BioCentury
ARTICLE | Translation in Brief

Passage’s mouse, dog data for Krabbe disease; plus a predictive model of COVID-19 mortality, Sorrento’s COVID-19 antibody candidate and more

BioCentury’s roundup of preclinical news

May 15, 2020 11:46 PM UTC

Efficacy for Passage’s Krabbe disease gene therapy
Passage Bio Inc. (NASDAQ:PASG) has presented data supporting an IND it plans to submit next half for PBKR03 to treat Krabbe disease. In mouse and dog models of Krabbe, the adeno-associated virus (AAV) vector encoding GALC increased functional GALC activity and peripheral nerve myelination, normalized peripheral nerve conduction, reduced brain inflammation and improved motor function and survival. The disease, which features central and peripheral nervous system damage, is caused by mutations that reduce GALC activity. Data were presented Tuesday at the virtual American Society of Gene and Cell Therapy (ASGCT) meeting.

Machine learning to predict COVID-19 survival
A Huazhong University of Science and Technology team has developed a machine-learning model to predict the COVID-19 patients at highest risk for mortality based on three blood biomarkers: CRP, LDH and lymphocyte count. The model, described in a Nature Machine Intelligence paper, was trained and validated on blood samples from 485 patients in Wuhan. When applied to an external test data set from 110 patients, the model correctly predicted outcomes about 11 days in advance with an area under the curve (AUC) of 95%...