(420) Towards Clinical Translation of Autophagy Inhibitor BAQ13 LNP (TR002) for Cancer Therapy
Introduction: Targeting autophagy is a promising strategy to overcome drug resistance in cancer.[1] BAQ13, a novel autophagy inhibitor, has shown promising antitumor activity in preclinical models of pancreatic, brain, and gastrointestinalcancers.[2] BAQ13 self-assembles into lipid nanoparticles (LNPs) alone or with helper lipids. This study aimed to (1) develop a tunable and scalable LNP formulation of BAQ13 (TR002) via microfluidics, (2) produce GLP/cGMP-grade batches, and (3) conduct toxicology in rats and dogs. The work led to FDA IND approval (165331) for Phase 1 trials.
Learning Objectives:
Understand the optimization strategy and scale up process using microfluidics
Develop a stable formulation that meets cGMP standards
Assess the safety profile of TR002 in both rodent and non-rodent preclinical models
