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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.
Synthetica Bio is a privately held healthcare technology company based in Laguna Beach, California that is developing the next generation of safe and secure generative artificial intelligence (GenAI) solutions for the biopharma industry. The Synthetica Bio platform leverages and optimizes multiple large language models (LLMs) and vector databases to ingest real-time data streams and make actionable knowledge directly available to biopharma discovery, clinical, and commercial teams. The platform enables healthcare professionals to rapidly leverage the power of GenAI within compliance of data security standards required by the industry.
Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious and life-threatening diseases.
eGenesis is revolutionizing the field of transplantation with an unparalleled, multiplexed gene editing platform for the development of human-compatible organs, tissues and cells. Harnessing the latest gene-editing techniques, eGenesis has the capability to solve the global organ crisis by providing an alternative to allotransplantation. eGenesis is uniquely positioned to reinvigorate the field of xenotransplantation by addressing both the key virology and immunology hurdles that have impeded its advancement to date and provide commercially-viable products to save and enhance the lives of patients in need.
Access Genetics LLC is one of the leading companies in the Healthcare, Pharmaceuticals, and Biotech sector.