AAV9 immunogenicity as a scalability trap. PROJECT OPTIMUS to boot. The anti-AAV9 titer cutoff of ≤1:50 reflects a real immunological constraint: patients with prior AAV9 exposure mount a neutralizing immune response that reduces transduction efficiency and risks severe inflammation. https://www.sciencedirect.com/science/article/pii/S1359644626001571?dgcid=authorhttps://lnkd.in/g2YVjCDb Pre-existing anti-AAV9 antibodies are present in roughly 30–50% of the general population, with the exact […]
Archive for August 24th, 2026
AAV9 immunogenicity as a scalability trap. PROJECT OPTIMUS to boot.
Posted by Michael A. S. Guth on August 24th, 2026
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A methodological audit: Where the glasses may be rose-colored
Posted by Michael A. S. Guth on August 24th, 2026
A methodological audit: Where the glasses may be rose-colored https://authors.elsevier.com/a/1nYxN4r9Rkz1wZ The open-label problem KOANEWA is a Phase Ib, open-label, non-randomized, single-dose study (ClinicalTrials.gov identifier: NCT07401121). The CENTAUR trial was open-label in its earliest iterations, and its 25% ALSFRS-R signal dissolved entirely under the blinded conditions of the PHOENIX Phase III trial (https://www.amylyx.com/news/amylyx-pharmaceuticals-announces-topline-results-from-global-phase-3-phoenix-trial-of-amx0035-in-als).(p2),(p3) Because the subjective ALSFRS-R is […]