The compound is an antisense oligonucleotide inhibiting certain micro-RNAs in heart tissue that regulate the pathological growth of heart muscle cells. In the trial, CDR132L met all endpoints and showed excellent tolerability and safety. The infusion was very well tolerated by all patients, and no safety signals or unexpected adverse events were observed. Moreover, PK data showed strong dose-dependent linearity and specific target engagement could be confirmed. These data further support the potential of RNA-based therapies as a causal approach to treat heart diseases.
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