Nicolas Moniotte: Dual-Platform Gene Editing for Haemophilia A
Nicolas Moniotte, Biopharma consultancy at Gutaperka, shared a post on LinkedIn:
“Two delivery systems, one gene-editing therapy.
How do you control potency?
Metagenomi has received FDA IND clearance for MGX-001, a gene-editing therapy for hemophilia A.
The approach combines (i) an AAV delivering a promoterless Factor VIII gene cassette and (ii) an LNP delivering CRISPR nuclease mRNA and guide RNA. Both are needed for targeted integration into the albumin locus and durable Factor VIII production.
In NHP, Metagenomi reported FVIII activity within the normal physiological range, with dose dependency for both AAV and LNP.
How do you establish potency and comparability when therapeutic activity depends on two distinct delivery platforms?
One approach could combine individual component assays with a functional assay in hepatocytes exposed to both components, measuring targeted integration by ddPCR and FVIII activity.
Such an assay could be valuable for characterization and comparability, but its complexity and variability may make routine batch release challenging.
The real challenge is connecting the quality of each component to the performance of the combination.”
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