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Stoke Therapeutics Announces Publication of Data in the Journal Nature Communications That Support the Company’s Proprietary Approach to Addressing Severe Genetic Diseases by Precisely Upregulating Protein Expression
Stoke’s TANGO antisense oligonucleotides showed dose-dependent reductions in non-productive mRNA and increases in both productive mRNA and protein levels
About this update from Stoke Therapeutics, Inc.
[{"type":"text","content":"\nStoke’s TANGO antisense oligonucleotides showed dose-dependent reductions in non-productive mRNA and increases in both productive mRNA and protein levels from genes of diverse size, type and function\n\n BEDFORD, Mass.--(BUSINESS WIRE)--\nStoke Therapeutics, Inc. (Nasdaq: STOK), a biotechnology company pioneering a new way to treat the underlying cause of severe genetic diseases, today announced the publication of data in the journal Nature Communications that support the company’s proprietary approach to precisely upregulate protein expression using TANGO (Targeted Augmentation of Nuclear Gene Output) antisense oligonucleotides (ASOs).\nThis press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20200709005385/en/TANGO (Targeted Augmentation of Nuclear Gene Output)\n\n“Stoke was founded on the idea that we could use unique insights in RNA biology to do something that has never been done before,” said Isabel Aznarez, Ph.D., Co-Founder and Vice President, Head of Biology of Stoke Therapeutics and the corresponding author on the paper. “Rather than address genetic diseases by replacing, repairing or editing faulty genes, we set out to increase – or stoke – protein output from healthy genes. These data show that we can increase full-length, fully functional protein expression from a variety of healthy genes, which supports our hypothesis and may lead to a new way of treating severe genetic diseases.”\n\n\nTo evaluate the approach broadly, Stoke selected four gene targets that vary in type and abundance of non-productive splicing events, gene size and protein function: PCCA (propionic acidemia); SYNGAP1 (autosomal dominant mental retardation 5); CD274 (autoimmune diseases, including uveitis); and SCN1A (Dravet syndrome). Stoke designed TANGO ASOs to target the non-productive splicing events in these genes and their activity was evaluated. Dose-dependent reductions of non-productive mRNA were observed to lead to increases in both productive mRNA and protein levels for each of the target genes.\n\n\nMore than 10,000 genetic diseases are caused by mutations in a single gene, however, current therapeutic approaches address as few as 5% of these diseases. In the experiments published today, a proprietary bioinformatics analysis of RNA sequencing datasets was used to identify a variety of n...