Summary of therapies targeting RNA splicing described in Fig. 3
| Target . | Agent . | Phase . | Patient population . | References . |
|---|---|---|---|---|
| SF3B1 | H3B-8800 | 1 | MDS, AML, CMML | Steensma et al. (2021) |
| RBM39 degrader | Indisulam + idarubicin and cytarabine | 2 | R/R AML, HR-MDS | Assi et al. (2018) |
| E7820 | 2 | R/R splicing factor-mutant AML, MDS, or CMML | Bewersdorf et al. (2023b) | |
| PRMT inhibitor | GSK3326595 monotherapy or + azacitidine | 1/2 | R/R MDS, CMML, AML evolved from prior MDS | Watts et al. (2019) |
| PRT543 | 1 | MDS, MF | Patel et al. (2021) | |
| JNJ-64619178 | 1 | Low-risk MDS | Haque et al. (2021) | |
| CLK inhibitor | CTX-712 | 1/2 | R/R AML, HR-MDS | Al-Kali et al. (2023), Shimizu et al. (2022) |
| ASO | BP1001 monotherapy or + cytarabine | 1 | AML, MDS, CML-BP | Ohanian et al. (2020) |
| Cenersen (EL625) + cytarabine and idarubicin | 2 | R/R AML | Alvarado et al. (2007) | |
| Cenersen (EL625) + idarubicin with or without cytarabine | 2 | R/R AML | Cortes et al. (2012) | |
| RNA capping | Ribavirin + cytarabine | 1 | R/R AML | Assouline et al. (2015) |
| RNA polyadenylation | JTE-607 targeting of CPSF3 | N/A | Preclinical | Ross et al. (2020) |
| UHM/ULM | NSC 194308 | N/A | Preclinical | Chatrikhi et al. (2021) |
| Target . | Agent . | Phase . | Patient population . | References . |
|---|---|---|---|---|
| SF3B1 | H3B-8800 | 1 | MDS, AML, CMML | Steensma et al. (2021) |
| RBM39 degrader | Indisulam + idarubicin and cytarabine | 2 | R/R AML, HR-MDS | Assi et al. (2018) |
| E7820 | 2 | R/R splicing factor-mutant AML, MDS, or CMML | Bewersdorf et al. (2023b) | |
| PRMT inhibitor | GSK3326595 monotherapy or + azacitidine | 1/2 | R/R MDS, CMML, AML evolved from prior MDS | Watts et al. (2019) |
| PRT543 | 1 | MDS, MF | Patel et al. (2021) | |
| JNJ-64619178 | 1 | Low-risk MDS | Haque et al. (2021) | |
| CLK inhibitor | CTX-712 | 1/2 | R/R AML, HR-MDS | Al-Kali et al. (2023), Shimizu et al. (2022) |
| ASO | BP1001 monotherapy or + cytarabine | 1 | AML, MDS, CML-BP | Ohanian et al. (2020) |
| Cenersen (EL625) + cytarabine and idarubicin | 2 | R/R AML | Alvarado et al. (2007) | |
| Cenersen (EL625) + idarubicin with or without cytarabine | 2 | R/R AML | Cortes et al. (2012) | |
| RNA capping | Ribavirin + cytarabine | 1 | R/R AML | Assouline et al. (2015) |
| RNA polyadenylation | JTE-607 targeting of CPSF3 | N/A | Preclinical | Ross et al. (2020) |
| UHM/ULM | NSC 194308 | N/A | Preclinical | Chatrikhi et al. (2021) |
Abbrevations: BP: blast phase. HR-MDS: high-risk MDS. MF: myelofibrosis. R/R: relapsed/refractory.