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Showing 1 - 26 of 26 results found.
| Company | Activity | Company Description | Location | |
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Company Aceragen Inc. | Activity: Research and Development | Company Description Aceragen is a clinical-stage biopharmaceutical company developing therapeutics for rare and orphan pulmonary and rheumatic diseases with high unmet medical need. | Location Research Triangle Park |
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Company AMO Pharma Ltd. | Activity: Research and Development | Company Description AMO Pharma is developing new treatments for serious and debilitating diseases including rare genetic disorders. | Location Durham |
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Company Artiam Bio Inc. | Activity: Research and Development | Company Description Artiam Bio is targeting the endocannabinoid system to treat diseases using pharmacology and medicinal chemistry approaches. Artiam Bio is initially focused on non-alcoholic and alcoholic liver diseases, Prader Willi syndrome and cannabis use disorder. | Location Morrisville |
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Company AskBio | Activity: Research and Development | Company Description Asklepios BioPharmaceutical (AskBio) uses adeno-associated virus (AAV) technology to develop and manufacture gene therapies for rare diseases and other genetic disorders. | Location Research Triangle Park |
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Company Astellas Gene Therapies | Activity: Production and Manufacturing | Company Description Astellas Gene Therapies manufactures AAV gene therapy products for rare diseases. | Location Sanford |
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Company Baebies Inc. | Activity: Production and Manufacturing | Company Description Baebies is a medical device company that develops and commercializes products to enable early disease detection and comprehensive diagnosis for children and adults. | Location Durham |
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Company BridgeBio Gene Therapy | Activity: Research and Development | Company Description BridgeBio Gene Therapy is developing a gene therapy for the treatment of a monogenic disease that presents in childhood. | Location Raleigh |
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Company Chiesi USA Inc. | Activity: Research and Development | Company Description Chiesi USA, the U.S. affiliate of Chiesi Farmaceutici S.p.A, develops and commercializes products in the therapeutic areas of rare disease, respiratory, cardiovascular, neonatology and cystic fibrosis. | Location Cary |
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Company Collaborations Pharmaceuticals Inc. | Activity: Contract Research and Testing | Company Description Collaborations Pharmaceuticals develops drugs for rare and neglected diseases using machine learning software, providing preclinical and clinical expertise to rare disease foundations, academic scientists and companies developing clinical candidates. Collaborations also provides machine learning expertise and software to consumer product and agrochemical companies. | Location Raleigh |
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Company Couragen Biopharmaceutics LLC | Activity: Research and Development | Company Description Couragen Biopharmaceutics develops gene and protein therapy products for preclinical and clinical use for the treatment of genetic and chronic diseases. Couragen also provides custom adeno-associated virus vectors as a tool for laboratory research. | Location Chapel Hill |
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Company DuraBull Pharmaceuticals Inc. | Activity: Research and Development | Company Description DuraBull Pharmaceuticals develops medical therapies for patients with hereditary aortopathy. | Location Durham |
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Company Enzerna Biosciences Inc. | Activity: Research and Development | Company Description Enzerna Biosciences is a pre-clinical stage company that is leveraging its proprietary RNA editing technology to develop long-term curative gene therapies for rare genetic disorders such as Huntington's Disease and Myotonic Dystrophy. | Location Morrisville |
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Company Enzyvant Inc. (Cary) | Activity: Research and Development | Company Description Enzyvant, a subsidiary of Sumitovant Biopharma, develops regenerative therapeutics for rare diseases such as congenital athymia. The Cary location is their headquarters. | Location Cary |
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Company Genencine Therapeutics LLC | Activity: Research and Development | Company Description Genencine Therapeutics develops mRNA vaccines for the prevention and treatment of cancer, viral diseases and autoimmune diseases, as well as mRNA therapeutic drugs for genetic diseases. | Location Durham |
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Company Glycomine Inc. | Activity: Research and Development | Company Description Glycomine is developing orphan drugs for serious rare disorders of metabolism and protein misfolding. | Location Chapel Hill |
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Company Jaguar Gene Therapy LLC (Analytical Development) | Activity: Research and Development | Company Description Jaguar Gene Therapy develops gene therapies for patients suffering from severe genetic diseases. The company's analytical development lab is located in Durham. | Location Durham |
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Company Jaguar Gene Therapy LLC (Process Sciences Lab) | Activity: Research and Development | Company Description Jaguar Gene Therapy develops gene therapies for patients suffering from severe genetic diseases. The company's process sciences lab is located in Cary. | Location Cary |
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Company Juno Metabolomics LLC | Activity: Production and Manufacturing | Company Description Juno Metabolomics is developing next-generation metabolomics for precision medicine. | Location Chapel Hill |
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Company MitoRainbow Therapeutics Inc. | Activity: Research and Development | Company Description MitoRainbow is developing treatments for TK2 deficiency, a mitochondrial disease that manifests as severe myopathy. | Location Cary |
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Company ML Bio Solutions Inc. | Activity: Research and Development | Company Description ML Bio Solutions is developing an oral treatment for patients with limb girdle muscular dystrophy 2i. | Location Charlotte |
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Company NeuroGT Inc. | Activity: Research and Development | Company Description NeuroGT is developing gene therapy products to treat rare neurogenetic diseases. | Location Chapel Hill |
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Company Novartis Gene Therapies | Activity: Production and Manufacturing | Company Description Novartis Gene Therapies develops and commercializes gene therapies for patients and families devastated by rare and life-threatening neurological genetic diseases. The Durham site is Novartis Gene Therapies' manufacturing operations. | Location Durham |
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Company QOL Medical LLC | Activity: Sales and Operations | Company Description QOL Medical commercializes existing and complementary new orphan products for metabolic disease and pediatric gastroenterology. | Location Raleigh |
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Company Resilience (Durham) | Activity: Production and Manufacturing | Company Description National Resilience manufactures viral vectors, a component of cell and gene therapies. | Location Durham |
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Company Sobi (Durham) | Activity: Research and Development | Company Description Sobi develops treatments for orphan diseases with a focus on inflammation and genetic diseases. | Location Durham |
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Company Tellus Therapeutics Inc. | Activity: Research and Development | Company Description Tellus Therapeutics is developing novel small molecules derived from human maternal breast milk for the treatment of newborns with perinatal brain injury. | Location Hillsborough |