Alnylam preclinical data latest sign companies still plugging away at oral oligos

Proof-of-concept mouse data from Alnylam suggests the company has found a formulation that supports oral delivery of its oligonucleotide therapy, a long-standing goal in the field that has produced splashy deals but few clinical results.

In a presentation announced Friday, Alnylam Pharmaceuticals Inc. (NASDAQ:ALNY) showed that combining its GalNAc RNAi chemistry with an undisclosed formulation containing a permeation enhancer enabled dose-dependent reduction of serum levels of Factor XII, a coagulation factor expressed in the liver. Potency and duration of Factor XII knockdown by the oral formulation were similar to that induced by same GalNAc RNAi delivered subcutaneously. ...