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HEДокторантска мрежа2023–2027

MMM · Medicine Made to Measure.

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

Период
2023-10-01 → 2027-09-30
Финансиране от ЕС
2 198 700 €
Участници
23
Схема
HORIZON-TMA-MSCA-DN

Линиите свързват координатора с партньорите.

Накратко на български

Синтетичните РНК молекули (ASO) се изследват като средство за персонализирано лечение на хора с изключително редки неврологични заболявания. Това е важно, защото в момента 95% от тези пациенти нямат подходяща терапия поради специфичността на техните генетични мутации.

Този кратък обзор е генериран от изкуствен интелект

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

Резултати накратко

Medicine Made to Measure.

MMM is an intersectoral, innovative training network that receives funding from the European Commission (2023-2027) as part of the HE MSCA programme. It consists of nine beneficiaries, hosting 10 doctoral students, plus 14 associated partners providing training, PhD enrolment or secondment opportunities. MMM stands for “Medicine Made to Measure” and furthers all pathways in the pipeline towards single-patient treatment of nano-rare neurological diseases. Our network brings together international academic, industrial, governmental and patient organisation partners in a cross-specialty platform. Although the last decade has seen a breakthrough in the field of personalised genetic medicine, currently 95% of rare neurological diseases (RNDs) still lack disease modifying treatment options. This is due to the rarity of individual RNDs, their sheer number, and the lack of appropriate translational and trial methodology for nano-rare diseases. Antisense-oligonucleotides (ASOs) are short, chemically modified, synthetic RNAs which, upon intrathecal delivery, distribute throughout the central nervous system (CNS), making them ideally suited for treating CNS diseases. ASOs offer particular promise for precision medicine due to their comparatively low production cost, a “programmable” sequence that can be flexibly targeted to almost any gene transcript target of choice, relatively low toxicity and flexible dosing regimens. The ultimate goal of MMM is to establish the field of tailored ASO treatments for individuals with nano-rare RD mutations. We seek to develop core modules of a novel “out-of-the-box”, yet EMA-advised treatment development path for single patients with nano-rare mutations. At the same time these core modules offer a unique opportunity for doctoral candidates to be trained and involved in developing all aspects needed for tailored ASO treatment, eventually becoming the forerunners of developing tailored genetic treatments and streamlining tailored ASO treatment for RNDs in Europe, both in academia and industry.

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

Цел на проекта

Modern medicine seeks to personalize diagnosis and treatment to the needs of individual patients. This need is heightened in particular for those individual patients where no treatments are available – and where current standard treatment development paradigms exclude to generate such. The ultimate goal of Medicine made to Measure (MMM) is to go even beyond the latest genetic medicine approaches, implementing a novel paradigm of treatment development: the field of single patient tailored antisense oligonucleotide (ASO) treatments for patients with nano-rare disease mutations. Specifically, MMM will develop core modules of a novel “out-of-the-box”, but still EMA-advised treatment development path for these patients - from omics-based theranostics, via preclinical target validation to innovative trial methodology and a pioneering ethical framework for individualised genetic therapies. MMM provides a unique opportunity for doctoral candidates to be involved in building - and to receive training - in all core modules needed for tailored ASO treatment in Europe. This new scientific field requires a new generation of open-minded, technology-fluent and applications-oriented experts. MMM will help educate future drug development and translational experts in adopting and disseminating such a cross-specialty approach. Our idea for this innovative training network is built on our personal experience and understanding of the importance of single patient tailored genetic treatments in the years to come. We propose a consortium of world-leading experts and partners that present with extended experience and relevant know-how in molecular biology, neurology, translational medicine, biochemistry, bioinformatics, philosophy, mathematics, regulatory science and engineering. Thus, MMM provides an exceptional platform to young, ambitious and talented researchers who like to engage in supporting the concept of single patient tailored treatments.

Оригинален текст от CORDIS (на английски).

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Данни: CORDIS, © Европейски съюз