Biotech Values

[genisi]

BXLT switched to an FVIII variant, which also increased gene expression in animal models. Whether the increased gene expression was the result of luck or skill is unclear to me.

Lots of work has been done in order to overcome the size problem of the cDNA for packaging into viral vectors and it is known for over a decade that a deleted B-domain FVIII variant is the way to go because it reduces the size and increases mRNA levels compared to the wild type. From here, designing and testing different partially deleted B-domain FVIII variants was clearly the path and I would think it takes both luck and skills but mainly hard work to come up with the best variant (improved properties to result in better dosing, efficacy, safety, and durability).