De-LIVER – Solving Extra-Hepatic Targeting Bottlenecks With Delivery to Combat Rare Diseases
Time: 10:31 am
day: mRNA Technology & Innovation Day
Details:
Recent advances in mRNA technology and its delivery have enabled mRNA-based therapeutics to enter a new era in medicine with unlimited potential. The rapid, potent, and transient nature of mRNA-encoded proteins, without the need to enter the nucleus or the risk of genomic integration, makes them desirable tools for treatment of a range of diseases.
Nonetheless, challenges remain with regards to mRNA expression, delivery efficiency, and targetability, to broaden the applicability of mRNA therapeutics. This workshop brings together key experience from novel proof-of concept and pre-clinical programs venturing into new target areas including the kidney and lung to optimize mRNA platforms to meet clinical needs of each disease.
Case-Study Examples Include:
- Strategies for active targeting of LNPs to the kidney
- Respiratory delivery of mRNA – overcoming hurdles for therapeutic application
