Join the 8th CCP Phenogenomics Conference on Rare Diseases, Experimental Models and Therapies
The Czech Centre for Phenogenomics (CCP) invites researchers, clinicians, infrastructure partners, patient organisations and industry representatives to the 8th CCP Phenogenomics Conference, taking place 9–11 September 2026 in Prague and online.
Celebrating the 10th anniversary of the Czech Centre for Phenogenomics, this special edition will bring together internationally recognised experts to explore how advances in rare disease research, experimental disease models (in vivo and in vitro), gene and cell therapies, and disease informatics are converging to accelerate the development of new treatments. The programme follows the full path from gene function and disease mechanisms to preclinical intervention and bottom‑up efforts to develop gene therapies in collaboration with patient communities.
Over three days, participants will explore:
- Models to understand gene function in vivo
- Advanced in vitro and preclinical models
- Disease informatics and variant interpretation
- Experimental models for therapy development
- Rare diseases and bottom‑up gene therapy initiatives
- Technology talks and selected poster presentations
- Patient & Research Session connecting patients, foundations and researchers
A special INFRAFRONTIER & EATRIS distinguished session will highlight how state‑of‑the‑art in vivo and in vitro platforms can amplify translational research when combined across research infrastructures and countries.
The 2026 programme features Keynote & Featured lectures by:
- Tim Bertram (NSF Regenerative Medicine Engine, USA), on convergent cell and gene therapy strategies and translating scientific discovery into commercial outcomes.
- Stanley T. Crooke (n‑Lorem Foundation & Ionis Pharmaceuticals, USA), on responding to nano‑rare diseases and on developing philanthropic antisense therapies for patients with ultra-rare genetic variants.
- Kiran Musunuru (Perelman School of Medicine at the University of Pennsylvania, USA), on developing and deploying personalised gene‑editing therapies for individual patients.
In addition, invited speakers will cover topics including precision organoid models, mitochondrial disease, hearing and vision disorders, neuromuscular and neurodegenerative diseases, RNA‑based mechanisms, and gene therapies for rare diseases. Confirmed speakers include José A. Sánchez Alcázar, Hans Tómas Björnsson, Mariana Branco, Marcela Buchtová, Anna Cereseto, Heon Yung Gee, Manoe J. Janssen, Mariah Lelos, Kotaro Nakanishi, Carlo Rivolta, Markus A. Rüegg, Francisco J. Sánchez‑Rivera, Jaewhan Song, David Staněk, Barbara Vona and many others.
A distinctive feature of the CCP conference is the Patient & Research Session, which brings together patient foundations and researchers in joint blocks dedicated to SPATA5/Spata5L1 disorders, Angelman syndrome and Prader-Willi syndrome. These sessions showcase bottom‑up initiatives to develop relevant disease models, design gene therapy strategies and translate them towards clinical application.
By bringing together functional genomics, phenotyping, advanced experimental models and therapy development, the CCP Phenogenomics Conference provides a unique forum to explore how integrated research infrastructures and international collaborations are transforming the future of rare disease research and treatment.
Join leading researchers, clinicians, infrastructure partners, patient organisations and industry representatives in Prague or online for three days of cutting-edge science, collaboration and innovation.
We look forward to welcoming you to the 8th CCP Phenogenomics Conferencenogenomics Conference!