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Oligonucleotide-based therapeutics (OLNs), including antisense oligonucleotides (ASOs) and small interfering RNAs (siRNAs), modulate gene expression at the RNA level, offering powerful tools for treating genetic disorders. Oligonucleotide drugs can be designed to specifically target and modulate the expression of disease-related genes or proteins.
By exploiting complementary base pairing, oligonucleotides can selectively bind to mRNA sequences, leading to degradation, inhibition of translation, or modulation of splicing, resulting in precise and targeted therapeutic effects. They typically exhibit high specificity for their target sequences, minimizing off-target effects on unrelated genes or proteins which enhances their safety profile. This high specificity also reduces the risk of drug-drug interactions with conventional small-molecule drugs or biologics and simplifies treatment regimens in combination therapies.
Going beyond natural oligonucleotide constructs by using chemical modifications, such as backbone modifications, nucleobase modifications, or conjugation with delivery vehicles, can enhance the potency, specificity, and bioavailability of oligonucleotide therapeutics.
In recent years, experts at SpiroChem have gained hands-on experience in oligonucleotide chemistry in both scale-up solutions and discovery of new methodologies (ribose and morpholine modifications) as well as different conjugation chemistries for delivery (GalNAc, cholesterol). Innovative chemistry strategies are developed to prepare nucleoside analogues modified on the nitrogenous base, and/or sugar, and/or phosphate moieties to provide compounds with increased response rates and reduced side effects.
Our chemists are also highly skilled in the design and preparation of:
By integrating OLNs into drug discovery pipelines, we help clients develop targeted therapies that address the root causes of genetic diseases. Our expertise in OLNs enables us to deliver innovative solutions that advance the field of genetic therapeutics.
We work closely with clients to understand their specific needs and tailor our approaches to maximize therapeutic potential and safety. Our state-of-the-art facilities and advanced analytical tools ensure that each OLN we develop meets the highest standards of quality and performance, driving progress in the treatment of genetic disorders.
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