Bioactivity | PEG2000-C-DMG, a pegylated lipid, can be used for the preparation of Onpattro. Onpattro, a hepatically directed investigational RNAi therapeutic agent, harnesses this process to reduce the production of mutant and wild-type transthyretin by targeting the 3′ untranslated region of transthyretin mRNA[1][2]. |
Name | PEG2000-C-DMG |
Formula | C126H249NO52 |
Molar Mass | 2610.29 |
Appearance | Solid |
Transport | Room temperature in continental US; may vary elsewhere. |
Storage | -20°C, stored under nitrogen *In solvent : -80°C, 6 months; -20°C, 1 month (stored under nitrogen) |
Reference | [1]. Garber K. Alnylam launches era of RNAi drugs. Nat Biotechnol. 2018;36(9):777-778. [2]. Adams D, Gonzalez-Duarte A, O'Riordan WD, et al. Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis. N Engl J Med. 2018;379(1):11-21. |