On Oct. 18, 2021, LogicBio Therapeutics announced clinical trial results that demonstrated the first-ever in vivo genome editing in children. Early data from the company’s Phase 1/2 SUNRISE clinical trial showed measurable levels of albumin-2A, a technology-related biomarker indicating site-specific gene insertion and protein expression.

The SUNRISE trial evaluated the safety, tolerability and preliminary efficacy of LB-001, the company’s investigational, single-administration genome editing therapy, in pediatric patients with methylmalonic acidemia.