Needs

Overview for Non-Experts

NANOSPRESSO is a groundbreaking project that brings personalised RNA treatments directly to hospitals. This approach is crucial for patients with rare diseases, such as Duchenne muscular dystrophy, cystic fibrosis, methylmalonic acidemia, and Crigler-Najjar syndrome, who currently have few treatment options. By allowing hospitals to create RNA nanomedicines on-site, NANOSPRESSO enables patients to receive tailored treatments faster and more affordably, while maintaining strict safety standards.

In a nutshell

The Urgent Need for Personalized RNA-Based Nanomedicines

Personalised RNA-based nanomedicines offer transformative potential for treating rare diseases like Duchenne muscular dystrophy, spinal muscular atrophy (SMA), and metabolic disorders such as methylmalonic acidemia, or the Crigler-Najjar syndrome. These diseases often have no to limited therapies, leaving many patients without viable alternatives.

A major challenge in RNA therapies is their delivery, as RNA molecules are unstable and must reach target cells intact. Nanomedicine, particularly lipid nanoparticles (LNPs), is revolutionizing this process, enabling stable and efficient delivery of RNA directly into cells, opening new doors for therapeutic interventions.

Critical Needs Addressed by NANOSPRESSO

Need for RNA-Based Therapies for Specific Diseases

RNA therapies, such as mRNA vaccines, have demonstrated their power in the fight against COVID-19. However, many rare diseases, including cystic fibrosis, hereditary cancers, methylmalonic acidemia, and Crigler-Najjar syndrome, still lack effective treatments. By modifying mRNA sequences, RNA technologies can be adapted to address different enzyme deficiencies, maximising their potential impact.

Adapting RNA Platforms for Multiple Rare Diseases

RNA therapies offer significant adaptability. Using the same lipid nanoparticle delivery platform, different RNA sequences can be employed to target various genetic disorders, reducing development time and cost. This approach makes personalised nanomedicine a feasible solution for many rare diseases.

Overcoming the Limitations of Centralised Production

The centralised nature of pharmaceutical production leads to high costs, long production timelines, and limited access, particularly for patients with rare diseases. Nanospresso addresses this issue by enabling RNA nanomedicine production directly in hospitals, providing faster, personalised treatments and reducing costs and waiting times.

Expected impact

Innovating Microfluidic Technology for Local Production

RNA molecules are notoriously fragile, which poses challenges for consistent delivery. Nanospresso employs advanced microfluidic platforms to produce RNA nanomedicines at the point of care. This ensures RNA stability, potency, and safety, making personalised treatments more reliable and accessible.

Expanding Access to Personalised RNA Therapies

Rare disease patients, especially those in underserved regions, often face significant barriers to accessing advanced treatments. By making RNA nanomedicines more economically and geographically accessible, Nanospresso aims to democratise cutting-edge therapies, ensuring all patients, regardless of location, can benefit.

Who we are

Addressing Regulatory Challenges

To ensure RNA nanomedicines reach patients safely, they must meet strict regulatory standards. Nanospresso studies the alignment of current regulatory systems with scientific needs, paving the way for innovative treatments.

Perspectives from Industry Experts and Stakeholders

For rare diseases, commercial therapies are not always in the best interest of patients and or society. Nanospresso merges platform technologies to allow local hospitals to make high quality nucleic acid nanomedicines at the bedside.

Prof. Raymond Schiffelers, at UMC Utrecht

Lipid nanoparticles are essential for delivering RNA therapies. NANOSPRESSO’s approach could redefine how we make these therapies accessible.

Peter Hölig , Lipoid GmbH

For patients with rare diseases, having access to personalised RNA treatments is life-changing. NANOSPRESSO’s effort to bring these therapies closer to patients is a vital step forward.

Patient Advocate

Why NANOSPRESSO is Unique and Promising

Patient-Centric Innovation

NANOSPRESSO is designed around the needs of patients with rare diseases, delivering RNA therapies efficiently and locally, reducing costs and improving accessibility.

Advanced Microfluidic Systems

By using microfluidic technology, NANOSPRESSO ensures that RNA nanomedicines are produced rapidly and safely, directly in hospital pharmacies.

Collaborative Expertise

NANOSPRESSO brings together leading experts in RNA biology, LNP chemistry, and regulatory engagement to establish a safe, decentralised model for personalised treatment.

Expanding Access

The project aims to democratise healthcare by making RNA therapies accessible to those with rare diseases, ultimately improving patient care.

Shaping the Future of Personalized RNA Nanomedicine Together

NANOSPRESSO is at the forefront of creating a future where personalised RNA-based treatments are available to all patients in need. We invite you to join us in revolutionising healthcare and ensuring advanced therapies are a reality for rare and orphan diseases.

News and Events

LOCAL PREPARATION OF HIGH-QUALITY, PERSONALISED NUCLEIC ACID NANOMEDICINES

NANOSPRESSO is funded by the Dutch Research Agenda (NWA) ORC 2020/21 program. For more information about our partners, funding, and legal notices, please visit our contact page and follow us on social media.