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.
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.