09.16.26 -- The Delivery Challenge For In Vivo Cell Engineering

PMWC 2027: The program is live. Explore 15 focused programs spanning next-generation therapeutics, AI and computational medicine, precision diagnostics, and integrated precision medicine. Co-hosted by Stanford, UCSF, and Yale, PMWC features leaders including David Baker, Emmanuelle Charpentier, Andrew Ng, Fei-Fei Li, and Mary-Claire King, January 27-29, 2027, at the Santa Clara Convention Center.

Over the past few weeks, I’ve been sharing my takeaways from the overall event, as well as from a specific panel featuring JHU’s Jeff Coller, CHOP’s Rebecca Ahrens-Nicklas, and Plowshare Therapies’ Kevin Strauss. Together, these three discussed the biggest learnings and takeaways from the miraculous Baby KJ experience that we very much hope to replicate in the future. As you can likely imagine, patient and physician communication played a large role in the discussion at hand. 

SynaptixBio CEO Dan Williams explains how the company selected an ASO for TUBB4A leukodystrophy by balancing potency, safety, and translational rigor to advance a rare disease therapy.

As RNA therapies expand beyond the liver, delivery—not payload design—has become the defining challenge for precise, scalable in vivo cell engineering.

AI can optimize mRNA only if it's trained on biologically relevant data. Better therapeutic datasets — not just better algorithms — will drive the next generation of RNA medicines.