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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.
Replimune is creating a new class of oncolytic immunotherapy designed to ignite a powerful immune response to treat cancer and vaccinate against future relapse. Replimune`s mission is to revolutionize cancer treatment with therapies designed to activate a powerful and durable full-body anti-tumor response. Replimune is pioneering the development of novel tumor-directed oncolytic immunotherapies. We imagine a world where cancer is a curable disease.
Eterna Therapeutics is developing breakthrough mRNA cell engineering technologies to repair cellular dysfunction and treat a range of therapeutic indications. We and our strategic partners are advancing innovative nucleic acid and cell therapies that offer the hope of radically improving the health outcomes of patients with high unmet medical needs. We are committed to creating a world in which patients and their families have access to effective, life-changing treatments for serious illnesses.
Cue Biopharma™ is an innovative immunotherapy company developing a novel, proprietary class of biologics engineered to selectively modulate the human immune system to treat a broad range of cancers and autoimmune disorders. We design biologics to engage and modulate the activity of disease-associated T cells in the patient`s body, offering significant therapeutic advantages while potentially minimizing or eliminating unwanted side effects. We believe our biologics allow us to target antigen-specific T cell populations in a variety of indications using a simple peptide exchange within previously-validated drug frameworks developed from the Cue Biologics Platform™. This flexibility could truncate the drug selection and development process, moving effective therapeutics from discovery to clinical validation more rapidly and cost efficiently than current industry standard timelines and costs. Headquartered in Kendall Square, Cambridge, MA, we are led by an experienced management team and scientific and clinical advisory board (SAB/CAB) with deep expertise in the design and clinical development of protein biologics, immunology and immuno-oncology.
Nautilus Environmental is a San Diego, CA-based company in the Healthcare, Pharmaceuticals, and Biotech sector.