Data from ongoing Phase 1 study of AX-0810 in healthy volunteers demonstrated dose-dependent target engagement on all key biomarkers in the evaluable 3 mg/kg and 6 mg/kg cohortsAX-0810 demonstrated up to 8-fold change (6 mg/kg) in total bile acids in serum, exceeding the
ProQR Therapeutics today announced positive results from a planned analysis of its Phase 1/2 Stellar trial of QR-421a in adults with Usher syndrome and non-syndromic retinitis pigmentosa (nsRP) due to USH2A exon 13 mutations.