In principle, any RNA molecule in the transcriptome can be inhibited using nucleic acid based drugs, using for instance antisense technology. The newest generation of such nucleic acid based drugs are self-delivering, obviating the need for a delivery vehicle to cross cell membranes in the body and bypassing a major challenge in targeted drug development.
Once inside the cell, these drugs typically display high target-specificity, being able to distinguish a single nucleotide mismatch. Compared to small molecules, rational drug development using nucleic acid based drugs has a much faster development track.
Related topics: RNA sequencing, non-coding RNA, test development, data analysis, quality, why Biogazelle, Biogazelle’s vision and mission
Speed up your antisense oligo drug development with our unique massively parallel
in vitro antisense oligo efficacy screen, using a fully optimized high-throughput workflow.
Biogazelle supports the development of RNA based therapeutics
Biogazelle has extensive experience with various antisense technologies to modulate RNA expression. Through four unique modules, we provide expertise to support your antisense oligonucleotide (ASO) drug development pipeline.
- in vitro ASO efficacy screening
In the absence of efficient ASO design algorithms, experimental identification of efficient ASOs against the RNA target of interest is required. Using a fully optimized high-throughput workflow, Biogazelle simultaneously tests hundreds to thousands of ASOs in vitro. This module results in the selection of ASOs that induce knockdown of the target RNA in a dose-responsive manner.
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ASO specificity analysis
Knowing potential side effects and off-target related toxicity, allows you to further prioritize candidate ASOs. Via in silico assessment and experimental validation of putative off-target transcripts in human and preclinical model systems, Biogazelle maps ASO off-target effects of candidate ASOs. This module results in a list of validated off-target genes for each candidate ASO. Read more about our expertise in data analysis and custom in silico analyses.
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understanding drug mode of action
Insight in pathways affected by ASO administration, allows rational selection of combination therapies. Via gene expression profiling and differential gene and pathway analysis in relevant cell lines treated with candidate ASOs, Biogazelle provides a list of genes and pathways regulated directly or indirectly by the candidate ASOs, providing direct insights in the pathways in which the therapeutic target plays a role. Read more about our expertise in data analysis and custom in silico analyses.
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development of a companion diagnostic test
One of the key advantages of RNA targeting therapies is that the RNA molecule is not only the therapeutic target, but also the companion or complementary diagnostic target. Via gene expression profiling of blood or plasma samples, Biogazelle assesses the circulation of the target, providing a non-invasive diagnostic test and enabling precision medicine. Read more about our expertise in liquid biopsies and biomarker test development.
Antisense oligo screening at Biogazelle
- We have a proven scientific background with more than a decade of expertise in gene expression and more advanced analysis, supported by more than 15,000 citations of peer reviewed articles. Find our publications.
- We have internationally recognized expertise the field of RNA.
- We have proprietary knowledge to prioritize candidate therapeutic RNA targets in specific cell and disease types, building on years of expertise in exploring the transcriptome as 'RNA solutions' service provider
- our workflow for ASO specificity analysis includes the recommendations regarding the evaluation of ASO off-target effects before moving into clinical trials, described by the oligonucleotide safety workgroup in a letter to the editor published in Nature Biotechnology.