Inherited autoinflammatory diseases (AID) are devastating and often life-threatening disorders with a prevalence far below 5/10,000. For most AID no efficient treatment options are available. Auto-ALARM brings together renowned research groups with complementary expertise ranging from molecule to patient care. Auto-ALARM aims to build the foundation for cutting-edge new therapies with disease-relevant animal and in vitro disease models, preclinical screening technology and good manufacturing practice (GMP) production of new compounds.
OBJECTIVES Auto-Alarm Based on the expertise and the recent scientific breakthroughs of its partners, Auto-ALARM aims to develop new treatment options for non-classical AID, as reflected in the following objectives:
▪ Objective 1: To develop disease- and molecular pathway-specific preclinical animal models and human ex vivo approaches for development of new and specific therapeutic options.
▪ Objective 2: To develop a small molecular compound for blocking S100-TLR4 driven auto inflammation and a blocking agent against pro-inflammatory HSP60.
▪ Objective 3: To establish inhibitory activity of compounds developed in objective 2 in preclinical models: mice and in ex-vivo assays with patient derived cells (disease in a dish).
▪ Objective 4: To define new molecular targets for innovative treatment of non-classical AID by identification of novel gene mutations and epigenetic modifications causing autoinflammation.