort 1
Administration route infusion
Dosage 5E10~1E11 cells
Donor type autologous
Pts 12
Age Adult, Older_Adult
Outcome The median CD4 T-cell count significantly increased from 448 to 1517 cells per cubic millimeter at week 1 (P<0.001). The median concentration of CCR5-modified CD4 T cells was 250 cells per cubic millimeter, constituting 8.8% of circulating peripheral-blood mononuclear cells and 13.9% of circulating CD4 T cells, with an estimated mean half-life of 48 weeks. During treatment interruption, the decline in CCR5-modified cells was significantly less than unmodified cells (-1.81 vs. -7.25 cells per day, P=0.02). HIV RNA became undetectable in one of four evaluable patients, and most patients showed a decrease in blood levels of HIV DNA.
Adverse reactions 1/12(Fever; chills; joint pain; back pain)
References PMID: 24597865

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