LogicBio Therapeutics Announces Early Clinical Trial Results Demonstrating First-Ever In Vivo Genome Editing in Children
October 18, 2021
- Early data from Phase 1/2 clinical trial in pediatric patients with methylmalonic acidemia showed measurable levels of a
biomarker indicating site-specific gene insertion and protein expression
- Based on safety data from first two patients, independent Data Safety Monitoring Board recommended continuation of
trial, enabling enrollment of children as young as six months and dose escalation
Company remains on track to report additional interim clinical data by end of 2021
LEXINGTON, Mass., Oct. 18, 2021 /PRNewswire/ — LogicBio Therapeutics, Inc. (Nasdaq:LOGC), a clinical-stage genetic medicine company, today
announced clinical trial results demonstrating 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 is evaluating the safety, tolerability and preliminary efficacy of LB-001, the company’s investigational, single-administration genome
editing therapy, in pediatric patients with methylmalonic acidemia (MMA).