HEИндивидуална стипендия2024–2026

BOOST · Backbone-Optimized OligonucleotideS for Therapeutics

„Хоризонт Европа“ — Действия „Мария Склодовска-Кюри“

Период: 2024-04-01 → 2026-03-31
Финансиране от ЕС: 214 934 €
Участници: 1
Схема: HORIZON-TMA-MSCA-PF-EF

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Накратко на български

Терапевтичните олигонуклеотиди, като siRNA, се модифицират химически, за да станат по-стабилни и ефективни в тялото. Това помага за създаването на по-достъпни лекарства за генетични и редки заболявания чрез намаляване на разходите и времето за производство.

Този кратък обзор е генериран от изкуствен интелект

Кратко обяснение, генерирано от езиков модел по текста на CORDIS. Оригиналът е по-долу.

Резултати накратко

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.

Текст от CORDIS, на английски · Данни: CORDIS, © Европейски съюз

Цел на проекта

Therapeutic oligonucleotides (ONs) have recently gained increasing importance as new drugs. While conventional drugs are limited to targeting proteins, oligonucleotides are able to target diseases upstream at the mRNA level. This specificity in combination with their easy design renders them highly valuable. Furthermore, their applications are limitless, involving treatment of so-called undruggable diseases. The bottleneck in the development of new therapeutic oligonucleotides lies in the efficient synthesis of modified ONs. Often, these modifications require multi-step syntheses and are laborious resulting in a time-consuming process of drug development. Additionally, these modifications often negatively impact physiological and biological properties. In our proposal, “Backbone-Optimized OligonucleotideS for Therapeutics” (BOOST), we outline a methodology for efficiently modifying oligonucleotides. Key to success will be the development of novel reagents capable of intercepting solid-phase oligonucleotide synthesis due to their high reaction rate. This will enable rapid evaluation of mono- and multiple-modified oligonucleotides to assess their physiological and biological properties. Furthermore, the introduction of multiple functionalities will become possible. BOOST will serve as a new platform for designing therapeutic antisense oligonucleotides (ASOs) and siRNA. As a proof of concept, we will evaluate the most promising modified oligonucleotides in biological settings.

Оригинален текст от CORDIS (на английски).

Участници

AARHUS UNIVERSITET · Aarhus CКоординаторДания