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We are delighted to share with you the almost final agenda of our event “Building a Research and Innovation Ecosystem for Rare Diseases,” taking place on 29 April 2025, from 14:30 to 16:30, at the European Parliament in Brussels, Room P4B001.

This important event is organized by the Brains for Brain Foundation and will be kindly hosted by MEP Vytenis Andriukaitis, who will co-chair alongside Prof. Maurizio Scarpa, President and Founder of the Brains for Brain Foundation and coordinator of the European Reference Network for Metabolic Disorders (MetabERN).

Join us for a multi-stakeholder dialogue aimed at strategically positioning research and innovation within EU life science and health policy, using rare diseases as a model to highlight the EU’s added value. Key discussions will focus on:

• Leveraging existing EU research infrastructure
• Fostering an innovation ecosystem
• Shaping effective policies to accelerate progress in rare disease research and treatment

Confirmed Speakers:

• Olivér Várhelyi, European Commissioner for Health and Animal Welfare
• Donata Meroni, Head of Unit, Health Monitoring and Cooperation, DG SANTE
• Arjon van Hengel, Deputy Head of Unit, Health Innovations and Ecosystems Unit, DG RESEARCH
• Szymon Bielecki, Head of Sector, Research and Innovation, eHealth, Well-Being and Ageing Unit, DG CONNECT
• Peter Arlett, Head of Data Analytics and Methods, European Medicines Agency (EMA)
• Loizos Solomou, Research, Innovation and Space Policy Attaché, Permanent Representation of Cyprus to the EU
• Alexander Natz, Secretary General, European Confederation of Pharmaceutical Entrepreneurs (EUCOPE)
• Luca Sangiorgi, Coordinator of ERN on Rare Bone Disorders (ERN BOND)
• Ruth Ladenstein, Coordinator, European Reference Network for Paediatric Oncology (ERN PaedCan)
• Leire Solis, Senior Manager, IPOPI (International Patient Organisation for Primary Immunodeficiencies)

Event Endorsements

The event is proudly endorsed by:

• European Business Summit
• EUCOPE
• International MPS and Related Diseases Network
• IPOPI
• Screen4Rare
• Ultragenyx
• Sanofi
• Chiesi
• Denali Therapeutics

REGISTRATION

If interested, please register through  this form by 24 April.

All registrations will be reviewed given the limited room capacity. Confirmed participants will be informed by email.

We hope you can join us in Brussels or participate online

For any questions or registration issues, please contact Davide Patteri at Burson: davide.patteri@bursonglobal.com.

We look forward to seeing you there!

Streaming Page

https://app.livecasts.eu/building-a-research-and-innovation-ecosystem-for-rare-diseases

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The fight against rare diseases requires a unified and strategic approach, leveraging the collective expertise of researchers, policymakers, industry leaders, and civil society. By fostering collaboration and innovation, we can build a sustainable ecosystem that ensures better diagnosis, treatment, and care for millions of individuals. Together, we can drive forward a European Health Union that truly delivers for those affected by rare diseases.

 The Brains4Brain Foundation is delighted to announce the launch of an ambitious initiative aimed at addressing one of the most critical challenges in healthcare today: the establishment of a concrete ecosystem for rare diseases. To this aim, we are organising a preparatory event to present our project to selected policymakers -other MEPs but also representatives from the European Commission and the Member States – and a wider range of stakeholders within the rare disease ecosystem.

This hearing, titled “Building a Research and Innovation Ecosystem for Rare Diseases”, was initially scheduled on Tuesday, 25 March but due to requests from several stakeholders who expressed a strong interest in participating but had prior commitments, we have decided to postpone the meeting to Tuesday, 29 April, from 14:30 to 16:30 in the European Parliament, Room P4B001 – Bruxelles.

A Collaborative Effort

This landmark event is convened by Vytenis Andriukaitis, Member of the European Parliament (MEP), in partnership with the Brains4Brain Foundation and a diverse coalition of stakeholders. It forms part of the public consultation launched by the European Parliament’s Committee on Public Health (SANT) and aligns with the EU4H-2024-PJ-03 Call under the EU4H Action Grants 2024.

Focus of the Hearing

The hearing is designed to inform future political and policy activities in the rare disease space and will explore the piloting of the Research and Innovation Union concept, as outlined in Draghi’s Report, specifically tailored to meet the needs of individuals living with rare diseases. The objective is to establish a robust ecosystem, anchored in European Reference Networks, fostering collaboration between institutional, public, private, and civil society stakeholders.

Key Objectives

• Promoting Scientific Excellence – Supporting fundamental research and attracting world-class talent in science and innovation.
• Enhancing Industrial Competitiveness – Strengthening partnerships between academia and industry to advance crucial technologies and strategic sectors.
• Implementing a Mission-Oriented Approach – Developing targeted solutions to improve the quality of life for the 36 million Europeans living with rare diseases (PLRD).

The Need for Collaboration

The challenges faced by healthcare providers and patients affected by rare diseases are multifaceted, and fragmented approaches have proven inadequate. There is a pressing need to align financial and non-financial incentives to secure sustainable, long-term investment in rare disease research and development.

This hearing provides a platform for key stakeholders to engage in meaningful dialogue, shaping future policy and political action in the rare disease sector.

The fight against rare diseases requires a unified and strategic approach, leveraging the collective expertise of researchers, policymakers, industry leaders, and civil society. By fostering collaboration and innovation, we can build a sustainable ecosystem that ensures better diagnosis, treatment, and care for millions of individuals. Together, we can drive forward a European Health Union that truly delivers for those affected by rare diseases.

REGISTRATION

If interested, please register through this form. All registrations will be reviewed given the limited room capacity. Confirmed participants will be informed by email.

The Brains for Brain Foundation is pleased to announce the upcoming 2nd “Drug Delivery to the Brain” Keystone Symposium, scheduled for February 17–20, 2025, at the Keystone Resort in Colorado. This conference will be held jointly with the “Antibodies as Drugs” meeting and aims to bring together researchers and professionals from both academia and industry to discuss advancements in drug delivery systems for the central nervous system (CNS).

Following the success of the inaugural symposium in January 2023, which attracted over 200 attendees and featured two keynote talks, 18 plenary sessions, and 12 short talks selected from nearly 70 poster abstracts, this upcoming event promises to be equally impactful. The previous meeting notably achieved a balanced representation of participants from academic and private sectors, fostering collaborative discussions.

The 2025 symposium will be organized by a team of esteemed researchers, including James Gorman from Harvard’s Wyss Institute and Stina Syvänen from Uppsala University, alongside Robert G. Thorne, who will deliver the opening keynote address. Thorne’s talk, titled “Delivery of Therapeutics to the CNS via Different Routes of Administration: An Overview of Applications, Advantages and Disadvantages,” will explore the current state of the field and emerging approaches, highlighting a critical time for CNS delivery research.

With new approvals and advancements in brain-targeted therapies for conditions such as Alzheimer’s disease, multiple sclerosis, and brain cancer on the horizon, this symposium represents an essential gathering for those involved in CNS drug delivery.

Key dates to note include:

• Scholarship and Abstract Submission Deadline: November 26, 2024
• Early Registration Deadline: December 23, 2024
• Poster Abstract Deadline: January 27, 2025

The symposium will also feature multiple awards for junior presenters, sponsored in part by the International Brain Barriers Society, which will honor the scientific legacies of Joan Abbott and Danica Stanimirovic.

The Brains for Brain Foundation encourages all interested parties to consider attending this unique meeting. Collaboration and knowledge sharing are vital for advancing the field, and we look forward to welcoming both returning and new participants.

For further information on registration, abstract submissions, and conference details, please click here

P.S. This is definitely a Keystone Symposium where the mountain will feature with some great skiing for those who might be interested to combine the meeting with winter recreation: