TCL053

TCL053 is an ionizable amino lipid.1 It has been used in the generation of lipid nanoparticles (LNPs) and has a pKa value of 6.8. LNPs containing TCL053 and encapsulating mRNA encoding the Cas9 nuclease, in combination with LNPs containing TCL053 and encapsulating single-guide RNA (sgRNA) targeting the Rosa26 locus, have been used to induce CRISPR-mediated gene editing in the mouse gastrocnemius muscle.TCL053 is an ionizable lipid that has received FDA approval
for preparing mRNA vaccines. It is a three-tailed ionizable lipid
to overcome the disadvantage of nonrepeatable administration of
AAV vectors. In addition, combined with limb perfusion administration,
TCL053 iLNPs could transiently deliver CRISPR-Cas9
mRNA and sgRNA to multiple muscle tissues, reducing
immunogenicity and increasing the safety of iLNPs. It is
great progress for treating Duchenne muscular dystrophy and
other diseases that require multiple doses.