PeptiDream and Alnylam Pharmaceuticals announced they have reached a key preclinical milestone in their multi-year collaboration to develop peptide-siRNA conjugates capable of delivering small interfering RNA (siRNA) therapeutics to specific extrahepatic tissues outside the liver—a longstanding challenge in RNA interference (RNAi) drug development. The achievement highlights progress in building a modular, scalable therapeutic platform that could broaden the scope of RNAi medicines across diverse disease areas.
Using PeptiDream’s Peptide Discovery Platform System® (PDPS), the peptide-siRNA conjugates demonstrated robust and targeted uptake in predetermined tissues in wild-type animal models following subcutaneous administration, coupled with deep knockdown of the target gene. The specificity of the peptide-mediated delivery was confirmed in receptor knockout models, where negligible activity was observed without the intended receptor interaction, underscoring the precision of the targeting mechanism.
This milestone validates the potential of peptide ligand-mediated delivery to enable RNAi therapies beyond the liver—where current GalNAc-based conjugates predominantly act—and could open paths to treating indications involving tissues such as muscle, lung, CNS and others not readily accessible with existing delivery technologies.
PeptiDream will receive a milestone payment from Alnylam under the collaboration terms and remains eligible for future development, regulatory and commercial milestones, as well as royalties on any resulting products. The teams began the collaboration in 2021, combining PeptiDream’s peptide discovery expertise with Alnylam’s RNAi therapeutic development capabilities to expand precision delivery solutions.
