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Takeda Oncology,, is a biopharmaceutical company based in Cambridge, Massachusetts.
AVROBIO`s vision is to bring personalized gene therapy to the world. We aim to prevent, halt or reverse disease throughout the body with a single dose of gene therapy designed to drive durable expression of therapeutic protein, even in hard-to-reach tissues and organs including brain, muscle and bone. Our ex vivo lentiviral gene therapy pipeline includes clinical programs in Fabry disease, Gaucher disease type 1 and cystinosis, as well as preclinical programs in Hunter syndrome, Gaucher disease type 3 and Pompe disease. AVROBIO is powered by our industry leading plato® gene therapy platform, our foundation designed to deliver gene therapy worldwide. We are headquartered in Cambridge, Mass., with an office in Toronto, Ontario.
Enzyvant is also advancing the development of RVT-802, an investigational tissue-based biologic therapy for the potential treatment of primary immune deficiency associated with complete DiGeorge Syndrome. RVT-802 has been granted orphan drug designation, Breakthrough Therapy designation, Regenerative Medicine Advanced Therapy designation, and pediatric rare disease designation by the U.S. Food and Drug Administration. Enzyvant anticipates a potential BLA filing for RVT-802 in the first half of 2018. Enzyvant plans to develop treatments for additional rare diseases with high unmet need.
Pacific Biosciences is developing an environment in which scientists can seamlessly integrate multiple data types from multiple sources and deploy advanced bioinformatics methods to elucidate the complexity of living systems. Therefore, we are committed
Our Mission: Change the world for patients with cardiovascular disease through bold and innovative science. MyoKardia is a clinical stage biopharmaceutical company pioneering a precision medicine approach to discover, develop and commercialize targeted therapies for the treatment of serious and neglected rare cardiovascular diseases. Our initial focus: treatment of heritable cardiomyopathies, a group of genetically-driven forms of heart failure that result from biomechanical defects in cardiac muscle contraction. We are a growing community of employees who enjoy working with and learning from each other. We collaborate in a way that ensures the growth of the company as well each employee individually. MyoKardia is committed to creating a culture that`s centered on our core values. We seek individuals who not only offer expertise in their respective fields, but can serve as cultural leaders and role models for our values.