Gene Editing Technology & Applications - CRISPR technology, inspired by the Nobel Prize-winning discovery of Jennifer Doudna and Emmanuelle Charpentier, functions as a programmable editor to repair genetic defects [5][6] - The technology utilizes a CRISPR protein and guide RNA complex to target and precisely repair problematic DNA sequences [7][8] - Ultra-compact CRISPR systems have been developed to overcome delivery challenges, particularly for tissue-specific delivery via AAV [12][13] - The first FDA-approved CRISPR therapy involves editing blood stem cells outside the body to treat sickle cell disease [14] Delivery Systems - Two primary gene delivery systems exist: Lipid Nanoparticles (LNPs) and Adeno-Associated Viruses (AAVs) [10] - LNPs act as cargo ships, carrying large CRISPR components, primarily docking in the liver [10][11] - AAVs function like drones, delivering smaller CRISPR payloads to specific cell types while minimizing immune responses [11][12] Challenges & Future Directions - Access to cells and tissue types beyond the liver remains a significant challenge for widespread CRISPR deployment [9][16] - Addressing the safety, efficacy, scalability, and accessibility of CRISPR medicines for over 5,000 known genetic diseases is crucial [16] - Advances in AI are accelerating the development of next-generation CRISPR medicines [17] - The industry envisions a future where curing genetic diseases becomes as simple as pairing an ultra-compact CRISPR system with a targeted delivery method [17][18]