80/20 Gene Therapy

What is the most undervalued area for longevity progress we should pursue? Where are we today? Where would we like to be? Only a small fraction of potential gene therapies are actually viable. This blocks many very good approaches to therapy, but it is an invisible cost. Few people have the vision to see what might be, rather than what is. We want a gene therapy platform enabling 80% of important arbitrary combinations of specific tissue expression, mid-term duration of expression, regulation acceptance, and repeat dosing. What public and private actions have the biggest impact on those goals? What people, funding, resources, experiments would be required to test this hypothesis? Option 1: Make AAV work – progress is underway, but too slowly and siloed. It may cost $100M to accelerate this process. Option 2: Local delivery – make the magic injection robot that enables 0% mortality for deep organ injection. Is Academia up to the challenge of making a robot that performs deep organ injections? Option 3: Make LNP+DNA/RNA work – targeting research and companies to work on developing nuclear targeting of plasmids, tissue specific control of RNA expression, promotor combinations, and Lipid Nanoparticle (LNP) immunogenicity reduction would be a roughly $20M challenge. Option 4: Accelerate research – drive the finalization of a better model than the above. Unknown as to what it might entail, maybe lipid nanocrystals or nanodiamonds. This is probably a $100M challenge.