EFFecT

📢 New publication from the EFFecT network! We’re excited to share one of the first scientific outputs from the EFFecT network: a commentary published in Molecular Therapy Nucleic Acids by Gijs-Jan Scholten, and the EFFecT network’s own Jamie Hyde (DC7) and Ronald Buijsen. The article discusses new research by Kentaro Maeda and colleagues describing a chemically modified siRNA strategy targeting expanded CAG repeats, a promising RNA-based therapeutic strategy for polyglutamine disorders, such as Huntington’s disease and several spinocerebellar ataxias. The commentary highlights how serinol nucleic acid (SNA) modifications may improve the stability, distribution, and selectivity of RNA therapeutics in the central nervous system, while also discussing key challenges like target specificity and long-term safety. Research like this aligns with the EFFecT network’s mission to advance the development of antisense and RNA-based therapeutics and to train the next generation of scientists in this rapidly evolving field. Commentary link: https://lnkd.in/ddeZXUem #EFFecT #MSCA #DoctoralNetworks #RNAtherapeutics #HorizonEurope

This week, the EFFecT Network PhD students gathered in Trento, Italy for our EFFecT Symposium: Dissemination, Communication & Open Science, a training school designed to strengthen the skills of our 12 doctoral researchers beyond the lab. Over several days, the network combined scientific exchange with professional development, focusing on how we communicate, manage, and maximize the impact of our research on antisense oligonucleotide (ASO) therapeutics. The symposium included three key training sessions: 🔬 Adaptive Communication & Dissemination – Led by Michela Catenacci , this workshop focused on making complex scientific research accessible and impactful across different audiences, from the scientific community to the public. 📊 Data Management, Open Data & FAIR Principles – Organized by Elvina Sakellariou and Sergiu Siminiuc from the Duchenne Data Foundation , this session explored best practices for responsible data stewardship and ensuring research data is Findable, Accessible, Interoperable and Reusable (FAIR). 🌍 Understanding Research Impact – Hosted at the Faculty of Sociology, University of Trento, this course examined how scientific research translates into societal and policy impact. The symposium also included a half-day research meeting where our PhD candidates presented updates on their projects, highlighting the progress being made across the EFFecT network. A special thank you to Michela A. Denti for organizing such a stimulating and collaborative event. The week began with a hike in the beautiful surroundings of Trento, a great opportunity for the network to connect outside the lecture room. Events like this are central to the EFFecT Doctoral Network, helping train the next generation of scientists working to advance RNA-based therapeutics. #MSCA #DoctoralNetworks #OpenScience #ScienceCommunication #ASO #HorizonEurope #EFFecT

Yesterday we explored the FAIR data principles and lifecycle, by stepping into the role of data curators, working through how datasets evolve from raw data to well-structured, enriched resources. The hands-on session led by Elvina Sakellariou and Sergiu Siminiuc from Duchenne Data Foundation focused on adding meaningful metadata and mapping datasets to standardized ontologies to improve discoverability, interoperability, and reuse. A key takeaway: FAIRness should be built into the data pipeline from the very beginning.

The EFFecT DCs attended a hands-on training session led by Elvina Sakellariou and Sergiu Siminiuc from the Duchenne Data Foundation on Data Management, Open Data, and FAIR principles at the School of Innovation, in Trento. EFFecT is committed to making rare disease data reusable and interoperable.

Today is Rare Disease Day 💜🧬 Over 300 million people worldwide live with a rare disease, and for many, a diagnosis is only the beginning of a long search for treatment. At EFFecT, we are committed to changing this reality. By advancing the development of antisense oligonucleotides (ASOs), we work to design targeted therapies that address diseases at their genetic root, offering hope where few options exist. Innovation is only possible through collaboration, patient engagement, and persistence. On this day, we stand with the rare disease community, amplify patient voices, and reaffirm our mission to translate cutting-edge science into real impact. Together, we move toward a future where rare diseases are better understood, diagnosed, and treated 🔬💜 #RareDiseaseDay #ASO #Antisense #RareDiseaseResearch #GeneticMedicine #EFFecT

Today, we celebrate the International Day of Women and Girls in Science, a day to recognize the brilliance, resilience, and impact of women who are shaping the future through research and innovation, pushing boundaries, and inspiring the next generation of scientists. At EFFecT, we are incredibly proud of our outstanding PIs and DCs. Their dedication to advancing science drives not only our project forward, but also the broader scientific community toward meaningful progress. Together, they represent the collaborative spirit and excellence of EFFecT. Thank you for your leadership, your perseverance, and your passion for discovery. We are proud to work alongside you! #WomenInScience #InternationalDayOfWomenAndGirlsInScience #RNAResearch #Oligonucleotides #WomenInSTEM

Congratulations to Dr. Alejandro (Alex) Garanto, Principal Investigator and Coordinator of EFFecT, and Dr. Aurelie Goyenvalle, Principal Investigator and Chair of the EFFecT CDC Committee, on their new roles as Secretary and Director, respectively, of the Oligonucleotide Therapeutics Society Board of Directors for the 2026–2027 term.

We are happy to welcome our final DC, Maria Querol Canut! Maria joined us on November 1st as DC9 at ISIT, under the supervision of Dr. Hélène Tran. Her research will focus on understanding how sequences and/or modified chemistries can impact their potency, uptake, biodistribution, safety and elimination half-life with a particular focus on the cerebellum. Beyond the lab, she loves reading, going to the theatre, concerts, and musicals, discovering new TV series, and spending time on creative crafts. Welcome to the team, Maria — and congratulations to all as we officially have all our doctoral candidates on board!

Earlier this week, our consortium came together for the kick-off meeting of EFFecT, followed by a training school for the doctoral candidates. EFFecT brings together leading institutions and industry partners to train the next generation of scientists. The program aims to provide 12 predoctoral researchers with the skills and knowledge required to advance antisense nucleic acid therapeutics. A big thank you to all our doctoral candidates, beneficiaries, associated partners and external advisory board, for their contributions and enthusiasm during the meeting – this marks the beginning of an impactful journey ahead!