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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.
AavantiBio is a pre-clinical biopharmaceutical company committed to developing and commercializing novel gene transfer and gene editing therapies for the treatment of rare and ultra-rare genetic diseases. The company is rapidly building a diverse portfolio of gene transfer and gene editing therapies to target debilitating genetic diseases for which the unmet medical need is high for which there are typically no approved therapies treating the underlying disease. AavantiBio is headquartered in Cambridge, MA with additional locations in Gainesville, FL. We are looking for exceptional individuals who share our passion for developing novel therapies to treat rare diseases and advancing the field of gene transfer therapy. Developing genetic treatments for rare diseases is inspiring but challenging work and it takes dedication and courage which will make a meaningful impact on those who suffer from rare diseases. At AavantiBio, we will push each other to perform at our very best, and build a company that not only our employees are proud of, but also the rare disease community, because everyone knows we are trying to make a difference in patients` lives. Come join our team!
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Ambrx is a clinical stage biopharmaceutical company using an expanded genetic code technology platform to discover and develop next generation antibody drug conjugates (ADCs) and other engineered therapies to modulate the immune system. Ambrx is advancing a focused portfolio of clinical and preclinical programs designed to optimize efficacy and safety in multiple cancer indications. Specifically, ARX517, its proprietary antibody-drug conjugates (ADC) targeting the prostate-specific membrane antigen (PSMA) and ARX788, its proprietary ADC targeting HER2. Ambrx spun out of The Scripps Research Institute in 2003 and has several other product candidates involving ADCs and other aspects of Ambrx`s protein engineering technology.
Astra Zeneca Neuroscience is a Cambridge, MA-based company in the Healthcare, Pharmaceuticals, and Biotech sector.