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
Expert Review of Neurotherapeutics
Advancing treatment of spinal muscular atrophy through inhibition of the myostatin signaling pathway
February 2026
Immuno-Oncology
Linavonkibart publication
Linavonkibart and pembrolizumab in immune checkpoint blockade-resistant advanced solid tumors: a phase 1 trial
January 2026
Spinal Muscular Atrophy
The Lancet Neurology
Safety and efficacy of apitegromab in nonambulatory type 2 or type 3 spinal muscular atrophy (SAPPHIRE): a phase 3, double-blind, randomised, placebo-controlled trial
August 2025
Recent Posters
Spinal Muscular Atrophy
SMA Europe 2026
Understanding physical therapist perspectives on the clinical meaningfulness of ‘any point differences’ on the Hammersmith Functional Motor Scale-Expanded in SMA
March 2026
Spinal Muscular Atrophy
SMA Europe 2026
Understanding neurologist perspectives on the clinical meaningfulness of ‘any point differences’ on the Hammersmith Functional Motor Scale-Expanded in SMA
March 2026
Facioscapulohumeral Muscular Dystrophy
Muscular Dystrophy Association Clinical & Scientific Conference 2026
Trial design for the Phase 2 FORGE study evaluating apitegromab in adults with facioscapulohumeral muscular dystrophy
March 2026
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.
