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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.
Exonics Therapeutics was launched in February 2017 to advance the research of our scientific founder, Dr. Eric Olson and his laboratory at the University of Texas Southwestern Medical Center (UTSW) to develop treatments for patients with neuromuscular diseases. Dr. Olson is one of the world’s leading experts in the study of muscle cells and the application of gene editing to treat these types of diseases. In particular, Dr. Olson’s laboratory has used adeno-associated virus (AAV) to deliver a CRISPR/Cas9 technology that can identify and repair exon mutations to restore the production of dystrophin, a protein that helps stabilize and protect muscle fibers. Dystrophin is the protein missing in boys with Duchenne. The loss of dystrophin causes Duchenne.
ALK-Abello A/S is a Denmark-based company active within the pharmaceutical industry. It is primarily engaged in the development of pharmaceutical products that target the cause of allergy. The Company is focused on allergy vaccination. Its product
Incyte is a biopharmaceutical company focused on the discovery, development and commercialization of innovative medicines that will meet serious unmet medical needs in oncology and other diseases. Incyte is a company driven by innovation, and we believe that investment in cutting-edge science and the rigorous pursuit of R&D excellence can translate into new medicines for serious unmet medical needs that positively impact patients` lives. Incyte has a diverse and growing portfolio of first-in-class and best-in-class product candidates, including both small and large molecules. Incyte is headquartered in Wilmington, Delaware with operations in 10 European countries and Japan. Our more than 1,000 employees are led by distinguished executives with proven records of success. We are proud to have been recognized by Forbes as one of the World`s Most Innovative Companies for each of the past three years.
Rakuten Aspyrian, Inc is a privately funded clinical stage biotechnology company with offices in the USA, Japan and Germany.