Decoding RNA regulation: Challenges and opportunities for RNA-based therapies in Europe - PubMed
3 days ago
- #RNA therapeutics
- #Personalized medicine
- #Regulatory framework
- The study analyzed 13 RNA-based therapeutics in the EU for non-infectious diseases, with 10 approved, 2 refused, and 1 withdrawn by March 2025.
- RNA-based medicinal products offer personalized medicine potential but face regulatory uncertainties in the EU, including inconsistent definitions and evidence requirements.
- Most products (12 of 13) use sequence-specific hybridization to target pre-mRNA or mRNA, primarily for rare diseases, with liver diseases being a common focus.
- Delivery systems such as GalNAc conjugation and lipid nanoparticles are frequently employed in these therapies.
- Clinical challenges include limited efficacy data (e.g., small trials and surrogate endpoints) and recurring safety concerns, especially coagulation-related risks for ASOs.
- A classification framework based on RNA mechanism and chemical modifications is suggested to improve regulatory clarity and support development, particularly for rare diseases.