Volanesorsen
Volanesorsen is a triglyceride-reducing drug. It is a second-generation[2] 2’-O-methoxyethyl (2’-MOE) chimeric antisense therapeutic oligonucleotide (ASO)[3] that targets the messenger RNA for apolipoprotein C3 (apo-CIII).
Clinical data | |
---|---|
Other names | ISIS 304801, ISIS-APOCIIIRx |
Routes of administration | Subcutaneous injection |
ATC code |
|
Identifiers | |
IUPAC name
| |
CAS Number |
|
PubChem CID | |
ChemSpider | |
UNII | |
KEGG | |
Chemical and physical data | |
Formula | C230H320N63O125P19S19 |
Molar mass | 7165.05 g·mol−1 |
SMILES
| |
InChI
|
It is in phase III clinical trials for the treatment of hypertriglycidemia, familial chylomicronemia syndrome and familial partial lipodystrophy.[4][5]
The drug was discovered and developed by Ionis Pharmaceuticals.
Chemistry
The complete sequence of volanesorsen is:[1]:165–166
3’—A*—G*—mC*—T*—T*—dmC—dT—dT—dG—dT—dmC—dmC—dA—dG—dmC—T*—T*—T*—A*—T*—5’
*
= 2’-O-(2-methoxyethyl)
m
= 5-methyld
= 2’-deoxy
References
- "International Nonproprietary Names for Pharmaceutical Substances (INN). Recommended International Nonproprietary Names: List 75" (PDF). World Health Organization. Retrieved 18 March 2017.
- Pechlaner, R; Tsimikas, S; Yin, X; Willeit, P; Baig, F; Santer, P; Oberhollenzer, F; Egger, G; Witztum, JL; Alexander, VJ; Willeit, J; Kiechl, S; Mayr, M (21 February 2017). "Very-Low-Density Lipoprotein-Associated Apolipoproteins Predict Cardiovascular Events and Are Lowered by Inhibition of APOC-III". Journal of the American College of Cardiology. 69 (7): 789–800. doi:10.1016/j.jacc.2016.11.065. PMC 5314136. PMID 28209220.
- Graham, MJ; Lee, RG; Bell TA, 3rd; Fu, W; Mullick, AE; Alexander, VJ; Singleton, W; Viney, N; Geary, R; Su, J; Baker, BF; Burkey, J; Crooke, ST; Crooke, RM (24 May 2013). "Antisense Oligonucleotide Inhibition of Apolipoprotein C-III Reduces Plasma Triglycerides in Rodents, Nonhuman Primates, and Humans". Circulation Research. 112 (11): 1479–1490. doi:10.1161/CIRCRESAHA.111.300367. PMID 23542898.
- Taylor, Nick Paul (20 December 2016). "Ionis' volanesorsen hits efficacy endpoint in phase 3". www.fiercebiotech.com. Retrieved 22 December 2016.
- Digenio, Andres; Dunbar, Richard L.; Alexander, Veronica J.; Hompesch, Marcus; Morrow, Linda; Lee, Richard G.; Graham, Mark J.; Hughes, Steven G.; Yu, Rosie; Singleton, Walter; Baker, Brenda F.; Bhanot, Sanjay; Crooke, Rosanne M. (August 2016). "Antisense-Mediated Lowering of Plasma Apolipoprotein C-III by Volanesorsen Improves Dyslipidemia and Insulin Sensitivity in Type 2 Diabetes". Diabetes Care. 39 (8): 1408–1415. doi:10.2337/dc16-0126. PMID 27271183.
External links
This article is issued from
Wikipedia.
The text is licensed under Creative
Commons - Attribution - Sharealike.
Additional terms may apply for the media files.