Claire Zeng
Founder, Chief Technology Officer & Chief Executive Officer DoriNano
Seminars
The breakthroughs in RNAi delivery are no longer confined to the liver, with a new generation of platforms, from antibody conjugates and DNA nanostructures to novel ligands and chemical modifications, enabling precise targeting of metabolic, muscular, and renal tissues. This roundtable unites pioneers to discuss:
- Weighing the advantages of modular platforms against bespoke conjugate strategies for specific tissue targets, considering factors like valency, manufacturability, and immunogenicity.
- Addressing the unique synthesis, characterization, and manufacturing challenges of complex delivery modalities to ensure quality, stability, and scalability for clinical and commercial supply.
- Strategizing on how to design platforms with inherent adaptability to rapidly retarget new tissues and diseases, maximizing platform value and accelerating development timelines.
- n selection for optimal risk/reward, ensuring portfolio companies can attract pharma partnerships or IPO success
- Engineering multi-valent delivery vehicles to optimize cellular uptake and endosomal escape to avoid the limitations of conventional lipid nano particles
- Leveraging programmable nanoscale architecture and enabling modular assembly of nano particles for precision delivery of siRNa Payloads
- Demonstrating potent efficacy of DNA origami-siRNA constructs in preclinical models of disease
Chemical engineering of RNAi therapeutics is pivotal for expanding their delivery beyond the liver, to tissues like the CNS, muscle, and eye. Attendees will gain insights into optimizing RNAi constructs for improved stability,
potency, and delivery efficiency, bridging the gap between preclinical promise and clinical success. A must-attend for researchers aiming to unlock the full potential of RNAi in extrahepatic applications by discussing:
- Incorporating phosphorothioate and 2-modifications to boost siRNA nuclease resistance, enabling prolonged activity in extrahepatic tissues
- Designing lipid-conjugated siRNAs for enhanced cellular uptake without carriers, simplifying delivery to challenging targets like skeletal muscle or the eye
- Tailoring hydrophobicity and charge to optimize biodistribution and endosomal escape, maximizing target gene knockdown in non-liver organs
