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March 26, 2026Cell and Gene Therapy Insights

From dose quantity to dose quality: elevating rAAV genome integrity as a CMC attribute

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Authors

XWXushan WangSRSarah M. RicherBHBeverly A. Heinz

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Overview

This framework elevates genome integrity metrics in rAAV programs, suggesting implications for development decisions.

Key Points

  • The aim is to enhance the assessment of rAAV genome integrity as a critical quality attribute for dosing.
  • Outlined a mechanism-based framework for assessing DNA-related dose quality.
  • Summarized analytical limitations in quantifying intact genomes.
  • Described platform-dependent outcomes affecting dose quality.
  • Proposed a strategy for incorporating genome integrity into development and regulatory decisions.
  • Identified that intact genomes correlate with biologically effective exposure.
  • Showed that improving intact genome fraction can lower clinical vg dosing needs.
  • Highlighted the importance of genome integrity for systemic administration at high doses.

Cite This Study

Wang et al. (2026) studied this question.

synapsesocial.com/papers/69c4ccc9fdc3bde44891854ahttps://doi.org/10.18609/cgti.2026.020
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