Why is this training course important?
Many opportunities still exist to develop the next wave of novel, extra-hepatic oligonucleotide therapeutic conjugates for patients, with optimum delivery, safety, and efficacy profiles. Advanced understanding of this drug modality has led to greater successes in identifying novel ligands that address target organs and cells beyond the liver, including kidney, heart and lung and others. In addition, these novel ligands require creative and unique chemistry strategies, beyond established methods, to link them to specific classes of oligonucleotides in order to create the optimal new oligonucleotide drug conjugate.
With the appropriate strategy in place, oligonucleotide conjugates can be developed which have a higher chance to provide:
- Improved pharmacokinetics of the oligonucleotide
- Improved biodistribution of the oligonucleotide
- Improved (productive) uptake of the oligonucleotide
The goal of this approach is to yield an efficacious oligonucleotide therapeutic which does not impair the biological behaviour of either the ligand or the oligonucleotide, and which retains the desired chemical structure and safety properties.
In addition to covering the topics listed above, this training course will help you to choose and apply the correct strategy for your oligonucleotide conjugate program, to give you a greater chance of success in generating oligonucleotide conjugates with the desired properties.
This course will focus on the exciting developments in the field of targeted oligonucleotide conjugates. We discuss why oligonucleotide conjugates are in demand and when they are specifically needed. We discuss standard ligands (GalNac) as well more recently pursued ligand opportunities (lipid, peptide, antibody, small molecule) to target the oligonucleotide of choice (ASO, SSO, siRNA) to the tissue of interest.
This course discusses how to select the right chemistry strategy to conjugate your selected ligand to the correct position on the oligonucleotide of choice, which is the key to maximizing its biological potential. The course will also discuss what to do once you select your conjugation strategy including which “toolbox” items you will need to put the correct linker in place (which allows the conjugate to remain intact). And which tools you will need to facilitate where and when the linker should disengage.
What Will You Learn?
- Why there is an ever-increasing interest in targeted oligonucleotide delivery
- How to address specific disease areas with targeted (ligand) oligonucleotide conjugates
- How to maximize the biological potential of your targeted oligonucleotide conjugate by strategic design of your conjugate
- To obtain a full overview of all the toolbox opportunities to generate targeted oligonucleotide conjugates
- Different routes of synthesis of targeted oligonucleotide conjugates