About the Experimental Therapeutics Institute

Mount Sinai's Experimental Therapeutics Institute in New York City is a hub for the development of new drugs, devices, and intellectual property that result from Mount Sinai’s basic and clinical research programs. Integrated research programs focus on:

• Small-molecule drug discovery
• Biological therapeutics
• Therapeutic vaccine development
• Target validation and side effects
• Preclinical testing

Institute investigators conduct scientific and commercial assessments of promising research and technology, perform preclinical testing, and foster commercial relationships with pharmaceutical and medical device companies.

The Small-Molecule Drug Discovery Program

This program is developing small-molecule therapeutic agents against new disease targets identified by Experimental Therapeutics Institute investigators and creating technologies that will streamline the drug discovery process for small molecules.

The program has three specific goals:

• Leverage the commercial potential of academic drug discovery through genome-wide target discovery and profiling
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• Accelerate lead optimization with focused chemical synthesis through the use of rational design and combinatorial or parallel synthesis strategies to optimize lead compounds efficiently
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• Enhance the intellectual property values of the most promising new therapeutic targets by generating toxicology profiles

In building on a record of discovery, molecular targets have been identified and drug discovery processes are being developed for cancer, HIV/AIDS, pain, osteoporosis, psychiatric disorders, peanut allergy, type II diabetes, obesity, inflammatory bowel disease, polycystic kidney disease, and heart failure. This work will allow researchers throughout Mount Sinai to apply cutting-edge technologies in chemical biology, cheminformatics, and medicinal chemistry.

Biotherapeutics Division

Designed to be highly specific, biotherapeutics promise to replace many of the currently marketed, chemically synthesized drugs. Mount Sinai has proven expertise in the research and development of biotherapeutics, as well as experience in bringing these products to market. This division has three units:

• Protein-based biotherapeutics
• Gene-based biotherapeutics
• Cell-based biotherapeutics

The protein-based biotherapeutics unit will build upon a successful tradition of product development at Mount Sinai. The gene-based biotherapeutics unit will focus its efforts on gene transfer and stem cell research. The cell-based biotherapeutics unit translates knowledge of stem cell biology into therapies for tissue regeneration.

Therapeutic Vaccine Division

The Therapeutic Vaccines Division combines the strengths of Mount Sinai in the discovery, animal testing, and clinical testing of new generations of vaccines. Traditionally, vaccines have been developed to prevent transmission of infectious diseases or to elicit a beneficial immune response against infectious, inflammatory, and malignant processes, such as cancer.

Today, researchers are focusing on developing therapeutic vaccines for chronic viral infections, including HIV, hepatitis C, antibiotic-resistant bacteria, and anthrax, to name a few. In addition, therapeutic vaccines could treat other inflammatory diseases, including autoimmune diseases, severe allergic responses, and malignancies.

Target Validation and Side Effects Program

The Target Validation and Side Effects (TVSE) Program applies mathematical, statistical, and bioinformatics approaches to develop a systems-level understanding of how all classes of therapeutics (small molecule, protein, gene, and cell medicines) work at the whole-organism level. This integrated systems pharmacology approach enables the Experimental Therapeutics Institute to analyze and predict both the beneficial and unwanted effects of a given compound.

The TVSE program uses the emerging discipline of network sciences as a bridge between mathematical and statistical models and the more traditional applications of statistical analyses for predicting drug effects. The program will integrate its activities with those of the Charles R. Bronfman Institute for Personalized Medicine and the clinical informatics initiatives at The Mount Sinai Hospital. These collaborations will enable the Experimental Therapeutics Institute to develop computational models that can predict the effects of potential therapeutics on patients with different genotypes.

Preclinical Testing Program

The Preclinical Testing Program will serve as a central, institution-wide resource. The program will provide expertise for toxicology and side effect testing in cellular models in an integrated manner with the TVSE program and the Charles R. Bronfman Institute for Personalized Medicine.