Technical writing from the Biopathio team on LNP formulation for CNS delivery, BBB transcytosis mechanisms, CRISPR payload encapsulation, and the rare CNS disease delivery landscape.
AAV has the clinical track record in CNS; LNPs have the cargo flexibility and redosing potential. A technical comparison of what each platform actually does well — and where each has unresolved limitations for CNS gene therapy programs.
Read articleDLS, RiboGreen EE, and cryo-TEM confirm that a particle formed. They do not tell you whether it will cross the BBB or escape endosomes in neurons. What an expanded CNS characterization panel needs to include — and why the standard panel falls short for non-hepatic delivery.
Read articleTfR1 and LRP1 are expressed at high levels on brain endothelial cells and mediate vesicular transport into the CNS parenchyma. What receptor-mediated transcytosis actually is, what the hCMEC/D3 transwell evidence shows, and what engineering LNPs for this pathway requires in practice.
Read articleHuntington's (HTT), Friedreich's ataxia (FXN), and CLN3 Batten disease: validated genetic targets, credible in vitro editing evidence, and no non-viral delivery system that can reach these neurons after systemic administration. A target-by-target breakdown of the delivery gap.
Read articleFenestrated endothelium, ApoE-mediated hepatocyte uptake, and hepatocyte-specific pKa windows — the three design assumptions that make mainstream LNP platforms work well in the liver are the same three assumptions that break down at the BBB. A mechanistic account of why, and what CNS-first LNP design actually requires.
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