Rovanersen  (Synonyms: WVE-120101)

Rovanersen (WVE-120101) is an antisense oligonucleotide that specifically targets mutated mRNA copies of the huntington (HTT) gene without affecting healthy mRNA of HTT gene, thereby preventing the production of faulty Huntingtin protein. Rovanersen can be used for huntington’s disease research^[1].

In vitro, Rovanersen (WVE-120101) leads to the selective RNase H cleavage of mutant Huntingtin allele (mHTT) over wtHTT, with no complement activation. Rovanersen selectively decreases mHTT mRNA and protein levels compared with wtHTT in multiple cells lines^[1].

MCE has not independently confirmed the accuracy of these methods. They are for reference only.

In non-human primates (NHPs), Rovanersen (WVE-120101) is found in nuclear and perinuclear compartments of neurons in brain areas believed to be involved in HD pathology^[1].

MCE has not independently confirmed the accuracy of these methods. They are for reference only.

6617.00

2072901-32-5

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White to off-white

DNA,d(rGm-[P(S)]-sp-rGm-rCm-rAm-rCm-[P(S)]-sp-A-[P(S)]-sp-A-[P(S)]-sp-G-[P(S)]-sp-G-[P(S)]-sp-G-[P(S)]-sp-C-[P(S)]-sp-A-[P(S)]-sp-C-[P(R)]-sp-A-[P(S)]-sp-G-[P(S)]-sp-rAm-rCm-rUm-rUm-[P(S)]-sp-rCm)

Room temperature in continental US; may vary elsewhere.

-20°C, stored under nitrogen

*In solvent : -80°C, 6 months; -20°C, 1 month (stored under nitrogen)

• [1]. M Meena, et al. Selectivity and Biodistribution of WVE-120101, a Potential Antisense Oligonucleotide Therapy for the Treatment of Huntington’s Disease (S56.001). AAN Enterprises. April 18, 2017; 88 (16 Supplement).