Novel macrodiolide Immunosuppressants

Introduction

Several major unmet medical needs, such as autoimmune disorders and neuroinflammatory conditions, are characterized by aberrant Type-1 interferon production. This suggests that new drugs specifically targeting this central inflammatory signaling axis could provide significant value for patients and society.

Project Overview

In this project, we will develop the foundation for future commercial exploitation of a class of novel macrocyclic immunosuppressants. These compounds have been shown to possess unprecedented broad inhibitory activities against several innate immunological pathways that converge on the transcription factor IRF3 linked to interferon production.

Objectives

Based on diverse preliminary data that span both cellular biology and chemical synthesis strategies, the planned work aims to:

1. Secure a strong IP position by providing novel structure-activity relation data on relevant cellular activities linked to IRF3 activity.
2. Identify potential compound leads with favorable pharmacokinetic properties to be pushed forward for future in vivo experiments.

Focus Areas

The primary focus will be on:

• Ability to permeate the blood-brain barrier (to address major neuroinflammatory conditions).
• Achieving good plasma stability.

Business Strategy

Alongside the concrete experimental tasks and strengthening of the IP position, a broader business plan and associated funding strategy will be prepared. This involves:

• Engagement of key future stakeholders.
• Establishment of an advisory board.

Conclusion

Overall, the planned activities serve to make the project ready for a pre-seed investment or acceptance into a biotech accelerator program following completion of the ERC-PoC project.