Vutrisiran: Blood-Brain Barrier

Vutrisiran: Blood-Brain Barrier

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 Summary

Index

Pre-Clinical Data – Abbreviations – References

 Pre-Clinical Data

The PK and ADME properties of vutrisiran were evaluated in various in vitro and in vivo studies to support pivotal toxicology studies and clinical development of vutrisiran.1

Vutrisiran Quantitative Whole-body Autoradiography in Rats

Quantitative tissue distribution of total drug-related radioactivity was investigated in male rats following administration of a single 3 mg/kg SC dose of [3H]‑vutrisiran. [3H]‑Vutrisiran was radiolabeled on the tenth nucleotide (adenosine) from the 3' end of the antisense strand. [3H]-Vutrisiran-derived radioactivity was distributed to limited tissues over time. Vutrisiran did not appear to cross the blood-brain barrier as there was no radioactivity detected in the brain or any part of the central nervous system.1

Abbreviations

ΑDME = absorption, distribution, metabolism, and excretion; PK = pharmacokinetics; SC = subcutaneous

Updated 19 March 2026

References

1.  Alnylam Pharmaceuticals. Data on file. MED-US-TTRSC02-2200053.