Frontiers Forum Deep Dive series

NANOSPRESSO: toward personalized locally produced nucleic acid nanomedicines

9 July 2025

A new point-of-care model for affordable, on-demand production of personalized nucleic acid nanomedicines could revolutionize how hospitals treat rare diseases.


Speakers

  • Prof Raymond Schiffelers headshot

    Prof Raymond Schiffelers

    University Medical Center Utrecht, the Netherlands

  • Prof Sabine Fuchs headshot

    Prof Sabine Fuchs

    University Medical Center Utrecht, the Netherlands

  • Dr Alexandre Ceccaldi headshot

    Dr Alexandre Ceccaldi

    European Technology Platform on Nanomedicine (ETPN), France

  • Olivia Lewis headshot

    Olivia Lewis

    Utrecht University, the Netherlands

  • Alexandra Heumber Perry

    Alexandra Heumber Perry

    Rare Diseases International, Switzerland

A new frontier in rare disease treatment

The NANOSPRESSO platform, published in a Frontiers in Science lead article, could enable hospital pharmacists to rapidly create inexpensive, personalized therapeutic cartridges—akin to espresso capsules—for rare disease patients at their bedside.

The article authors discuss how NANOSPRESSO could boost access to personalized nucleic acid therapies, even in low-resource settings, and how to overcome regulatory challenges.

Hear our expert panel explore how NANOSPRESSO could open up treatments for underfunded and underserved rare conditions worldwide—which affect 36 million patients in the EU alone.

Agenda


Why rare diseases demand a new model: the case for decentralizing personalized treatment production


Overcoming pharmaceutical and regulatory barriers for nucleic acid therapeutics


Ensuring quality, affordability, and equity in local production: practical, regulatory, and ethical considerations. 


RNA and LNP-based therapeutics: from pandemic response to personalized rare disease care 


NANOSPRESSO: a scalable platform for bedside manufacturing of nucleic acid nanomedicines 


Charting a path forward | Panel discussion and Q&A

Speaker and contributor bios

  • Raymond Schiffelers

    Professor of Nanomedicine, CDL Research, University Medical Center Utrecht, the Netherlands

    Prof Raymond Schiffelers is a leading expert in nanomedicine and targeted drug delivery. His pioneering research focuses on the development of nanoparticle-based systems for precision drug transport, with particular emphasis on targeting tumor vasculature, developing polymers for siRNA delivery, and utilizing extracellular vesicles as novel templates for drug delivery and diagnostics.

    A key figure in several European consortia, he is also a founding member of the International Society for Extracellular Vesicles (ISEV) and the immediate past chair of the European Technology Platform on Nanomedicine. Raymond serves as Associate Editor of the Journal of Extracellular Vesicles and is an editor for both the International Journal of Pharmaceutics and the Journal of Controlled Release.

    He continues to shape policy and regulatory frameworks to accelerate the safe and effective integration of nanotechnologies into modern healthcare. Recently, Raymond received the prestigious Advanced Grant from the European Research Council (ERC) for his research on smart nanoparticles for cancer therapy.

  • Sabine Fuchs 

    Professor of Metabolic Diseases and Innovative Therapies, University Medical Center Utrecht, the Netherlands

    Prof Sabine Fuchs employs innovative strategies—such as liver organoids and precise gene editing—to develop treatments for the significant unmet clinical needs of patients with metabolic diseases. Her dedication has led to the development of an improved liver organoid model and to the founding of award-winning company, HeLLO R&D. 

    Sabine combines her clinical expertise as a pediatrician with her leadership of a research group at the Regenerative Medicine Center Utrecht, part of the Hubrecht Institute. Having established a large metabolic biobank containing over 300 patient-derived organoid lines, her team was the first to demonstrate that prime editing can restore disease phenotypes—both genetically and functionally—in patient-derived intestinal and liver organoids without causing unwanted genetic effects. Building on this work, Sabine has received prestigious grants, including the ERC Starting Grant and the Elisabeth von Freyburg Penning.  

    Sabine is dedicated to advancing patient care and mentoring clinician-scientists through her leadership in education and talent programs such as TULIPS (Training Upcoming Leaders in Pediatric Science) and the EUREKA Certificate Program for Translational Medicine.  

    A firm believer in “doing the right research right,” she is a member of the University Medical Center Utrecht’s Open Science Team.   

  • Alexandre Ceccaldi

    General Secretary, European Technology Platform on Nanomedicine (ETPN), France

    Dr Alexandre Ceccaldi leads the European Technology Platform on Nanomedicine (ETPN), the driving force behind Europe’s nanomedicine agenda—a network of over 130 academic, industrial and healthcare organizations. He is also coordinator of the NOBEL European Project—spanning diagnostics, targeted drug delivery, regenerative medicine, and digital health convergence.  

    Alexandre earned a PhD in Biology from Université Pierre et Marie Curie and a degree in engineering from AgroParisTech, grounding his career in cutting‑edge cancer research and stem‑cell therapeutics. Previously, he managed INGESTEM, France’s first national public consortium dedicated to induced pluripotent stem cells and their medical applications. 

    Alexandre’s passion for innovation in healthcare is driven by the desire to change the life of millions of patients every year. He is committed to uniting Europe’s nanomedicine community to accelerate clinical translation: driving collaborative frameworks and propelling innovative technologies that improve patient outcomes on a continental scale. 

  • Olivia Lewis

    PhD student, Utrecht University, the Netherlands

    Olivia Lewis (MSc) is a PhD student specializing in drug regulatory science at Utrecht University. As part of the NANOSPRESSO-NL project, Olivia’s role and the focus of her doctoral research is to investigate regulatory pathways and challenges related to the localized preparation of high-quality, personalized nucleic acid nanomedicines.  

    With two Master's degrees, one in molecular bioscience and one in global health, Olivia is motivated by complex transdisciplinary issues that bridge scientific innovation and society, aiming to enhance public health through smarter, more responsive drug regulation. She is committed to supporting the development of innovative understandings, perspectives, and solutions to these complex challenges. 

  • Alexandra Heumber Perry

    Alexandra Heumber Perry, CEO, Rare Diseases International, Switzerland

    Alexandra Heumber Perry is an accomplished global health leader who has dedicated her entire career to improving healthcare policies for people living with diseases, with a particular focus on neglected and vulnerable populations. 

    With over two decades of international experience in policy, patient advocacy, and public affairs, Alexandra has advised major organizations such as The Lancet Commission and the World Obesity Federation. She established the International Alliance for Myalgic Encephalomyelitis, significantly raising the disease’s profile at the World Health Organization (WHO) and among policymakers. She served as Head of Policy Affairs at the Drugs for Neglected Diseases initiative (DNDi), achieving impactful policy shifts for neglected diseases, and held earlier roles with Médecins Sans Frontières and Health Canada. 

    In her current role as CEO of Rare Diseases International, Alexandra drives global strategies to raise awareness of rare diseases, improve access to diagnostics, treatments and care, advocate for the rights of patients worldwide, to improve the lives of people living with rare diseases.