gene-therapy

The FDA has cleared Intellia Therapeutics to resume its MAGNITUDE Phase 3 trial of nexiguran ziclumeran for ATTR cardiomyopathy, five months after a patient death and subsequent clinical hold prompted a safety overhaul.

MagicRNA's mRNA lipid nanoparticle therapy reprogrammed patients' own T cells inside the body, wiping out disease-driving B cells in refractory lupus patients without conventional cell manufacturing.

The FDA rejected uniQure's Phase I/II data for AMT-130 and now demands a sham surgery-controlled Phase 3 trial, reversing its own prior guidance and sending shares down 40 percent.

UNSW and St. Jude researchers demonstrate that removing methyl tags from DNA can switch genes back on, opening safer paths for treating sickle cell disease.