AC Immune SA
AC Immune SA, a clinical stage biopharmaceutical company, discovers, designs, and develops medicines, and therapeutic and diagnostic products for the prevention and treatment of neurodegenerative diseases associated with protein misfolding. The company's SupraAntigen and Morphomer platforms are designed to generate biologics and small molecules, which selectively interact with misfolded proteins that are common in a range of neurodegenerative diseases. It develops Crenezumab, a humanized, conformation specific monoclonal antibody used to slow Alzheimer's disease (AD) progression; and Semorinemab, an investigational monoclonal anti Tau antibody that targets the N-terminal portion of the Tau protein and is designed to bind to Tau and slow its spread between neurons for the treatment of AD. The company is also developing ACI-24.060 for the treatment of down syndrome related AD; ACI-7104.056 to treat Parkinson's disease; and ACI-35.030, designs to stimulate a patient's immune system to produce antibodies against pathological phosphorylated Tau. In addition, it develops diagnostic programs consisting of PI-2620, a Tau diagnostic for AD, as well as non-AD Tauopathies; and ACI-12589, a-sync PET tracer that supports the differential diagnosis of multiple system atrophy from other neurodegenerative disease. Further, the company is researching and developing Morphomer Tau aggregation inhibitors to evaluate candidates in AD; Morphomer a-syn to compete with small molecule inhibitors of a-syn aggregation; and NeuroOrphan Tauopathies. Additionally, it generates discovery and preclinical stage molecules targeting range of neurodegenerative diseases, which include diagnostics targeting TDP-43, alpha-synuclein, and NLRP3. The company has license agreements and collaborations with Genentech, Inc.; Janssen Pharmaceuticals, Inc.; Life Molecular Imaging SA; and Eli Lilly and Company. AC Immune SA was incorporated in 2003 and is headquartered in Lausanne, Switzerland.
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BioMarin Pharmaceutical Inc., a biotechnology company, engages in the development and commercialization of therapies for life-threatening rare diseases and medical conditions in the United States, Europe, Latin America, the Middle East, the Asia Pacific, and internationally. The company's products include VIMIZIM, an enzyme replacement therapy for the treatment of mucopolysaccharidosis (MPS) IV type A, a lysosomal storage disorder; VOXZOGO, a once daily injection analog of c-type natriuretic peptide (CNP) for the treatment of achondroplasia; NAGLAZYME, a recombinant form of N- acetylgalactosamine 4-sulfatase for patients with MPS VI; and PALYNZIQ, a PEGylated recombinant phenylalanine (Phe) ammonia lyase enzyme delivered through subcutaneous injection to reduce blood Phe concentrations. It also develops BRINEURA, a recombinant human tripeptidyl peptidase 1 for the treatment of patients with ceroid lipofuscinosis type 2, a form of Batten disease; ALDURAZYME, a purified protein designed to be identical to a naturally occurring form of the human enzyme alpha-L-iduronidase; KUVAN, a proprietary synthetic oral form of 6R-BH4 that is used to treat patients with phenylketonuria, an inherited metabolic disease; and ROCTAVIAN, an adeno associated virus vector for the treatment of severe hemophilia A. The company's products under development include BMN 333, a longer-acting CNP for the treatment of multiple growth disorders, such as achondroplasia and hypochondroplasia; BMN 349, an oral therapeutic for the treatment of liver disease associated with alpha-1 antitrypsin deficiency; and BMN 351, an oligonucleotide for the treatment of duchenne muscular dystrophy. It serves specialty pharmacies, hospitals, non-U.S. government agencies, distributors, and pharmaceutical wholesalers. The company has license and collaboration agreements with Catalyst Pharmaceutical Partners, Inc., and Ares Trading S.A. The company was incorporated in 1996 and is based in San Rafael, California.
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