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MOLN vs RARE

Molecular Partners AG American Depositary Shares and Ultragenyx Pharmaceutical Inc.

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Molecular Partners AG American Depositary Shares

Molecular Partners AG, a clinical-stage biotechnology company, designs and develops designed ankyrin repeat proteins therapeutics for the treatment of oncology diseases in Switzerland. Its product pipeline comprises MP0317, which is in Phase 1 clinical trial for the treatment of advanced solid tumors; MP053, novel tetra-specific T cell-engaging DARPin, which is phase 1 clinical trial for the treatment of acute myeloid leukemia; and MP0712, a 212Pb Radio-DARPin therapy (RDT) candidate targeting the tumor-associated protein delta-like ligand 3 (DLL3) which is in preclinical trial to treat SCLC and neuroendocrine tumors. It also researches for Switch-DARPin T cell engager to treat CD3 x costim x; and Radio-DARPin Therapy to treat ovarian disease. In addition, the company develops MP0621, a molecular partners' first Switch-DARPin program, which is in preclinical trial designed to induce killing of hematopoietic stem cells (HSCs) as a next-generation conditioning regimen for HSC transplantation (HSCT); and Radio-DARPin Therapy (RDT) platform, a delivery system for effective and selective delivery of radioactive payloads to solid tumors. It has collaboration agreement with Orano Med SAS to develop novel Radio-DARPin therapeutics and an additional six targeted alpha therapeutics candidates. Molecular Partners AG was incorporated in 2004 and is headquartered in Schlieren, Switzerland.

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Ultragenyx Pharmaceutical Inc.

Ultragenyx Pharmaceutical Inc., a biopharmaceutical company, focuses on the identification, acquisition, development, and commercialization of novel products for the treatment of rare and ultra-rare genetic diseases in North America, Latin America, Europe, the Middle East, Africa, and the Asia-Pacific. Its biologic products include Crysvita (burosumab), an antibody targeting fibroblast growth factor 23 for the treatment of X-linked hypophosphatemia, as well as tumor-induced osteomalacia; Mepsevii, an enzyme replacement therapy for the treatment of children and adults with Mucopolysaccharidosis VII; Dojolvi for the treatment of long-chain fatty acid oxidation disorders; and Evkeeza (evinacumab) for the treatment of homozygous familial hypercholesterolemia. The company's products candidates that are in Phase 3 clinical trials include UX143, a human monoclonal antibody for the treatment of osteogenesis imperfecta; UX111, an AAV9 gene therapy product candidate for the treatment of patients with Sanfilippo syndrome type A, or MPS IIIA, a rare lysosomal storage disease; DTX401, an adeno-associated virus 8 (AAV8) gene therapy clinical candidate for the treatment of patients with glycogen storage disease type Ia; DTX301, an AAV8 gene therapy for the treatment of patients with ornithine transcarbamylase; and GTX-102, an antisense oligonucleotide for the treatment of Angelman syndrome. It also develops UX701 that is in Phase 2 clinical trial for the treatment of Wilson disease; and UX053 for the treatment of glycogen storage disease type III. Ultragenyx Pharmaceutical Inc. has collaboration and license agreement with Kyowa Kirin Co., Ltd.; Saint Louis University; Baylor Research Institute; REGENXBIO Inc.; GeneTx; Mereo; University of Pennsylvania; Solid Biosciences Inc.; Regeneron; Abeona; and Arcturus Therapeutics Holdings Inc. The company was incorporated in 2010 and is headquartered in Novato, California.

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