QurAlis reposted this
The recent FDA approval of #imeltestat is a significant development in #oligonucleotide #therapeutics with many firsts. Imeltestat is the first antagonist, the first N3’-P5’ phosphoramidate backbone chemistry, and the first lipid oligonucleotide conjugate (C16) to be approved in the U.S. C16 conjugates have recently received more attention by companies like Alnylam for the use in their siRNA targeting the #CNS. N3’-P5’ phosphoramidites were introduced by Sergei Gryaznov and coworkers in 1995 and imetelstat (GRN163L) was developed shortly thereafter. It took more than 20 years from lead identification of imeltestat until its FDA approval, but it eventually prevailed. I was privileged to work on this molecule for many years to optimize and scale up the manufacturing process while working at Girindus (now Avecia). Congratulations to the entire imeltestat team for bringing this much-needed new #precisionmedicine to people with MDS.