BioCentury
ARTICLE | Translation in Brief

A sickle cell disease gene therapy; plus a dengue virus inhibitor, a small molecule to enhance memory in Alzheimer’s and a cytokine that mediates synaptic function

BioCentury’s roundup of translational news

February 9, 2021 12:00 AM UTC

A team led by Matthew Porteus at Stanford University corrected the HBB gene in hematopoietic stem cells ex vivo and showed that, after autologous transplantation in a Townes mouse model of sickle cell disease, the cells produced hemoglobin A and led to reduced red blood cell abnormalities. Described in Nature Communications, the researchers corrected the gene using Cas9-mediated genome cutting in combination with recombinant adeno-associated virus serotype 6 (AAV6) homologous recombination; a method that Porteus is employing at Stanford spinout Graphite Bio Inc.

Novartis’ dengue virus inhibitor reduces viremia
Novartis Institutes for BioMedical Research scientists revealed in Science Translational Medicine that NITD-688, a dengue virus nonstructural protein 4B inhibitor, reduced viremia in infected mice lacking interferon-ɑ, β and γ receptors. Studies in rats and dogs demonstrated that NITD-688 was well-tolerated over seven days of repeat dosing...