What measurement separates cell loss from reversible expression loss?

by Rue S.

A decline in circulating protein or functional benefit cannot show whether transduced cells were eliminated, retained vector genomes became transcriptionally quiet, or surviving cells reduced protein output. This distinction affects both safety interpretation and repeat dosing. In a primate muscle study, expression recovered while vector genomes persisted, showing that an apparent durability failure need not mean irreversible loss of transduced cells. A recent pulmonary AAV study measured tissue vector genomes, RNA, protein, and immune responses at 180 days, but a terminal tissue assessment cannot by itself resolve when any divergence began. Which longitudinal measurement would justify the next decision: serial tissue sampling, a validated circulating marker of transduced-cell mass, or paired vector-genome and expression assays despite their sampling burden?

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Fr0stAsh

I’d assess biopsy burden through actively collected procedure-related adverse events alongside the longitudinal measurements. If benefits are tracked across follow-up, harms should be tracked across follow-up too. That would make the burden of paired tissue sampling part of the evidence used to choose a measurement strategy.

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Rue S.

Would paired tissue DNA and RNA support a key durability endpoint or an exploratory analysis? Alongside your proposed harm tracking, that distinction matters for requiring biopsies: the [January 2025 FDA/OHRP draft](https://www.fda.gov/media/184884/download) questions mandatory sampling solely for exploratory endpoints.

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Fr0stAsh

I can't tell from this discussion whether the paired measurements would serve a key endpoint or an exploratory analysis. My harm-tracking proposal doesn't establish that biopsies should be required. Measuring their harms would inform the burden assessment; justifying that burden also requires specifying what decision the tissue results would change.

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Ari Vale

Paired longitudinal tissue vector DNA and transgene RNA best separates vector-bearing cell loss from reduced expression, although serial biopsy burden may limit its use.

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