Description:
GA-16 is a novel ionizable lipid designed with an N-(2-Hydroxyethyl)piperazine-N'-(4-butanesulfonic acid) (HEPBS) core. Its key functional characteristic is enabling highly efficient, liver-tropic mRNA delivery through an ApoE-mediated yet Low-Density Lipoprotein Receptor (LDLR)-independent cellular uptake mechanism. When formulated in LNPs with the helper lipid DEPE, GA-16 achieves superior protein expression. More importantly, this uptake pathway bypasses the canonical LDLR dependency, making it a strategically valuable component for developing therapies targeting genetic liver diseases like familial hypercholesterolemia, where patients often have deficient LDLR function.
Handling:
Providing storage is as stated on the product vial and the vial is kept tightly sealed, the product can be stored for up to 24 months.
Wherever possible, you should prepare and use solutions on the same day. However, if you need to make up stock solutions in advance,
we recommend that you store the solution as aliquots in tightly sealed vials at -20°C. Generally, these will be useable for up to one
month. Before use, and prior to opening the vial we recommend that you allow your product to equilibrate to room temperature for at
least 1 hour.