Table 2.
Types of gene therapy in current clinical trials.
| Description (Trials) | Number of Trials | |
|---|---|---|
| Open-label trials | 11 | |
| Sickle Cell Disease (SCD) | 9 | |
| Sickle Cell Anemia | 3 | |
| Intervention | NCT Numbers | |
| Gene addition, modified β:β-AT87Q | 3 | 02,140,55402,151,52604,293,185 |
| Gene addition, modified β:β-AS3 | 1 | 03,964,792 |
| Gene addition, modified β:β-AS3-FB | 1 | 02,247,843 |
| Gene addition, modified γ:ARU-1801 | 1 | 02,186,418 |
| Gene addition, modified γ: with siRNA for selection:γ-G16D-RNA734 | 1 | 04,091,737 |
| Gene silencing, BCH-BB694 short hairpin RNA targeting:BCL11a | 1 | 03,282,656 |
| Zn finger editing of BCL11a:BIVV003 | 1 | 03,653,247 |
| CRISPR editing of BCL11a:CTX001 | 1 | 03,745,287 |
| Gene editing of BCL11a:OTQ923 and/or HIX763 | 1 | 04,443,907 |
| Description (Studies) | Number of Trials | |
| Long-term follow-up studies | 3 | |
| Disease | ||
| Sickle Cell Disease (SCD) | 3 | |
| Sickle Cell Anemia | 0 | |
| Intervention | ||
| γ-AT87Q | 2 | 02,633,94304,628,585 |
| CTX001 | 1 | 04,208,529 |