In the rapidly evolving landscape of precision medicine, oligonucleotide therapeutics are heralding a new era of targeted treatments. These groundbreaking molecules – capable of modulating gene expression with unmatched specificity – represent a paradigm shift in addressing genetic and rare diseases. From neuroscience to metabolic disorders, they offer hope for conditions once considered untreatable. 

Unlike traditional drugs, oligonucleotides target RNA, reaching the root of disease pathways. By their very design they minimize off-target effects and open up possibilities for transformative therapies such as RNA editing, protein upregulation and exon skipping. With over 20 approved drugs and many more in clinical trials, the field has shifted from high-risk newcomer to demonstrated clinical potential.

Evotec stands at the forefront of this revolution, combining scientific excellence with the ability to take an integrated, end-to-end approach to drug discovery and development. Through relentless innovation and collaboration, we have turned challenges into opportunities, empowering our customers to accelerate the journey from concept to clinic.

This eBook invites you to explore Evotec’s oligonucleotide journey, cutting-edge technologies and our path towards transformative patient care through precision medicine. Dive deeper into the oligonucleotide therapeutics revolution in an interview with the CEO of Ionis Pharmaceuticals. Learn how RNA-targeted therapeutics, including antisense oligonucleotides (ASOs) and siRNAs, are innovating the pharmaceutical industry offering enhanced delivery and potency, while reducing off-target side effects. And gain expert guidance on how to develop groundbreaking oligonucleotide therapeutics, from concept through to the clinic. Find the latest insights on aspects including in silico design, chemistry and synthesis, efficacy, safety, bioanalysis, stability and metabolic profiling, preclinical development and more.