💙 #CCPConference2026 closed with a powerful patient-research session: SPATA5, Angelman syndrome & ADSL deficiency, bringing families, foundations & scientists together. The conference concluded with Kiran Musunuru on therapeutic gene editing. 🧬

From genotype to phenotype 🧬 Thursday’s final session connected variant biology, precision medicine and therapy. Stanley T. Crooke presented n-Lorem’s approach to developing personalized ASOs for patients with pathogenic mutations—free for life. #CCPConference2026 #RareDiseases

From disease mechanisms to therapies 🧬 Session 3 at #CCPConference2026 explored genome editing, liver disease, epigenetic disorders and renal cyst formation. Understanding when, where and how gene function goes wrong is key to effective intervention.

From models to therapies 🔬
Yesterday’s Session 2 at #CCPConference2026 showcased how advanced preclinical models can bridge disease mechanisms and treatment:
The key message: the right model can turn genetic insight into therapeutic opportunity.
#RareDiseases #Phenogenomics

Meet our speaker: Kiran Musunuru! 🧬 At #CCPConference2026, he will explore therapeutic gene editing for cardiovascular & metabolic diseases, including emerging platforms for personalized gene-editing therapies. From human genetics to transformative treatments.
👉https://www.ccp-conference.cz/

Meet our speaker: Stanley T. Crooke! 🧬 Founder of n-Lorem, he pioneered antisense therapeutics. At #CCPConference2026 👉https://www.ccp-conference.cz/, he will present the n-Lorem approach to developing personalized ASOs for patients with nano-rare diseases—and providing them free for life.

Meet our speaker: Tim Bertram! 🔬 At #CCPConference2026 👉https://www.ccp-conference.cz/, he will explore how scientific discoveries become technologies—and ultimately medicines, highlighting our collaboration on UMOD & MUC1 rat models for rare kidney disease. 📍Prague & Online