TherapieTargets

TherapieTargets, commonly referred to as therapeutic targets, are biomolecules, genes, or pathways that can be modulated to treat disease. They include proteins such as enzymes, receptors, and ion channels; nucleic acids; and cellular processes. Target modulation can be achieved by small molecules, antibodies, RNA-based therapies, or gene editing, with the choice depending on target accessibility and disease relevance. Successful targets are typically causally linked to disease progression and show a favorable safety profile when modulated.

Identification of TherapieTargets relies on a blend of biology, chemistry, and data science. Researchers use literature mining, omics data (genomics, proteomics, transcriptomics), genetic association studies, and phenotypic screens. Computational methods map disease networks and prioritize targets based on genetic evidence, druggability, and potential for selective intervention. Validation involves demonstrating target engagement (that a modulator interacts with the target at therapeutic concentrations), functional modulation in cellular assays, and efficacy in relevant disease models. Off-target effects and safety implications are assessed early.

Druggability is a key consideration for TherapieTargets. Not all targets are druggable. Criteria include the presence of a binding pocket, the ability to achieve sufficient modulation without unacceptable toxicity, and a tractable path to a safe pharmacokinetic profile. Early pharmacology estimates, ADME, and toxicity studies guide progression.

Role and challenges in development: The target-based approach coexists with phenotypic screening. Targets may vary in relevance across diseases and patient subgroups, supporting precision medicine. Redundancy in biology, compensatory pathways, and resistance mechanisms pose ongoing challenges. Regulatory approval depends on demonstrable clinical benefit and manageable safety.