Table 2.
Biopharmaceutical companies targeting m6A-modifiers as a druggable candidate and current clinical trials.
| Company | Target | Product | Disease | Clinical Trial | Ref. |
|---|---|---|---|---|---|
| Storm TherapeuticsUK, 2015 | Mettl-3, Mettl-1 and ADARI | STC-15, BBB+/-ve 2nd Gen Mettl-3, Mettl-1 and ADARI | Solid tumors, AML, Alzheimer‘s | Pre-clinical & Phase-I NCT05584111 | [143,209,211] |
| Accent TherapeuticsUSA 2017 | DHX9 inhibitor, KIF18A inhibitor. Mettl3, ADAR1 | ATX-559 (an oral inhibitor of DHX), KIF18A inhibitor | TNBC, AML, Solid tumors | Phase-1 NCT06625515 | accenttx.com |
| Gotham Therapeutics/ 8five8 Thera.2014 USA | PARG, Mettl-3, Mettl-14, QPCT/L, ADAR1 | PARG (ETX-19477), QPCT/L, ADAR1 and Mettl-3 inhibitors | Solid tumors, haematological cancers | Pre-clinical & Phase-I NCT06395519 | 8five8tx.com |
| Epics TherapeuticsBelgium, 2018 | Mettl-3 inhibitor | EP102, EP282, ESN-196 (CCR5 agonist) | Solid tumors, Gastrointestinal infla. disorders, AML, HIV | Discovery stage & pre-clinical | [214] |
| 28/7 Therapeutics, Redona Therapeutics USA, 2016 | ncRNA: Lin28/let-7 pathway, CLK, cGAS, TUT4/TUT7, PAPD5/PAPD7 | Lin28 inhibitor, Terminal uridylyl transferase (TUTase targeting) | Solid tumors, Autoimmunity, Telomere disease, HBV | Discovery stage | https://www.twentyeight-seven.com |
| H3 biomedicineUSA, 2011 | SF3b (RNA splicing complex) | H3B-8800 | AML, CML, MDS | Phase-I NCT02841540 | [215,216,227] |
| Ribometrix TherapeuticsUSA, 2018 | c-MYC RNA, eIF4E | c-MYC, eIF4E, Regnase-1 | Solid tumors | Discovery stage | [217] |
| Skyhawk TherapeuticsUSA, 2018 | Small molecule targeting RNA splicing factor | SKY-0515 | Cancer, HD, rare genetic disease | Phase-IACTRN12624000602527 | www.skyhawktx.Com |
| Korro BioUSA, 2014 | Serpin-A1 gene mutation | AATD, ADAR1(OPERA technology) | liver and central nervous system | Discovery stage |
https://www.korrobio.com [218] |
| Wave Life SciencesUSA, 2012 | RNA editing, splicing | WVE-006 (AATD) Serpin-A1 gene | Lung and liver disease (G-to-A point mutation) | Phase-INCT06405633 |
https://www.wavelifesciences.com [219] |
| ProQR Therapeutics, Netherlands, 2012 | Axiomer™ ADAR RNA editing | AX-0810: for NTCP, AX-1412: for B4GALT1 | Cholestatic liver (SLC10A1 gene), and CVD | Proposed clinical trial by 2025 | https://www.proqr.com |