Farzaneh salem

• Development and validation of PBPK models for predicting in vivo CNS exposure, distribution, and cellular engagement
• Characterizing cell-type–specific targeting in the CNS through consideration of biological barriers, cellular diversity, and molecular design
• Bridging preclinical insights to clinical application and identifying gaps  

NEW DATA

• Discussing safety profiles, stability and translational outcomes across different oligo subtypes including ASO’s, siRNA, saRNA, aptamers and more
• Comparative safety and toxicity considerations across subtypes including off target effects, immunogenicity and long-term durability
• Industry perspective on room for innovation and strategies to stand out in a crowded therapeutic landscape
• What differentiates next-generation modalities and how biopharma can differentiate themselves in an increasingly competitive space