Patisiran: Blood Brain Barrier

Patisiran: Blood-Brain Barrier

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 Summary

Index

Pre-Clinical Data – Abbreviations – References

 Pre-Clinical Data

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

Patisiran Quantitative Whole-body Autoradiography in Rats

Quantitative tissue distribution of total drug-related radioactivity was investigated in male rats following a single IV bolus injection of patisiran-LNP containing a radio-label on the lipid component ([14C]-DLin-MC3-DMA). In the QWBA study, there were minimal amounts of radioactivity in the central nervous system suggesting patisiran-LNP does not penetrate the blood-brain barrier.1

Abbreviations

ADME = absorption, distribution, metabolism, and excretion; IV = intravenous; LNP = lipid nanoparticle; PK = pharmacokinetics; QWBA = quantitative whole-body autoradiography

Updated 19 March 2026

References

1.  Alnylam Pharmaceuticals. Data on file. MED-ALL-TTR02-2000182.