Andrew, golodirsen, like eteplirsen, is a PMO. My understanding is that there's no carrier involved; certainly no vector.
The reported toxicity in kidney occurred in preclinical when the drug was administered at very high dose levels. Other studies have also demonstrated toxicity with higher doses of antisense oligonucleotides.
In the clinical trial for golodirsen, the drug was administered via 30 mg/kg/week IV infusions, weekly. As far as I can see, preclinical studies took the dosage up to 320mg/kg in monkeys.
A paper on the safety and PK profile of golodirsen, eteplirsen and another Sarepta PMO, which was authored by Sarepta scientists, claimed that IND enabling preclinical studies presented no problems. “There were no hospitalizations, discontinuations, or clinically significant treatment-related changes for lab parameters, including liver enzymes, kidney function, coagulation, or platelet counts.”
The only mention of effect in kidney I’ve found was in a Sarepta presentation, which stated that golodirsen demonstrated “mild, reversible, non-adverse microscopic changes in the kidney” in the preclinical monkey trial.
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