Skip to main content

Table 1 Anti-HIV-1 gene therapy landscape

From: The therapeutic landscape of HIV-1 via genome editing

Therapeutic approach Gene therapy Interventions Targets Identifier Stage/status Company/Institute
Drug shRNA peptide Dual anti-HIV gene Transfer construct, LVsh5/C46 (Cal) CCR5 shRNA; C46 peptide Busulfan Host co-receptor; viral Env; transplant conditioning NCT01734850 Phase I/II recruiting Calimmune/CalTech/UCLA
shRNA Long term follow up of delayed adverse events in Cal-1 recipients Blood test for general health, complete blood count and Cal-1 specific analyses NCT02390297 Recruiting by invitation Calimmune/CalTech/UCLA
Endoribonuclease Redirected MazF-CD4 autologous T-cells CCR5 MazF Host co-receptor NCT01787994 Phase I ongoing University of Pennsylvania
Ribozyme Autologous CD34+ HSCs transduced with anti-HIV-1 ribozyme (OZ1) Tat-vpr anti-HIV ribozyme tat-vpr mRNA NCT00074997 Phase II completed Janssen-Cilag Pty Ltd, UCLA
Ribozyme Long term follow up of OZ1 Gene therapy Blood test for quantitative marking of the gene transfer product in PBMCs overtime NCT01177059 Phase II Recruiting by invitation Janssen-Cilag Pty Ltd, UCLA
Antisense Tolerability and therapeutic effects of repeated doses of autologous T cells with VRX496 VRX496 antisense RNA Env mRNA NCT00295477 Phase I/II Ongoing/ not recruiting University of Pennslyvernia/NIAID
Antisense Safety and efficacy of T-cell genetic immunotherapy VRX496 antisense RNA Env mRNA NCT00131560 Phase II Ongoing VIRxSYS Corporation
Ribozyme L-TR/Tat-neo in HIV+ Patients with Non-Hodgkin’s Lymphoma Tat ribozyme Tat-rev mRNA NCT00002221 Phase II completed Ribozyome/City of Hope
Inhibitory peptides M87o autologous HSCs for HIV+ patients with malignant disease C46 peptide Viral Env NCT00858793 Phase I/II suspended University Medical Center Hamburg-Eppendorf
Ribozyme, inhibitory peptide C46/CCR5/P140K modified autologous HSCs in patients with lymphoma C46 peptide, CCR5 ribozyme, MGMTP140 K mutant Viral Env, CCR5 mRNA, Alkylating agent resistance NCT02343666 Phase I, recruiting Fred Hutchinson Cancer Research Center/NCI/NHLBI
shRNA,peptide Autologous transplantation of HSCs with LVsh5/C46 (Cal-1) for treatment of HIV-related lymphoma CCR5 shRNA, C46 peptide Host co-receptor, viral Env NCT02378922 Phase I recruiting Fred Hutchinson Cancer Research Center/NCI
shRNA, ribozyme,RNA decoy, drugs rHIV7-shI-TAR-CCR5RZ-transduced HSC in patient with AIDS-related Non-Hodgkin Lymphoma tat/rev shRNA, TAR decoy, CCR5 ribozyme, Prednisone, Rituximab, Etoposide, Doxorubicin Hydrochloride, Vincristine Sulfate, Cyclophosphamide Viral mRNA, Viral tat proteinm, CCR5 mRNA, Transplant conditioning NCT02337985 Pilot recruiting City of Hope Medical Center/NCI
shRNA, ribozyme,RNA decoy, drugs rHIV7-shI-TAR-CCR5RZ-transduced HSC in patient with AIDS-related Non-Hodgkin Lymphoma tat/rev shRNA, TAR decoy, CCR5 ribozyme, Busulfan Viral mRNA, Viral tat proteinm, CCR5 mRNA NCT01961063 Pilot recruiting City of Hope Medical Center/NCI
shRNA, ribozyme,RNA decoy, drugs rHIV7-shI-TAR-CCR5RZ-transduced HSC in patient undergoing stem cell transplant for AIDS-related lymphoma tat/rev shRNA, TAR decoy, CCR5 ribozyme, carmustine, cyclophosphamide, etoposide viral mRNA, viral tat protein, CCR5 mRNA, transplant conditioning NCT00569985 Pilot ongoing, not recruiting City of Hope Medical Center/NCI
shRNA, RNA decoy shRNA/TRIM5alpha/TAR decoy-transduced Autologous HSC in patient with HIV-Related Lymphoma CCR5 shRNA, RNF88,TAR decoy Host co-receptor, Gag p24, Viral tat protein NCT02797470 Phase I/II City of Hope Medical Center/NCI
RNA decoy RNA decoy (ex vivo retroviral modified CD34+ HPC) Rev reponse element decoy Rev Protein NCT00001535 Phase 0-pilot Children’s Hospital
Redirected high affinity gag-specific autologous T cells WT-gag-TCR or alpha/6-gag-TCR CD8 TCR NCT00991224 Phase I completed University of Pennslyvernia/Adaptimmune
ZFN T-cells modified at CCR5 gene by ZFN SB-728mR CCR5 ZFN CCR5 DNA NCT02388594 Phase 1 recruiting University of Pennsylvania/NIAID
Repeated doses of SB-728mR-T after cyclophosphamide conditioning in HIV+ on HAART CCR5 ZFN (SB-728mR-T) CCR5 DNA NCT02225665 PHASE I/II ongoing Sangamo biosciences
Autologous T cells modified at CCR5 gene by ZFN SB-728 CCR5 ZFN CCR5 DNA NCT00842634 Phase 1 completed Sangamo biosciences/ Uni. Of Pennsylvania
Autologous T-cells genetically modified at the CCR5 gene by zinc finger nucleases (SB-728-T) in HIV-infected patients CCR5 ZFN CCR5 DNA NCT01044654 Phase I completed Sangamo Therapeutics
Safety of ZFN CCR5-modified HPS/progenitor cells in HIV+ SB-728mR-HSPC infusion after busulfan conditioning CCR5 DNA NCT02500849 Phase 1 City of Hope Medical Center|Sangamo Therapeutics
Autologous T-cells genetically modified at the CCR5 Gene by zinc finger nucleases in HIV-infected subjects CCR5 ZFN (SB-728-T) CCR5 DNA NCT01252641 Phase I/II completed Sangamo Therapeutics