| Drug product | Sponsor | Status and clinical trial identifier | Participant/n | Clinical trial result |
|---|
| Lentivirus-mediated β-globin gene addition | | | Zynteglo | Bluebird Bio | Phase Ⅰ/Ⅱ, NCT01745120 | 22 | Transfusion independence (TI) occurred in 12 patients with non-β0/β0 genotype and 3 patients with β0/β0 genotype or IVS1-110 homozygous mutation[12] | | | Phase Ⅲ, NCT02906202 | 24 | TI occurred in 20 patients with non-β0/β0 genotype while the average Hb level was 117 g/L, and the median level of HbAT87Q was 87 g/L at the 12th month after infusion[13] | | | Phase Ⅲ, NCT03207009 | 19 | One patient with β0/β0 genotype had TI for ≥ 12 months, while the Hb level of 3 patients with TI for ≥ 6 months was up to 105‒136 g/L (HbAT87Q accounting for 95‒126 g/L)[3] | | OTL-300 | Orchard Therapeutics | Phase Ⅰ/Ⅱ, NCT02453477 | 10 | TI occurred in 3 pediatric patients, and red-cell transfusions were reduced in 4 patients[11] | | HGI-001 | Hemu Gene Co., Ltd. | IIT, NCT05745532 | 10 | The average Hb level of 5 patients was > 90 g/L during TI ≥ 12 months, with the median Hb level of 105 g/L at the most recent follow-up[14] | | BD211 | BDgene Co., Ltd. | IIT, NCT05015920 | 10 | Two patients with β0/β0 genotype had TI for 25.5 months in average, with red blood cell lifespan being extended to more than 42 d[15] | | KL003 | Kanglin Biotechnology | IIT, ChiCTR2200055565 | 11 | The average neutrophil and platelet engraftment times were both 14 d. TI occurred in all patients, with the longest duration lasting 18 months[16] | | GMCN508B | Genmedicn Biopharma | IIT, NCT05762510 | 5 | Two patients with TDT had TI, including a 10-year-old pediatric patient with β0/β+ genotype | | Gene editing to re-activate the expression of HbF | | | ST-400 | Sangamo Therapeutics | Phase Ⅰ/Ⅱ, NCT03432364 | 6 | The low transduction efficiency of HSCs resulted in no long-term therapeutic effect[17] | | CTX001 | Vertex Pharmaceuticals | Phase Ⅲ, NCT03655678 | 59 | The median duration of TI among 48 patients was 22.5 months, with a total Hb level of 131 g/L and HbF level of 119 g/L in average[4] | | EDIT-301 | Editas Medicine | Phase Ⅰ/Ⅱ, NCT05444894 | 9 | TI occurred in 7 patients, including 6 with follow-up ≥ 6 months, whose total Hb and HbF levels were 121 g/L and 109 g/L, respectively[18] | | ET-01 | EdiGene Inc. | IIT, NCT04390971 | 3 | One patient (β0/β+) had TI for>15 months, with a total Hb level of 110 g/L at the 18th month[19] | | BRL-101 | BRL Medicine | Phase Ⅰ, NCT05577312 | 10 | TI > 22 months occurred in all patients, including 5 with β0/β0 genotype. The highest HbF level reached 140 g/L, with HbF-cells accounting for 98%‒99%[20] | | RM-001 | Reforgene Medicine | Phase Ⅰ, ChiCTR2300069244 | 12 | Twelve patients in Phase Ⅰ and 7 patients in IIT study had TI for> 6 months (including 13 with β0/β0 genotype). Thirteen patients with ≥12 months of follow-up had an average HbF level of 117 g/L[21] | | CS-101 | CorrectSequence Therapeutics | IIT, NCT06291961 | 8 | The first patient (β0/β+) had TI with HbF > 95 g/L at the 8th week |
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