CPT: pharmacometrics & systems pharmacology

How QSP Modeling May Help Design and Improve Gene Therapy

CRISPR Gene Editing Weekly Brief ↗PubMed ↗DOI ↗OA ↗

Updated

Abstract

Essence

modeling may help design and optimize gene therapies by supporting dose selection, biomarker interpretation, and mechanistic translation.

Evidence

This perspective reviews QSP applications and case examples across LNP-delivered mRNA therapeutics, PBPK-informed biodistribution models, and CRISPR-Cas9 editing systems.

Caveat

The argument is based on modeling examples and perspective-level synthesis, with limitations in generalizability, sparse data, and translational relevance explicitly noted.

Simplified

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Funding

Competing interests

3 of 3
authors report competing interests
PubMed
All 3 authors work for Alexion/AstraZeneca Rare Disease, AbbVie, or Neurocrine and received salary and stock from their employer.

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