Author: NVGCT

We would like to share the upcoming RNA Horizons 2025 Therapeutics Symposium, taking place on October 29-31, 2025 in Palermo, Sicily. This premier event is recommended to researchers with an interest in RNA therapeutics. It explores the latest advancements in the field, from established modalities to emerging areas like ASOs and RNA structure-based therapies. Discussions will encompass key trends: pipeline diversification, enhanced RNA modalities, AI integration, and improved delivery technologies. Notably, the symposium will address translating discoveries into viable therapies, exploring commercialization and market access.

Connect with scientists, industry professionals, and investors to shape the future of RNA-based therapies.

This year’s symposium will delve into the following key scientific themes:

RNA Biology and Disease: Exploring the fundamental mechanisms of RNA biology and their implications in various disease states.
RNA Therapeutics Development: Focusing on the latest advancements in the design, development, and optimization of RNA-based therapeutics.
Targeting and Delivery of RNA Therapeutics: Addressing the critical challenges of delivering RNA therapeutics to specific tissues and cell types.
Emerging RNA Technologies: Showcasing cutting-edge technologies and approaches in the field of RNA research and therapeutics.
Clinical Translation and Commercialization: Discussing the path to clinical application and the commercialization of RNA-based therapies.

FAST has collaborated with experts to create a renewed and expanded guidebook for developing Advanced Therapy Medicinal Products (ATMPs) in the Netherlands. Tailored to both academic and commercial developers, the guidebook delivers essential guidance on European and Dutch regulations and provides a roadmap to navigate the complex ATMP development process.

A guide to the ATMP landscape

The guidebook is the result of close collaboration with key partners, including the Dutch Medicines Evaluation Board (CBG), Hollandbio, DARE-NL, VIG, and input from over 25 experts. Building on the foundation of the VIG’s earlier guidebook, this updated and expanded edition integrates the latest insights, regulations, EMA processes, and advances in clinical research. With its practical tools and guidance, the handbook equips developers to handle complex procedures such as marketing authorization and reimbursement applications more effectively.

View the guidebook here

Patient-centered approach and collaboration

The guidebook underscores the value of early engagement with patient organizations and provides tools to facilitate this meaningful collaboration. It also emphasizes the importance of early engagement with stakeholders, such as the EMA, CBG, and the National Health Care Institute (ZIN). These collaborations contribute to faster patient access to ATMPs and long-term financial sustainability.

Structure of the guidebook
The guidebook is divided into three sections:

• Part 1 covers key lessons and offers guidelines for patient involvement.
• Part 2 provides background information and legal frameworks essential for ATMP development.
• Part 3 offers a detailed overview of the entire ATMP development pathway, from preclinical research to practical implementation.

Support and advice

Finally, the guidebook includes an overview of available support and relevant legislation. Developers are encouraged to seek advice from experts and centres of expertise, such as FAST and DARE-NL, early in the development process to simplify the path to market and minimize obstacles.

This guidebook is a valuable resource for everyone active in the rapidly growing ATMP sector, aligning with FAST’s mission to accelerate the delivery of innovative therapies to patients. In the spring of 2025, FAST, together with its partners, will host a series of webinars to present case studies and provide deeper insights into various sections of the guidebook.

De COGEM heeft een nieuwe signalering gepubliceerd, waarin zij verkent hoeveel nieuwe gentherapieën in de pijplijn zitten, wat de kosten zijn, en de dilemma’s en keuzes die met de komst van deze therapieën gepaard gaan. Ook wordt ingegaan op de instrumenten die de overheid heeft om gentherapieën betaalbaar en beschikbaar te houden.

Lees het document hier.

The Netherlands Commission on Genetic Modification (COGEM) commissions third-party research to support its work. The following project is open for enrollment: Germline modification and new gene therapies; an exploration. This project is a collaboration with the Central Committee on Research Involving Human Subjects (CCMO), and aims to gain insight into the likelihood of unintended germline modification in new (non-viral) gene therapies to support risk analysis. Interested parties are invited to submit a project bid. More information can be read here.

The National Institute for Public Health (RIVM) has, after close consultation with various parties including the NVGCT, written a report about the potential of DNA technology in improving public health(care). This will be used by the Ministry of Health, Welfare and Sport to develop a policy plan. More information and the report can be found here.