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BioClin Therapeutics, Inc. is a privately-held biotechnology developing biologics to address medical conditions where there is significant unmet need. Our lead program is a human monoclonal antibody, B-701, that targets and blocks the activity of FGFR3 (fibroblast growth factor receptor 3) and is the most advanced FGFR3-specific antagonist in development. B-701 has been tested in two Phase 1 clinical trials involving 40 patients and demonstrated potential efficacy in patients with metastatic bladder cancer who were receiving it as 2nd line therapy.
Liquidia Technologies is a biopharmaceutical company transforming the development of new therapies by precisely engineering drug particles.
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.
Bioskryb is a venture-backed startup bringing new solutions for cellular heterogeneity, in both the research and clinical space studies, using a proprietary genome amplification system.
Sana Biotechnology is focused on utilizing engineered cells as medicines for patients. The ability to modify genes and use cells as medicines will be one of the most important advances in healthcare over the next several decades. Sana is building differentiated capabilities across the spectrum of cell and gene therapy. Three aspirations drive Sana as we look to discover treatments for patients with poor outcomes or currently untreatable diseases. The first is the ability to repair and control the genes in any cell in the body. We are advancing novel delivery technologies with the goal of being able to deliver any payload to any cell in a specific, predictable, and repeatable manner, paving the way for next-generation in vivo gene therapy. Next is the ability to differentiate pluripotent stem cells ex vivo into immune-cloaked functional cells with the aspiration of being able to replace any missing or damaged cells in the body. Last is a belief we can enable broader access to our therapies through focusing on scalable manufacturing solutions, the cost of manufacturing, and aligning with key stakeholders. Sana launched in early 2019 and has approximately two hundred employees in Seattle, Cambridge, and South San Francisco. In 2019, we raised capital to enable our broad vision, began to acquire technology and establish an IP estate across cell and gene therapy, and made significant progress validating our cell and gene therapy platforms. Growing from this foundation, we intend to push forward our platforms and product pipeline in 2020 and beyond.