Oligonucleotide Drug Development

Overview

Oligonucleotide drugs are synthetic molecules designed to interact with specific nucleic acid targets within cells, exerting their therapeutic effects through various mechanisms such as gene silencing, mRNA degradation, and splicing modulation.

Therapeutic Field

Oligonucleotide drugs hold promise for treating a wide range of diseases, including neurodegenerative disorders, genetic diseases, and cancer.

(i) The ability to use antisense oligonucleotides (ASOs) to target disease-associated genes by means of RNA may provide an effective approach for the treatment of neurodegenerative disorders ^[1]. For example, Huntington disease (HD) is a fatal progressive neurodegenerative disorder caused by a genetic mutation in the huntingtin (HTT) gene ^[2]. ASOs can reduce HTT by targeting transcripts, so they are very suitable for the treatment of this disease.

(ii) In genetic disorders, splice-switching oligonucleotides (SSOs) hold promise for correcting splicing defects and restoring functional protein expression in diseases like duchenne muscular dystrophy (DMD) and spinal muscular atrophy (SMA).

(iii) Furthermore, siRNA-based therapeutics demonstrate efficacy in silencing oncogenes and inhibiting tumor growth, heralding a new era in cancer therapy with personalized RNA-based interventions.

Challenges and Innovations

Despite the remarkable potential of oligonucleotide therapeutics, several challenges remain to be addressed. These include delivery barriers, off-target effects, immunogenicity, and manufacturing scalability. Innovative delivery strategies such as lipid nanoparticles, conjugation with targeting ligands, and exosome-mediated delivery are being developed to enhance oligonucleotide uptake, biodistribution, and intracellular release. Moreover, chemical modifications and formulation optimizations are employed to improve oligonucleotide stability, potency, and safety profiles, paving the way for next-generation oligonucleotide drugs with enhanced therapeutic efficacy and reduced side effects.

Choose Us

With ongoing advancements in technology, delivery systems, and chemical modifications, oligonucleotide therapeutics are poised to revolutionize disease treatment paradigms, ushering in an era of personalized medicine tailored to individual genetic profiles. Our company has many years of experience in oligonucleotide synthesis. We have developed a stable and environmentally friendly oligonucleotide manufacturing platform that simplifies sequence design, synthesis, delivery, and bioassay screening to support your oligonucleotide drug development projects. If you are interested in our services, please feel free to contact us.