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Columbus Children's Research Institute is a Columbus, OH-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
RiboCor is a Minneapolis, MN-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
MB Research Laboratories is a Spinnerstown, PA-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
Scholar Rock is a clinical-stage biopharmaceutical company focused on the discovery and development of innovative medicines for the treatment of serious diseases in which signaling by protein growth factors plays a fundamental role. Our most advanced product candidate, apitegromab (SRK-015), a selective first-in-class inhibitor of the activation of the growth factor myostatin in skeletal muscle, is currently in development for the treatment of spinal muscular atrophy, or SMA. We are currently evaluating apitegromab in our TOPAZ Phase 2 proof-of-concept trial in patients with later-onset (Type 2 and Type 3) SMA. Positive six-month interim analysis results were announced in October 2020 and top-line data for the full 12-month treatment period are anticipated in the second quarter of 2021. In addition, we are conducting our DRAGON Phase 1 proof-of-concept trial for SRK-181, a highly specific inhibitor of latent TGFβ1 activation, in patients with locally advanced and metastatic solid tumors. We believe SRK-181 has the potential to overcome resistance and meaningfully increase the number of patients who may benefit from checkpoint inhibitors, such as anti-PD-1 or anti-PD-L1 therapies. Utilizing our proprietary platform, we are creating a pipeline of novel product candidates with the potential to transform the lives of patients suffering from a wide range of serious diseases, including neuromuscular disorders, cancer, fibrosis and anemia.
Actym Therapeutics has engineered a new drug modality harnessing the power of a genetically modified bacterial vehicle that safely introduces therapeutic payloads to activate the immune response in the tumor microenvironment. To achieve targeted anti-tumor effects, we have developed a systemically administered treatment that exploits intrinsic TME-specific metabolites, enabling selective enrichment of the bacterial vehicle in tumors. After cell-specific entry, our lead candidate, ACTM-838, positively activates tumor-resident myeloid cells and delivers two synergistic payloads, optimized IL-15 and STING, unlocking a comprehensive and durable innate and adaptive anti-tumor immune response. With the ability to tailor our platform utilizing a range of payload combinations, we aim to achieve a new level of therapeutic impact for cancer patients across multiple tumor types.