growth in sRNA focused biomaker publications over the last decade1
3%
of clinical trials in 2020 used sRNA biomarkers2
34
ODN drugs in active clinical trials3
9
FDA Approved ODN therapeutics4
Successful drug development requires a comprehensive view of sRNA biology
Powerful...
sRNA regulates all gene expression, driving disease and maintaining health
…but Complex
functionality is contextual, requiring a full picture of sRNA biology
Current approaches miss important aspects of sRNA biology
Annotation of transcript variants and non-templated nucleotides
Predicting regulatory targets and mechanism of action
Differential regulatory targets based on sRNA variants
Extracellular and cellular sRNA localization signals
Starting at the source: how sRNA variants drive disease3
GUIDED BY THE RISC COMPLEX, NON-CODING sRNAs IN THE GENOME MODIFY PROTEIN EXPRESSION
These sRNAs make up 80% of the genome and are crucial in human health and disease3,4
ANALYSIS OF RISC SHOWS THERE ARE MANY CLASSES OF FUNCTIONAL SMALL RNAS
Only 6% of non-coding sRNAs contained in the RISC complex are miRNA
SINGLE NUCLEOTIDE VARIANTS IN THESE SMALL RNAs DRIVE DISEASE BY DISRUPTING PROTEIN EXPRESSION
sRNA processing errors
Non-templated nucleotide additions
Polymorphic variants or RNA editing
'Seed' sequence
– directs RISC to target site RNA
Current data processing and annotation methods condense and obscure the sRNA that inform disease heterogeneity
Next: Applying the approach to accelerate R&D
REFERENCES:
1. National Center for Biotechnology Information. Accessed June 16, 2021. https://pubmed.ncbi.nlm.nih.gov/
2. National Library of Medicine. Accessed June 16, 2021. https://clinicaltrials.gov/
3. Data on file, Gatehouse Bio; 2021.
4. The ENCODE Project Consortium. An integrated encyclopedia of DNA elements in the human genome. Nature. 2012;489:57-74.
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David Salzman, PhD
As founder and CEO of Gatehouse Bio, David has over 14 years of research expertise and experience in microRNA and sRNA biology. He is the force behind the science and technological development required for identifying sRNA diagnostics, targeted assay design, validation, and subsequent product development. He was previously a postdoctoral fellow at the Yale School of Medicine and a scientist at Biogen, where he gained experience in developing clinical and preclinincal biomarker, PK/PD, and diagnostic platforms. David also served as the CSO of MiraDx, spearheading projects to commercialize germline DNA biomarkers in oncology. He earned his PhD in Molecular Biology and Biochemistry from the University of Connecticut and a BS in Environmental Science from the University of Hartford.
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