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Translation in Brief

James Wilson developing gene therapy version of Regeneron COVID mAb cocktail; plus an oncolytic adenovirus, MapLight-Michael J. Fox Foundation and more

BioCentury’s roundup of translational news

BioCentury’s roundup of translational news.

Dec 5, 2020 | 1:43 AM GMT

Penn, Regeneron on AAV-encoded REGN-COV2
A James Wilson-led University of Pennsylvania team is developing a gene therapy form of casirivimab/imdevimab (REGN-COV2) from Regeneron Pharmaceuticals Inc. (NASDAQ:REGN). The collaboration is based on the potential for a single, intranasal administration of an adeno-associated viral vector encoding the COVID-19 mAb cocktail to enable sustained expression for a longer duration of expression. The two-mAb cockatil gained emergency use authorization from FDA on Nov. 21 to treat high-risk COVID-19 outpatients.

An adenovirus-based oncolytic virus therapy
An article in Science Translational Medicine describes an engineered adenovirus, dubbed Ad5-3M, that suppressed tumor growth and prolonged survival in mice with lung cancer. The mutations in oncolytic virus therapy, generated by Emory University and Case Western Reserve University researchers, enables Ad5-3M to resist inactivation by antibodies, which can also trigger complement and mediate liver toxicity via antibody-mediated sequestration in liver macrophages.

MapLight receives $8.1M grant from Micheal J. Fox Foundation
MapLight Therapeutics Inc. received an $8.1 million grant from The Michael J. Fox Foundation for Parkinson’s Research. The company will use its platform, which includes transcriptomics and optogenetics, to explore the brain circuits and mechanisms that trigger psychiatric symptoms in Parkinson’s disease and identify new drug targets.

A blood-based marker of Alzheimer’s
Stanford University scientists showed in Science Advances that reduced PLCG2 activation in peripheral blood mononuclear cells could be a marker of Alzheimer’s disease. They identified the marker through single-cell profiling of immune stimulant-induced changes in intracellular signaling across 35 peripheral blood cell types derived from patient blood samples.

SARS-CoV-2 can enter the CNS via the olfactory mucosa
A team led by Charité-Universitätsmedizin Berlin researchers showed that SARS-CoV-2 can enter the CNS by crossing the olfactory neural-mucosal interface. They reported in Nature Neuroscience the detection of SARS-CoV-2 RNA and protein in olfactory mucosa cells that had morphologies indicative of neuronal or neural origin.

Dana-Farber, MMRF partner on smoldering multiple myeloma
The Multiple Myeloma Research Foundation and the Dana-Farber Cancer Institute are collaborating to identify markers of high-risk smoldering MM, and develop new treatments to prevent progression to active MM. MMRF is funding the initiative in which data from an in-home genomic blood test will be linked with patients’ whole genome and whole exome sequencing data, circulating tumor cell data and tumor and immune single-cell RNA sequencing data to identify genomic and immune markers that drive disease risk and progression.

TARGETS
PLCG2 – Phospholipase C γ 2 
STAT1 – Signal transducer and activator of transcription 1 
STAT5 – Signal transducer and activator of transcription 5

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