Target Validation / Biochemical and Cellular Assay Development OSUCCC - James
Target validation and assay development are essential steps in the drug discovery process. This document discusses several approaches to target validation, including using genetic tools like CRISPR/Cas9 and RNAi to interrogate targets. It also provides an example of developing a cellular assay using patient-derived cells to validate a target for cystic fibrosis. Additionally, the document describes a case study where phenotypic screening was used to discover a small molecule that restores function of a mutant protein associated with Usher Syndrome type III.
Biomarkers can be used at various stages of drug development from target discovery through clinical trials. In clinical trials, biomarkers are used to demonstrate safety and efficacy. Safety biomarkers monitor organ function while efficacy biomarkers can serve as surrogate endpoints. Validation of biomarkers is required and involves establishing a relationship between the biomarker and clinical outcome through various phases of evaluation. Biomarkers must also be fit-for-purpose and their clinical validity depends on the trial design used to evaluate them.
This document outlines reverse pharmacology, which involves starting drug discovery with clinically documented effects from traditional medicines and exploring the underlying mechanisms through laboratory and clinical research. It discusses the definition, scope, and phases of reverse pharmacology. It provides examples of drugs discovered through reverse pharmacology from India, including drugs for hypertension, amoebiasis, and Parkinson's disease. It also discusses some challenges to reverse pharmacology and promising future perspectives if more collaboration between academia and industry can help validate traditional knowledge through rigorous clinical research.
Target Validation / Biochemical and Cellular Assay Development OSUCCC - James
Target validation and assay development are essential steps in the drug discovery process. This document discusses several approaches to target validation, including using genetic tools like CRISPR/Cas9 and RNAi to interrogate targets. It also provides an example of developing a cellular assay using patient-derived cells to validate a target for cystic fibrosis. Additionally, the document describes a case study where phenotypic screening was used to discover a small molecule that restores function of a mutant protein associated with Usher Syndrome type III.
Biomarkers can be used at various stages of drug development from target discovery through clinical trials. In clinical trials, biomarkers are used to demonstrate safety and efficacy. Safety biomarkers monitor organ function while efficacy biomarkers can serve as surrogate endpoints. Validation of biomarkers is required and involves establishing a relationship between the biomarker and clinical outcome through various phases of evaluation. Biomarkers must also be fit-for-purpose and their clinical validity depends on the trial design used to evaluate them.
This document outlines reverse pharmacology, which involves starting drug discovery with clinically documented effects from traditional medicines and exploring the underlying mechanisms through laboratory and clinical research. It discusses the definition, scope, and phases of reverse pharmacology. It provides examples of drugs discovered through reverse pharmacology from India, including drugs for hypertension, amoebiasis, and Parkinson's disease. It also discusses some challenges to reverse pharmacology and promising future perspectives if more collaboration between academia and industry can help validate traditional knowledge through rigorous clinical research.
This document provides short summaries of several famous artists from different eras:
- It mentions Italian Renaissance painter and polymath Leonardo da Vinci.
- It notes Spanish painter Diego Velázquez who was a leading artist for King Philip IV.
- It references French Post-Impressionist Paul Cézanne who influenced Cubism and 20th century art.
This document provides information on several artists including Jeff Koons, Cy Twombly, Damien Hirst, Cindy Sherman, Roy Lichtenstein, Francoise Nielly, Helen Constant, Tatiana Mikhina, and Maja Borowicz. It includes details about each artist's nationality, medium, notable works, and background.
This document provides an overview of Ecuador, including its geography, culture, history, food, and attractions. It notes that Ecuador has astounding biodiversity and is divided into three main regions - the sierra, Oriente, and coastal region. It highlights several cities and sites to visit, including Quito, Cuenca, Guayaquil, Cotopaxi volcano, and the Galapagos Islands. Ecuadorian culture blends indigenous traditions with Spanish influences. The document also discusses Ecuadorian cuisine, including ceviche, chocolate, coffee, and the importance of family in social culture.
This document provides short summaries of several famous artists from different eras:
- It mentions Italian Renaissance painter and polymath Leonardo da Vinci.
- It notes Spanish painter Diego Velázquez who was a leading artist for King Philip IV.
- It references French Post-Impressionist Paul Cézanne who influenced Cubism and 20th century art.
This document provides information on several artists including Jeff Koons, Cy Twombly, Damien Hirst, Cindy Sherman, Roy Lichtenstein, Francoise Nielly, Helen Constant, Tatiana Mikhina, and Maja Borowicz. It includes details about each artist's nationality, medium, notable works, and background.
This document provides an overview of Ecuador, including its geography, culture, history, food, and attractions. It notes that Ecuador has astounding biodiversity and is divided into three main regions - the sierra, Oriente, and coastal region. It highlights several cities and sites to visit, including Quito, Cuenca, Guayaquil, Cotopaxi volcano, and the Galapagos Islands. Ecuadorian culture blends indigenous traditions with Spanish influences. The document also discusses Ecuadorian cuisine, including ceviche, chocolate, coffee, and the importance of family in social culture.