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Sigilon Therapeutics’ mission is to create immune-protected, engineered human cells that restore normal physiology in a wide range of diseases without generating fibrosis or immune rejection, liberating patients from challenges associated with serious chronic diseases. We're based in Cambridge, Mass.
VinCro Clinical Management Services is a Ottawa, ON-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.
Clinical stage gene therapy company using a constellation of proprietary AAV vector technologies to develop novel treatments for central nervous system and lysosomal storage diseases
Alexion is a global biopharmaceutical company focused on developing and delivering life-transforming therapies for patients with devastating and rare diseases. Our shared purpose to serve patients in need drives us to find answers, change the world, and create a legacy. Alexion`s three highly innovative therapies treat patients with four severe and ultra-rare diseases, and we are committed to developing additional therapies that have the potential to transform patients` lives. Today, Alexion is advancing the most robust rare disease pipeline in the biotech industry, which, in addition to our complement and metabolic clinical programs, includes more than 30 diverse preclinical programs across a range of therapeutic modalities. Alexion has more than 3,000 employees serving patients in 50 countries. Our global headquarters and research operations are based in New Haven, Connecticut and our EMEA headquarters are in Zürich, Switzerland. We also have global supply chain and operations headquarters in Ireland, manufacturing facilities in the United States, and local and regional operations in countries around the world. Alexion was ranked number 5 by Science magazine in its Top Employer Survey and has also been ranked as one of the top companies on the Forbes "World`s Most Innovative Companies" list every year since 2012.