| Bioactivity | IONIS-MAPTRx (BIIB080) is the first Tau-lowering antisense oligonucleotide (ASO). IONIS-MAPTRx has the potential for the research of Alzheimer Disease[1]. |
| In Vivo | Intrathecal administrations of IONIS-MAPTRx in nonhuman primates showed a mean MAPT mRNA reduction of 77% in frontal cortex and 74% in hippocampus without dose-limiting side effects[1]. |
| Name | IONIS-MAPTRx |
| CAS | 2857842-32-9 |
| Molar Mass | 7182.00 |
| Appearance | Solid |
| Transport | Room temperature in continental US; may vary elsewhere. |
| Storage | 4°C, sealed storage, away from moisture *In solvent : -80°C, 6 months; -20°C, 1 month (sealed storage, away from moisture) |
| Reference | [1]. Laurence Mignon, et al. Design of the First-in-Human Study of IONIS-MAPTRx, a Tau-lowering Antisense Oligonucleotide, in Patients With Alzheimer Disease (S2.006). Neurology Apr 2018, 90 (15 Supplement) S2.006. [2]. Rationale for and Development of IONIS-MAPTRx, the First Tau-lowering Antisense Oligonucleotide, in Patients with Mild AD [3]. DeVos SL, Miller RL, Schoch KM, et al. Tau reduction prevents neuronal loss and reverses pathological tau deposition and seeding in mice with tauopathy. Sci Transl Med. 2017;9(374):eaag0481. |