Platform
Deep biological insights
Our foundational research focuses on diseases where the biological cause is understood, but difficult to target through traditional, non-selective approaches. Our proprietary platform has enabled us to develop a robust pipeline of novel, selective assets, including multiple clinical programs and a growing preclinical portfolio.
Our revolutionary science
Protein growth factors are regulators of many normal biological processes. Many of these growth factors are produced by the cell in an inactive, latent form and must be processed to a mature, activated form to have their biological effect.
Protein growth factors are key to unlocking advances in new therapies, but traditional approaches have focused mainly on fully matured, activated growth factors. This approach has historically been met with limitations, as mature, activated growth factors are structurally similar to one another, making it difficult for therapeutic drugs to precisely reach their intended target. This may lead to undesired off-target effects.
Latent—or inactive—growth factors, on the other hand, are much easier to tell apart. By designing antibodies to selectively target a single latent growth factor complex to exert a therapeutic benefit, we leave other growth factors intact to ensure their normal biological functions are not affected.
We believe that this unique approach to targeting latent growth factors has broad and differentiated therapeutic applications. Our approach is particularly well suited for neuromuscular disease, cardiometabolic disorders, and other conditions where selectively targeting growth factors can be transformational—enabling us to create new possibilities for patients.
Cultivate Curiosity
Passion for science is our pathway to excellence, allowing thoughtful questions to drive daring ideas.
Our Approach: Selectivity Drives Success
Setting the Scene
- Cells produce certain growth factors in a precursor, or latent form.
- For these growth factors to carry out their functions, they must first be activated (through molecular events) and separated from the prodomain.
- Traditional approaches to targeting these growth factors have focused on targeting the activated, mature growth factors, which are already separated from the prodomain and have a short half-life, limiting the therapeutic window. Many of these mature growth factors also have very similar structures, rendering them difficult to target selectively.
- Lack of selectivity often means more side effects.
Enter Scholar Rock
- Our antibodies target the latent form of growth factors and prevent release of the mature growth factor. Given the chemical and structural differences of the prodomain, our approach allows us to generate antibodies with extraordinary selectivity.
- This enables Scholar Rock to potentially treat diseases with high unmet need while limiting unintended side effects.
