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Rgenta Therapeutics is developing a pipeline of oral, small-molecule RNA-targeting medicines with an initial focus on oncology and neurological disorders. Our proprietary platform mines the massive genomics data to identify targetable RNA processing events and design small-molecule glues to modulate the interactions among the spliceosome, regulatory proteins, and RNAs
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.
SignalChem Lifesciences Corporation is a privately owned biopharmaceutical company specializing in the discovery and development of kinase inhibitors as innovative therapies for cancer, inflammation and neurological diseases. The Company has a robust kinase platform encompassing 450 kinase targets and assays for the development and commercialization of unique therapies in many diseases. This vast resource empowers SLC’s scientists to identify novel drug candidates against kinase targets of interest. Another key part of SLC’s kinase drug development platform is KineCore, a kinase focused small molecule library that was designed and assembled in-house.
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.
Stem Centrx is a South San Francisco, CA-based company in the Healthcare, Pharmaceuticals, and Biotech sector.