This program project envisions running two small proof of concept phase I clinical trials in human subjects. To facilitate the work of this program project grant, Pathologica will be responsible for all aspects of the manufacture, quality control, and quality assurance required to produce an investigational new drug. This component includes, the manufacture of the compound(s), packaging of the drug(s), stability testing, production of clinical investigator brochures, and filing of the INDs for both trials. The first trial is envisioned to involve the polyamine biosynthesis inhibitor PA-001 in the setting of HIV associated dementia and related syndromes. PA-001 has shown excellent activity against SIV encephalitis in rhesus macaque model (see Project 2: Animal model studies for more details). The compound for the second trial will be chosen based on the results of the in vitro and animal studies performed during the first two years of the program project, but will also be a polyamine biosynthesis inhibitor. Pathologica LLC is fortunate to have rights to evaluate and commercialize many PBIs including the polyamine analog CG47, and the PBI manufacture core will ensure that sufficient quantity and quality of these polyamine analogs will be available for comparative studies with PA-001 as described more fully in Projects 1 and 2 of this program project grant. To provide the drugs Pathologica will be assisted by Dr Fredrick Johnson, who has more than 20 years of experience in the manufacture and quality assurance testing of both small molecule and biological pharmaceutical products.
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