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Developing new treatments for rare and orphan neurological conditions.
BioHarp UNI US is a Sun Valley, CA-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
PTC is a science-driven, global biopharmaceutical company focused on the discovery, development and commercialization of clinically differentiated medicines that provide benefits to patients with rare disorders. PTC`s ability to globally commercialize products is the foundation that drives investment in a robust and diversified pipeline of transformative medicines and our mission to provide access to best-in-class treatments for patients who have an unmet medical need.
Absci is the AI-powered synthetic biology company unlocking the potential of proteins as the next generation of therapeutics. We built our Integrated Drug Creation™ Platform to accelerate discovery of novel biotherapeutic drug candidates and generation of the cell lines to manufacture them in a single efficient process. Biotech and pharma innovators partner with us to develop new protein-based drugs, including those that may be impossible to make with other technologies. Our goal is to enable the creation of better medicines by Translating Ideas into Drugs.
Senda Biosciences, Inc. is uniquely positioned to transform human health by harnessing millions of years of evolution to program targeted, potent and tunable medicines. Nature has provided the codes to program human cells – both within the cell (mRNA), and, remarkably, to the cell - using what surrounds it. The trillions of non-human cells in the human ecosystem have evolved natural nanoparticles that precisely shuttle biomolecules into human cells, providing the missing pieces to fully unlock programmable medicines. Senda`s proprietary platform includes the first-ever atlas of nature-derived programmable systems at the molecular level and across all kingdoms of life – accessing the entire code provided by nature required to program cells. With this platform, Senda is developing a new class of SendRNA™ medicines. The unique properties of these medicines create new frontiers for mRNA therapeutics and vaccines for infectious, genetic, autoimmune, and metabolic diseases and oncology indications – with further potential to transform the gene editing and protein-based therapy landscapes as well. Based in Cambridge, MA, Senda was founded by Flagship Pioneering.