pH-dependent anti-TfR1 NANOBODY® molecules deliver efficacious oligonucleotide payloads to muscle and CNS tissues

EA Hoyt K. Moonens C. Rapisarda AM Salvador TR Hammond S. Kharade R. Mondragon-Gonzalez F. Moran S. Ramkumar J. Thummapudi S. Zhou G. Haussy C. Capdevila F. Maillard A. Ismail L. Avery P. Sardi C. Nonne S. Cornelis N. Leksa

Aug 2026 · bioRxiv · 0 citations · 1 references

Biology

TL;DR

Extending the half-life of the anti-TfR1 NANOBODY®-ASO shuttles enhances heart, sciatic nerve, and brain exposure and enables up to 30-60% target knockdown in different CNS cell types are highlighted.

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