We Can’t Safely and Controllably Deliver Complex Molecular Payloads to the Targets We Want in the Body
Current in-vivo delivery systems (viral vectors, nanoparticles, microchips) face challenges such as off-target accumulation and inefficiency, particularly in delivering therapies to the brain. Novel delivery approaches are needed to improve targeting and performance.
Foundational Capabilities (5)
New delivery mechanisms are needed to target the desired tissues and avoid uptake by hepatocytes and macrophages. Off-target uptake necessitates higher dosing, increasing toxicity risks.
Develop improved gene therapy vectors that can be manufactured at scale, as well as completely novel therapeutic gene therapy delivery vehicles.
Utilize ultra-small microelectronic devices delivered via endovascular routes to target specific tissues (including the brain), bypassing the limitations of viral vectors and bulky implants.