Design of therapeutic oligonucleotides

Therapeutic oligonucleotides represent a breakthrough technology in genetic medicine, specifically addressing diseases at the molecular level. In this third instalment of our #DiscoveringOligonucleotides series, we dive into the development process, from target finding to custom oligonucleotide design.

Search and selection of new targets: innovative strategies

In the search for more effective treatments, systems biology stands as a beacon of light in the exploration of complex networks of genetic and protein interactions to identify key points of intervention. RNAseq is a powerful tool that analyses gene expression and, together with artificial intelligence (AI), composes a symphony of data capable of revealing hidden patterns.

Thus, with the support of the literature search, our knowledge is expanded, allowing a deeper understanding of the biological pathways involved. Candidate genes are prioritised with precision, focusing our efforts on the most promising targets.

Oligonucleotide design and modification: the art of customisation

Once targets have been identified, the focus is on the design and modification of therapeutic oligonucleotides. Artificial intelligence, such as that used by our partner Sylentis in its proprietary sirFinder software, plays a crucial role. Advanced algorithms analyse vast data sets to predict the efficacy of the designed oligonucleotides. Tools like these allow us not only to speed up the process, but also to improve accuracy, taking gene therapy to new heights.

When designing new therapeutic oligonucleotides, in addition to the targeting approach, the following must be taken into account:

• The generation of multiple candidates, which allows you to explore different strategies and optimise selection.
• Flexibility, as each patient and disease requires unique approaches.
• The implementation of base modifications that seek to enhance efficacy and release. This personalised approach is essential to address genetic variability between individuals and maximise therapeutic benefits.

As we move forward in the development of therapeutic oligonucleotides, we are witnessing an exciting convergence of science and technology. The combination of systems biology strategies, artificial intelligence and personalised design are transforming the landscape of personalised medicine. Each step brings us closer to more effective, less invasive treatments tailored to the uniqueness of each patient.

By harnessing the power of these technologies and the collaborative approach, at OLIGOFASTX we optimise the efficiency of the process. Our leitmotif is the acceleration of research through co-development, enabling us to move towards the common goal of delivering oligonucleotide-based therapies for rare diseases. This approach is setting a unique milestone in the field of oligonucleotides in Spain and worldwide in the search for innovative and personalised medical solutions.

Sources:

Gene therapy gets off the ground – Aceprensa

What is Gene Therapy? | FDA

Introduction to RNA-seq

Artificial intelligence for drug discovery: Resources, methods, and applications – ScienceDirect

Sylentis

Header image: https://www.aceprensa.com/ciencia/medicina/la-terapia-genica-echa-andar/