Dr. Carlo Ballatore

Drug Discovery, MedicinalChemistry

Carlo Ballatore Photo
Carlo Ballatore, Ph.D.

Skaggs School of Pharmacy and Pharmaceutical Sciences

(858) 822-3663
Research Summary

Our laboratory is engaged in collaborative, multidisciplinary probe- and drug-discovery programs. We specialize in the design and synthesis of small molecules as research tools or candidate therapeutics for a variety of human diseases. Our work typically involves Structure Activity and Structure Property Relationship (SAR/SPR) studies. These efforts are often directed towards the identification and development of probes suitable for mode of action studies, as well as candidate compounds optimized for pharmacokinetic and pharmacodynamic properties that could enable in vivo proof-of-concept studies or other preclinical evaluations of efficacy and safety. Over the past several years the primary focus of our research has been in the area of Alzheimer’s disease and related neurodegenerative tauopathies with specific programs directed towards the discovery and development of (a) tau aggregation inhibitors, (b) microtubule-stabilizing agents, and (c) thromboxane A2 receptor antagonists. The laboratory has also been actively involved in the investigation of basic, fundamental principles in medicinal chemistry, such as in the area of isosteric replacements.

Academic Achievements


“Laurea” in Chemistry and Pharmaceutical Technologies (1990-1995); University of Rome “La Sapienza”, Italy; Ph.D. in Medicinal Chemistry (1997- 2000); Cardiff University, University of Wales, U.K. (mentored by Prof. C. McGuigan); Post-doctoral training (2001-2002) in the laboratory of Prof. D. Farquhar, MD. Anderson Cancer Center, University of Texas, Houston. 

Awards and Honors:

Alzheimer’s Drug Discovery Foundation – research grant (2010)

Leadership Experience:

Dr. Ballatore has been responsible for medicinal chemistry at the Center for Neurodegenerative Disease Research, University of Pennsylvania (2005-2016).

Key Contributions
  • Our collaborative efforts in the area of microtubule-stabilizing agents as potential treatment for neurodegenerative tauopathies led to the identification of several non-naturally occurring small molecules that exhibit desirable properties, including brain penetration and oral bioavailability. Compounds of this type may be considered as research tools and/or potential therapeutic leads for a variety of diseases of the central nervous system (CNS).
  • Over the years, the laboratory made contributions in the area of isosteric replacements, namely carboxylic acid bio-isosteres. In this context, the lab helped characterize and develop novel surrogate structures of the carboxylic acid functional group with potential utility in drug design.
Potential Collaborative Programs
  • Our laboratory may be a useful resource for both academic and industrial collaborators with an interest in the use or evaluation of CNS-active microtubule stabilizing small molecules.
  • Given our long-term interest and expertise in the area of isosteric replacements, the laboratory is ideally suited to collaborate in drug discovery programs that may benefit from this strategy.