This unit gives support to principal investigators and innovation agencies in preparing a first target product profile, assists with platform and project coordination, and gives assessment of target dependent toxicity and support with the design of pharmacological studies in vivo.
Target Product Profiling
The concept of Target Product Profiling (TPP) was introduced by the USA Food and Drug Administration (FDA) in 2007. The purpose of a TPP is to provide a format for describing a drug project that can be used throughout the drug development process in discussions with reviewing bodies and investors. Directors Per Arvidsson and Kristian Sandberg work closely together with principal investigators (PIs) and Innovation Agencies to prepare a preProject report (the first draft of a TPP) when a new project enters the Drug Discovery and Development platform at SciLifeLab. The following dimensions of a drug discovery project are described in the preProject report:
Drug Safety Assessment
The DDD platform at SciLifeLab has a strategic collaboration with Björn Glinghammar (RISE AB) for safety assessment of drug projects in the platform. Every project is evaluated for potential target safety concerns, e.g. toxicity due to agonism or inhibition of the target protein. This analysis may give information which is vital for the success of the project, and can even help at early stage to eliminate non-druggable targets.
For small molecules, chemical toxicity properties are screened in the DDD process (see figure). Safety support to antibody projects focuses around avoidance of cytokine storms, prediction of immunogenicity and presence of anti-drug antibodies.
Design of conclusive In Vivo studies
Preclinical proof of concept studies are essential to demonstrate feasibility for further development of lead compounds and antibodies. Our project coordinator Rebecka Klintenberg has extensive experience in pharmacological studies and leads a group with the task to give advise to the principal investigator. Key questions discussed are: