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Selexys Pharmaceuticals is a Oklahoma City, OK-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
Akonni Biosystems is a Frederick, MD-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.
BillionToOne is a precision diagnostics company with the mission to make molecular diagnostics more accurate, efficient, and accessible. Our proprietary molecular counting platform can accurately count DNA molecules at the single-count level to help improve disease detection.
RDMD`s mission is to empower patients and communities to accelerate the development of treatments for rare diseases of all kinds.