Our Science
Unsolved problems require new solutions
Scholar Rock’s therapeutic approach applies structural insights and a deep understanding of the underlying biology of diseases to our proprietary platform. Our scientific philosophy: challenge traditional ideas and beliefs about what is possible to drive new discoveries and breakthrough treatments for patients with serious disease.
The biology behind our therapeutic approach
We aim to treat complex diseases at the root of biology. Our unique approach is to target growth factors in their latent form, prior to activation. This approach allows us to regulate growth factor activity with exquisite selectivity.
Accelerating new discoveries for patients
Learn more about Scholar Rock’s innovative therapeutic approach to treating complex diseases.
Harnessing the life-changing potential of TGFβ biology
We apply this approach across growth factors in the TGFβ (transforming growth factor beta) superfamily, which are proteins that play fundamental roles in many serious diseases. Our proprietary platform enables development of therapeutic antibodies that are designed to selectively regulate the activation of specific growth factors, while avoiding interference with unintended, related growth factors and potentially minimizing adverse effects. Some of the monoclonal antibodies we have identified through this approach have advanced to the clinic.
Recent Publications
Spinal Muscular Atrophy
Frontiers in Neurology
Long-term efficacy, safety, and patient-reported outcomes of apitegromab in patients with spinal muscular atrophy: results from the 36-month TOPAZ study
July 2024
Fibrosis
Science Signaling
An antibody that inhibits TGFβ1 release from latent extracellular matrix complexes attenuates the progression of renal fibrosis
July 2024
Spinal Muscular Atrophy
Neurology
Safety and Efficacy of Apitegromab in Patients With Spinal Muscular Atrophy Types 2 and 3
February 2024
Recent Posters
Neuromuscular
Muscular Dystrophy Association Clinical & Scientific Conference
SRK-015 builds muscle mass and strength in combination with dystrophin upregulation in a mouse model of DMD
March 2025
Spinal Muscular Atrophy
Muscular Dystrophy Association Clinical & Scientific Conference
Efficacy and safety of apitegromab in individuals with type 2 and type 3 spinal muscular atrophy evaluated in the phase 3 SAPPHIRE trial
March 2025
Immuno-Oncology
Society for Immunotherapy of Cancer (SITC) 2024
DRAGON trial: durable remission rate with the latent TGFβ1 inhibitor linavonkibart (SRK-181) and pembrolizumab in patients with immune checkpoint inhibitor-resistant advanced cancers
November 2024
Partnerships
We seek partnerships that accelerate scientific innovation and advance our mission to discover, develop, and deliver life-changing therapies.
Join Us
Like what you see? Visit our Careers page to see if Scholar Rock is the right fit for your next step.
Join Us
Like what you see? Visit our Careers page to see if Scholar Rock is the right fit for your next step.
