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We are a genome editing company developing genetic medicines for the fight against rare pediatric diseases. GeneRide™ is a promoterless and nuclease-free approach to gene insertion which may improve the safety profile of AAV based gene therapies With GeneRide™, the therapeutic gene is site-specifically integrated into the genome allowing durable gene expression in dividing cells and mature tissues. Furthermore, therapeutic gene expression is regulated by the targeted locus so production can be limited to specific cell types.
Insmed is a global biopharmaceutical company focused on the needs of patients with rare diseases. Headquartered in Bridgewater, NJ, Insmed is a dynamic and rapidly growing company with a strong pipeline of drug candidates. At Insmed, we are on a mission to transform the lives of patients battling serious rare diseases. We are developing novel, targeted therapies to help serve the critical unmet needs of these patients. In concert with developing therapeutic solutions, we are committed to improving the entire patient experience. “Insmed is a team of talented and motivated professionals who are dedicated to making a difference in the lives of patients with rare diseases, said Will Lewis, president and CEO at Insmed.
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.
Palvella Therapeutics was founded with the single-minded goal of serving individuals suffering from serious rare genetic diseases with no approved treatments.
Clementia is a biopharmaceutical company that is focused on exploiting the science of novel retinoic acid receptor gamma agonists to address bone disease. Our lead program is palovarotene for Fibrodysplasia Ossificans Progressiva (FOP).