Quality of life outcomes in APOLLO, the phase 3 trial of the RNAi therapeutic patisiran in patients with hereditary transthyretin-mediated amyloidosis

April 30th, 2020|Categories: Ago/siRNA/saRNA, Clinical Development|

Obici L, Berk JL, González-Duarte A, Coelho T, Gillmore J, Schmidt HH, Schilling M, Yamashita T, Labeyrie C, Brannagan TH 3rd, Ajroud-Driss S, Gorevic P, Kristen AV, Franklin J, Chen J, Sweetser MT, Wang JJ, Adams D. Amyloid. 2020 Mar 4:1-10. PubMed

MTL-CEBPA, a small activating RNA therapeutic up-regulating C/EBP-α, in patients with advanced liver cancer: a first-in-human, multi-centre, open-label, phase I trial

April 30th, 2020|Categories: Ago/siRNA/saRNA, Clinical Development|

Sarker D, Plummer R, Meyer T, Sodergren M, Basu B, Chee CE, Huang KW, Palmer DH, Ma YT, Evans TRJ, Spalding DR, Pai M, Sharma R, Pinato DJ, Spicer J, Hunter S, Kwatra V, Nicholls JP, Collin D, Nutbrown R, Glenny H, Fairbairn S, Reebye ...

Chimeric siRNAs with chemically modified pentofuranose and hexopyranose nucleotides: altritol-nucleotide (ANA) containing GalNAc-siRNA conjugates: in vitro and in vivo RNAi activity and resistance to 5′-exonuclease

March 31st, 2020|Categories: Ago/siRNA/saRNA|

Kumar P, Degaonkar R, Guenther DC, Abramov M, Schepers G, Capobianco M, Jiang Y, Harp J, Kaittanis C, Janas MM, Castoreno A, Zlatev I, Schlegel MK, Herdewijn P, Egli M, Manoharan M. Nucleic Acids Res. 2020 Mar 14. PubMed

Go to Top