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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.
Quintiles and IMSHealth have merged together to form the new QuintilesIMS. Each wanting to bring something new to customers, we deliver integrated information and technology solutions to drive healthcare forward. QuintilesIMS has approximately 50,000 employees conducting operations in more than 100 countries, dedicated to helping our clients improve their clinical, scientific and commercial results. As a global leader in protecting individual patient privacy, QuintilesIMS uses healthcare data to deliver critical, real-world disease and treatment insights.
Triplet Therapeutics is a biotechnology company developing transformational treatments for patients with repeat expansion disorders (REDs) -- a group of more than 50 known genetic diseases including Huntington`s disease, myotonic dystrophy, spinocerebellar ataxias, fragile X syndrome, and familial amyotrophic lateral sclerosis (ALS) -- leveraging insights from patient genetics. Triplet designs and develops potential therapeutics for REDs using its proprietary thRED Engine, which enables the Company to develop a single oligonucleotide targeting the DNA Damage Response (DDR) pathway to potentially treat multiple REDs. Triplet is backed by investments from Atlas Venture, MPM Capital and Pfizer Ventures, along with Invus, Partners Innovation Fund and Alexandria Venture Investments.
Total Repair Express is a Hillsborough, NJ-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
Vor Biopharma is pioneering engineered hematopoietic stem cells (eHSCs) to dramatically change the treatment paradigm for hematological cancers. Vor`s eHSCs are designed to generate healthy, fully functional cells with specific advantageous modifications, for example to protect the blood and bone marrow from the toxic effects of antigen-targeted therapies, protecting healthy cells from depletion but leaving tumor cells vulnerable. Vor`s platform could potentially be used to change the treatment paradigm of both hematopoietic stem cell transplants and antigen-targeted therapies such as antibody drug conjugates, bispecific antibodies and CAR-T cell treatments. A proof-of-concept study for Vor`s lead program has been published in Proceedings of the National Academy of Sciences. Vor is based in Cambridge, Mass. and has a broad intellectual property base including in-licenses from Columbia University, where foundational work was conducted by inventor and Vor Scientific Board Chair Siddhartha Mukherjee, MD, DPhil. Vor was founded by Dr. Mukherjee and PureTech Health and is supported by leading investors including 5AM Ventures and RA Capital Management, Johnson & Johnson Innovation – JJDC, Inc. (JJDC), Novartis Institutes for BioMedical Research and Osage University Partners.