Using Site-Specific Transglutaminase-Based ASO Conjugation to Maintain Optimal Pharmacokinetics with Peripheral Administration
Time: 8:00 am
day: Conference Day One
Details:
- Using a stepwise process to identify optimal conjugation site, tags, reaction conditions and linker design to show high conjugate fidelity of peptide tags YRYRQ and RYESK
- Mitigating increase hydrophobicity by ASO payload with appropriate choice of conjugations site at heavy chain position 297
- Reviewing In vitro and in vivo pharmacokinetic behaviour of optimized brainshuttle-ASO conjugates based on a MAPT targeting oligonucleotide as a more widespread methodology for peripheral administration of ASO-baseddrugs for the CNS