Accessible and Transformational Therapeutic Approaches for Hereditary Haemorrhagic Telangiectasia (HHT)

Our client, a world leader in therapies for rare genetic diseases, is seeking novel disease modifying therapeutic approaches for hereditary haemorrhagic telangiectasia (HHT) type 1 and type 2. In particular, the team is interested in approaches based on antibody, small molecule, oligo, re-dosable in vivo gene therapy or endothelial cell delivery approaches.

Approaches of Interest:

• Increasing endoglin (ENG) or activin receptor-like kinase 1 (ALK1 or ACVRL1) expression/function
• Targeting BMP 9/10 signalling pathways
• Decreasing vascular malformations