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Nature paper on RGLS4326Regulus have published their preclinical work on RGLS4326 for ADPKD in Nature. There’s no mention that I could see of the mouse tox that led to the clinical hold. I was interested to note that RGLS4326 uses a mixture of cEt, 2’-F and 2’O-Me modifications.
https://www.nature.com/articles/s41467-019-11918-y#Fig6
One interesting aspect is the preferential distribution of RGLS4326 to the kidney (8 to 13x the exposure in liver) which I think is unusual for an oligo. If they can figure out why, this would enable a unique kidney targeting capability.
I’ve pasted the discussion section below and highlighted the passage that affirms what got me interested in Regulus in the first place - the potential to normalise gene expression in complex diseases.
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