BOOST · Backbone-Optimized OligonucleotideS for Therapeutics

Backbone-Optimized OligonucleotideS for Therapeutics

Therapeutic oligonucleotides are a promising class of medicines that can regulate disease related genes and offer new treatment options for many genetic and rare disorders. Their wider use is limited because they require chemical modifications to stay stable and active in the body. These modifications are technically demanding and slow to produce, increasing development time and manufacturing costs. The BOOST project set out to overcome this challenge by creating a new generation of chemically modified oligonucleotides with improved biological performance. Its goal was to develop innovative backbone structures suitable for antisense oligonucleotides and siRNA, two key technologies in modern genetic medicine. By enabling faster and more efficient chemical modification, BOOST aimed to support the development of more accessible and affordable RNA based therapies. The project designed new chemical reagents and applied them during solid phase oligonucleotide synthesis, the standard method used to produce therapeutic oligonucleotides. It then evaluated how the new backbone structures behave in biological environments and explored how additional functional groups, such as targeting molecules or imaging dye, can be attached to create more precise and versatile therapeutic tools. BOOST successfully demonstrated that these new backbone designs can streamline chemical modification and expand the functional possibilities of therapeutic oligonucleotides. This provides a foundation for developing next generation RNA medicines with improved stability, reduced production costs, and enhanced targeting capabilities. The project is expected to contribute to EU priorities in health innovation, competitiveness, and sustainable production. By reducing chemical waste and energy use during synthesis, BOOST also supports greener manufacturing practices. Overall, the project advances the long term goal of making genetic medicines more efficient, affordable, and widely accessible.

Data: CORDIS, © European Union