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Ascendis Pharma A/S, a biopharmaceutical company, focuses on developing therapies for unmet medical needs. It offers SKYTROFA for treating patients with growth hormone deficiency (GHD). The company is also developing a pipeline of three independent endocrinology rare disease product candidates in clinical development, as well as focuses on advancing oncology therapeutic candidates. The company was incorporated in 2006 and is headquartered in Hellerup, Denmark.

Evotec SE engages in the discovery and development of new drugs for pharmaceutical and biotechnology companies. It operates through the following segments: EVT Execute and EVT Innovate. The EVT Execute segment provides stand-alone or integrated drug discovery solutions for collaborators targets and programmers on a typical fee-for-service basis or through a variety of commercial structures, which may include performance-based components, such as milestones and royalties. The EVT Innovate develops drug discovery projects, assets and platforms, both internally or through academic collaborations. The company was founded by Manfred Eigen, Karsten Henco, Ulrich Aldag, Freimut Leidenberger, Heinrich Maria Schulte, Rudolf Rigler, and Charles Weissmann on December 8, 1993 and is headquartered in Hamburg, Germany.

Perrigo Company plc provides over-the-counter health and wellness solutions to enhance individual well-being in the United States, Europe, and internationally. It operates through Consumer Self-Care Americas and Consumer Self-Care International segments. The company develops, manufactures, markets, and distributes self-care consumer products, such as upper respiratory products, including cough suppressants, expectorants, and sinus and allergy relief; nutrition products consisting of infant formulas and nutritional beverages; digestive health products, including antacids, anti-diarrheal, and anti-heartburn; pain and sleep-aids products comprising pain relievers and fever reducers; and oral care products, which include toothbrushes, toothbrush replacement heads, floss, flossers, whitening products, and toothbrush covers. It also offers healthy lifestyle products, such as smoking cessation, well-being, and weight management products; skin care products consisting of dermatological care, scar management, lice treatment, and other products for various skin conditions; women's health products comprising feminine hygiene and contraceptives; vitamins, minerals, and supplements; rare diseases business; and other miscellaneous self-care products. The company sells its products under the Compeed, Dr. Fresh, Firefly, Good Sense, Good Start, Mederma, Nasonex, Plackers, Prevacid24HR, REACH, Rembrandt, Steripod, Opill, Solpadeine, Coldrex, Physiomer, NiQuitin, ACO, ellaOne, Compeed Stops, XLS, Arterin, Davitamon, Apiserum, Abtei, and Nicorette brands. It also offers contract manufacturing services. The company sells its products through retail drug, supermarket, and mass merchandise chains; e-commerce stores; wholesalers; pharmacies; drug and grocery retailers; and para-pharmacies. The company was formerly known as Perrigo Company and changed its name to Perrigo Company plc in December 2013. Perrigo Company plc was founded in 1887 and is headquartered in Dublin, Ireland.

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, Japan, Europe, and internationally. 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 treating long-chain fatty acid oxidation disorders; and Evkeeza (evinacumab) for the treatment of homozygous familial hypercholesterolemia. The company's products candidatures include 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; UX143, a human monoclonal antibody for the treatment of osteogenesis imperfecta; GTX-102, an antisense oligonucleotide for the treatment of Angelman syndrome; 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; UX701, 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.; Bayer Healthcare LLC; GeneTx; Mereo; University of Pennsylvania; Arcturus Therapeutics Holdings Inc.; Solid Biosciences Inc.; Regeneron; Abeona; and Daiichi Sankyo Co., Ltd. The company was incorporated in 2010 and is headquartered in Novato, California.

Sarepta Therapeutics, Inc., a commercial-stage biopharmaceutical company, focuses on the discovery and development of RNA-targeted therapeutics, gene therapies, and other genetic therapeutic modalities for the treatment of rare diseases. It offers EXONDYS 51 injection to treat duchenne muscular dystrophy (duchenne) in patients with confirmed mutation of the dystrophin gene that is amenable to exon 51 skipping; VYONDYS 53 for the treatment of duchenne in patients with confirmed mutation of the dystrophin gene that is amenable to exon 53 skipping; AMONDYS 45 for the treatment of duchenne in patients with confirmed mutation of the dystrophin gene; and ELEVIDYS, an adeno-associated virus based gene therapy for the treatment of ambulatory pediatric patients aged 4 through 5 years with duchenne with a confirmed mutation in the duchenne gene. The company is also developing SRP-5051, a peptide conjugated PMO that binds exon 51 of dystrophin pre-mRNA; and SRP-9003, a limb-girdle muscular dystrophies gene therapy program. It has collaboration and license agreements with F. Hoffman-La Roche Ltd; Nationwide Children's Hospital; Genevant Sciences; University of Florida; Dyno Therapeutics; Hansa Biopharma; Duke University; Genethon; and StrideBio. The company was incorporated in 1980 and is headquartered in Cambridge, Massachusetts.