This administrative supplement grant application focuses on optimizing the sterilization method and large-scale synthesis of the lead nanoconstruct PDX-001-TX (developed in the parent award, R44CA217534), and supporting NIH?s mission to encourage institutions to diversify their student and faculty populations in biomedical, clinical, behavioral, and social sciences. Manufacturability is key in evaluating the translational potential of a lead drug candidate. To this effort, we aim to optimize the sterilization protocol of our material to produce clean and safe material for future clinical use. We also plan to perform large-scale synthesis of our lead nanoconstruct and evaluate its physicochemical characteristics, therapeutic efficacy, and sterility. Results will provide important data towards an investigational new drug (IND) application to the FDA. PDX Pharmaceuticals LLC has selected one post-baccalaureate researcher to design and conduct experiments, under the guidance and mentorship of the PIs, to achieve these goals. The post-baccalaureate candidate is classified by NIH?s guidelines as an individual who comes from an economically disadvantaged background. Thus, this supplement award will provide the candidate the opportunity to receive training in biomedical research, enhance his business skills, increase his prospect of graduate school admission, and help him develop into a successful scientist.
Capability to produce a lead nanotherapeutic in large quantities must be established for clinical translation. Therefore, we propose to study the manufacturability of our lead nanoconstruct by performing large-scale synthesis and establishing sterilization methods. The project will also support NIH?s mission to promote diversity in research and small businesses, as the post-baccalaureate candidate is an aspiring scientist who comes from an economically disadvantaged background.
| Ngamcherdtrakul, Worapol; Sangvanich, Thanapon; Reda, Moataz et al. (2018) Lyophilization and stability of antibody-conjugated mesoporous silica nanoparticle with cationic polymer and PEG for siRNA delivery. Int J Nanomedicine 13:4015-4027 |
| Gu, Shenda; Ngamcherdtrakul, Worapol; Reda, Moataz et al. (2018) Lack of acquired resistance in HER2-positive breast cancer cells after long-term HER2 siRNA nanoparticle treatment. PLoS One 13:e0198141 |
