Table 3.
Current status of nonviral vector-based DNA therapies in clinical trials.
| Delivery system | Gene (DNA) | Delivery route | Sponsor | Indications | Development phase | ClinicalTrials.gov Identifier |
|---|---|---|---|---|---|---|
| Cationic liposome (DMRIE/DOPE) | Cystic fibrosis gene (pGT-1) | Nasal instillation | University of Alabama at Birmingham | Cystic fibrosis | I | NCT00004471 |
| Cationic liposome (DMRIE/DOPE) | Cystic fibrosis transmembrane conductance regulator (CFTR) gene | Intranasal | University of Alabama at Birmingham | Cystic fibrosis | I | NCT00004806 |
| DC-cholesterol liposome | EGFR antisense DNA | Intratumoral | University of Pittsburgh | Head and neck cancer | I | NCT00009841 |
| DOTAP:Cholesterol | FUS 1 | Intravenous | M.D. Anderson Cancer Center | Non-Small-Cell Lung Cancer | I | NCT00059605 |
| GL67A (a mixture of GL67–DOPE–DMPE-PEG5000) | CFTR gene | Nebulization | Imperial College London | Cystic fibrosis | II | NCT01621867 |
| DOTAP: DOPE cationic liposome | Normal human wild type (wt) p53 DNA | Intravenous | SynerGene Therapeutics, Inc. | Solid tumors | I | NCT00470613 |
| DOTAP: DOPE cationic liposome | Normal human wild type p53 DNA | Intravenous | SynerGene Therapeutics, Inc. | Metastatic pancreatic cancer | II | NCT02340117 |
| DOTAP: DOPE cationic liposome | Normal human wild type (wt) p53 DNA | Intravenous | SynerGene Therapeutics, Inc. | Refractory or recurrent solid tumors | I | NCT02354547 |
| PEI | DTA-H19 | Intravesical | Hebrew University of Jerusalem | Bladder neoplasms | I/II | NCT00393809 |
| PEI | DTA-H19 | Intravesical | BioCancell Ltd. | Superficial bladder cancer | II | NCT00595088 |
| PEI | BC-819 BCG vaccine |
Intravesical | BioCancell Ltd. | Transitional cell carcinoma of bladder | I | NCT01878188 |
| PEI | HIV DNA Vaccine | Intradermal | Swedish Institute for Infectious Disease Control | HIV-1 | I | NCT01140139 |
| PEG-PEI-Cholesterol | EGEN-001-301 | Intraperitoneal | EGEN, Inc. | Colorectal cancer | I/II | NCT01300858 |
| PEG-PEI-Cholesterol | EGEN-001 (IL-12 DNA Plasmid Vector GEN-1) |
Intraperitoneal | Gynecologic Oncology Group | Fallopian tube, primary peritoneal, and recurrent ovarian carcinoma | II | NCT01118052 |
| PEG-PEI-Cholesterol | EGEN-001 (IL-12 DNA Plasmid Vector GEN-1) |
Intraperitoneal | Gynecologic Oncology Group | Ovarian, recurrent ovarian, undifferentiated ovarian, recurrent fallopian tube, and recurrent primary peritoneal cancer | I | NCT01489371 |
| PEG-PEI-Cholesterol | GEN-1 (IL-12 Plasmid) | Intraperitoneal | Celsion | Epithelial ovarian, fallopian tube, and primary peritoneal cancer | I | NCT02480374 |
| Poloxamer CRL1005/benzalkonium chloride | ASP0113 | Intramuscular | Astellas Pharma Global Development, Inc. | Cytomegalovirus (CMV) therapeutic vaccine | III | NCT01877655 |
| Poloxamer CRL1005/benzalkonium chloride | VCL-CB01 | Intramuscular | Astellas Pharma Inc. | CMV therapeutic vaccine | II | NCT00285259 |