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Translation in Brief

Dyne’s POC myotonic dystrophy data; plus pulmonary T cell subset contributes to local antibody production after reinfection, DiosCURE’s COVID candidates and more

BioCentury’s roundup of translational news

BioCentury’s roundup of translational news.

Jan 16, 2021 | 2:37 AM GMT

Dyne’s myotonic dystrophy antisense therapy reduces gene expression
Dyne Therapeutics Inc. (NASDAQ:DYN) reported data from a new mouse model of severe disease showing its myotonic dystrophy type 1 (DM1) antisense oligonucleotide therapy reduced nuclear DMPK RNA levels by 39-60% in the heart, diaphragm, tibialis anterior and gastrocnemius. The mice express human TFRC; and the company’s  FORCE platform conjugates nucleic acids to anti-TFRC antibody fragments to delivery payloads to muscle cells. Dyne plans to submit an IND for the DM1 program and its two others between 4Q21 and 4Q22.

T resident helper cells promote antibody responses in the lung
An article in Science Immunology revealed a population of CD4+ T cells in inducible bronchus-associated lymphoid tissue (iBALT) that are dependent on BCL6-mediated interactions with B cells, and present in the tissue during flu infection. The new cell subset, dubbed T resident helper cells (TRH), localize with memory B cells and are required for antibody production. A University of Basel team showed that BCL6 deletion in CD4+ T cells resulted in redistribution of the cells outside iBALT and impaired local antibody production in response to influenza reinfection.

DiosCURE’s multivalent camelid antibodies for COVID-19
DiosCURE Therapeutics SE, which launched in October, is developing multivalent single-domain antibodies for COVID-19. A team led by University of Bonn and Karolinska Institute scientists described, in Science, the generation of four neutralizing antibodies including DiosCURE’s candidate that were over 100 times more potent than monovalent single-domain antibodies in SARS-CoV-2 virus and pseudovirus neutralization assays. The multivalent camelid antibodies also suppressed the emergence of escape mutants.

Prenatal mRNA delivery using nanoparticles
University of Pennsylvania researchers have developed a library of ionizable lipid nanoparticles (LNPs) for in utero mRNA delivery. In proof-of-concept experiments reported in Science Advances, the mRNA delivered in utero accumulated in mouse fetal livers, lungs and intestines; and prenatal delivery of human erythropoietin mRNA with the technology induced fetal hepatic production of erythropoietin protein.

TARGETS
BCL6 – B cell CLL lymphoma 6 
DMPK – Dystrophia myotonica-protein kinase
SARS-CoV-2 S – SARS-CoV-2 spike protein 
TFRC (TFR) (CD71) – Transferrin receptor 

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