pH-dependent anti-TfR1 NANOBODY(R) molecules deliver efficacious oligonucleotide payloads to muscle and CNS tissues
Emily A Hoyt, Kristof Moonens, Chiara Rapisarda, Ane Miren Salvador, Timothy R. Hammond, Sujay V. Kharade, Ricardo Mondragón-González, Faith Elaine Yuantiao Moran, Shruthi Ramkumar, Jayaprakash Thummapudi, Shan Zhou, Gilles Haussy, Cécile Capdevila, Fiona Maillard, Ayman Ismail, , Nina C. Leksa
This study develops anti-TfR1 NANOBODY(R) molecules that deliver siRNA and ASO payloads to muscle and CNS tissues, achieving significant mRNA knockdown.
bioRxiv · Aug 3, 2026 · PreprintNeuroscience