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 GTC0157
Trial ID NCT01734850
Disease HIV Infection
Altered gene CCR5
Therapeutic/Target gene Target gene
TherapyshRNA
Treatment Cal-1
PhasePhase1|Phase2
Recruitment statusCompleted
TitleAn Adaptive Phase I/II Study of the Safety of CD4+ T Lymphocytes and CD34+ Hematopoietic Stem/Progenitor Cells Transduced With LVsh5/C46, a Dual Anti-HIV Gene Transfer Construct, With and Without Conditioning With Busulfan in HIV-1 Infected Adults Previously Exposed to ART
Year2012
CountryUnited States
Company sponsorCalimmune, Inc.
Other ID(s)CAL-USA-11
Vector information
Vectorlentivirus
Vector typeself-inactivating lentiviral vector
Transgene/Inserted geneshort hairpin RNA against the HIV-1 co-receptor CCR5 (sh5); HIV-1 fusion inhibitor, C46

Clinical Result

Cohort1: No Busulfan
Administration route None
Donor type autologous
Pts 4
Age Adult, Older_Adult
Lymph depletion No
Outcome Up to 48 weeks, the mean cell dose for CD4+ cells (Ttn) was 16.940E8 cells (range: 14.61 to 20.62) in 4 participants.
Adverse reactions No serious clinical adverse events
Cohort2: 4mg/kg busulfan
Administration route None
Donor type autologous
Pts 4
Age Adult, Older_Adult
Lymph depletion Yes
Outcome Up to 48 weeks, the mean cell dose for CD4+ cells (Ttn) was 81.855E8 cells (range: 38.94 to 138.68) in 4 participants.
Adverse reactions No serious clinical adverse events
Cohort3: 8mg/kg busulfan
Administration route None
Donor type autologous
Pts 5
Age Adult, Older_Adult
Lymph depletion Yes
Outcome Up to 48 weeks, the mean cell dose for CD4+ cells (Ttn) was 49.553E8 cells (range: 20.70 to 79.29) in 4 participants.
Adverse reactions 1/5(Gastrointestinal disorders)

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