Translational research--from gene to treatment: lessons from cystic fibrosis

Area of Science:

• Biomedical research
• Drug discovery
• Gene therapy

Background:

• Biomedical research is identifying numerous new therapeutic targets for human diseases.
• Translating this knowledge into effective treatments requires learning from pharmaceutical industry drug development experience.
• Cystic fibrosis serves as a model for new treatment development due to recent advancements and numerous potential targets.

Purpose of the Study:

• To explore new models for drug discovery and development.
• To emphasize the need for collaboration between various stakeholders in biomedical research.
• To investigate how to make drug discovery cheaper and faster.

Main Methods:

• Case study analysis using cystic fibrosis drug development.
• Review of traditional drug discovery patterns.
• Examination of emerging research organization models, particularly in gene therapy.

Main Results:

• Traditional drug discovery models may be insufficient.
• Collaboration among academics, charities, biotech, and pharmaceutical companies can enhance efficiency.
• New models of funding and collaboration are emerging, especially for gene therapy.

Conclusions:

• New therapeutic targets necessitate innovative approaches to drug development.
• Collaborative models are crucial for accelerating and reducing the cost of drug discovery.
• Emerging organizational structures in gene therapy research offer a template for future endeavors.