We are discovering and developing novel, selective inhibitors of TGFβ for the treatment of fibrotic diseases. Our emerging medicines could offer a powerful new approach to suppressing pro-fibrotic signaling in multiple organs, while avoiding known toxicities.
- Context-Independent Latent TGFβ1
- Context-Dependent Latent TGFβ1 / LTBP1 & LTBP3
While TGFβ is at the apex of the fibrotic signaling cascade, there are safety concerns linked to simultaneous targeting of multiple growth factors by non-selective approaches. Selectivity is key to the Scholar Rock platform and our approach to targeting TGFβ.
We identified multiple types of TGFβ1 inhibitors with unique selectivity profiles at the source of disease in the tissue microenvironment. All are selective for TGFβ1 relative to TGFβ2 or TGFβ3 and include:
- inhibitors that selectively target activation of latent TGFβ1 in all contexts
- inhibitors that selectively target activation of latent TGFβ1 localized to extracellular matrix
Further, we demonstrated in preclinical studies that these specific inhibitors can prevent the activation of the growth factor in the fibrotic matrix and may offer a powerful new approach to suppressing pro-fibrotic signaling in multiple organs. We’re advancing our TGFb portfolio with the aim of selecting molecules to be developed as new medicines for patients with fibrotic diseases.