NTLA-2001
Intellia Therapeutics
['crispr']
phase 1 cm arm of attr-cm in vivo crispr/cas9 gene editor &amp; guide sgrna ntla-2001 based on data circulating precursor ttr protein assoc w better survival 12 patients: median age 75 all men, well tolerated most ae mild 93% ttr by 28d in all pts at .7 &amp; 1 mg/kg dose #aha22 
AssistProfessor #cardiologist #SoMe #editor @CircOutcomes & @JournalASEcho #BOD @ase360 #ACCWIC @PittHealthSci #MedHumanities @ColumbiaMed @NUFeinbergMed