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Arrowhead Pharmaceuticals, Inc. develops medicines for the treatment of intractable diseases in the United States. The company's products in pipeline includes Plozasiran, which is in Phase 2b and one Phase 3 clinical trial to treat hypertriglyceridemia, mixed dyslipidemia, and chylomicronemia syndrome; Zodasiran that is in Phase 2b clinical trial for the treatment of dyslipidemia and hypertriglyceridemia; ARO-PNPLA3, which is in Phase 1 clinical trial to treat patients with non-alcoholic steatohepatitis; ARO-RAGE that is in Phase 1/2a clinical trial to treat inflammatory pulmonary conditions; and ARO-MUC5AC, which is in Phase 1/2a clinical trial to treat muco-obstructive pulmonary diseases. It also develops ARO-MMP7 that is in Phase 1/2a clinical trial for treatment of idiopathic pulmonary fibrosis; ARO-DUX4 for the treatment of facioscapulohumeral muscular dystrophy; ARO-SOD1 for the potential treatment of amyotrophic lateral sclerosis; and ARO-C3, which is in Phase 1/2a clinical trial for the treatment of patients with various complement mediated or complement associated renal diseases. In addition, the company is involved in the development of JNJ-3989, which is in Phase 2 clinical trial to treat chronic hepatitis B virus infection; Olpasiran that is in Phase 3 clinical trial to reduce the production of apolipoprotein A; GSK-4532990 that is in phase 2 clinical trial to treat liver diseases; HZN-457, which is in phase 1 clinical trial to treat uncontrolled gout; and Fazirsiran that is in Phase 3 clinical trial for the treatment for liver disease associated with alpha-1 antitrypsin deficiency. Arrowhead Pharmaceuticals, Inc. has license and research collaboration agreements with Janssen Pharmaceuticals, Inc.; Takeda Pharmaceutical Company Limited; Horizon Therapeutics Ireland DAC; Amgen Inc.; and Glaxosmithkline Intellectual Property (No. 3) Limited. The company was founded in 2003 and is headquartered in Pasadena, California.

CureVac N.V., a clinical-stage biopharmaceutical company, focuses on developing various transformative medicines based on messenger ribonucleic acid (mRNA). It is developing prophylactic vaccines, such as mRNA-based vaccine candidates CV2CoV, which is in Phase 1 clinical trial against SARS-CoV-2; CV7202, a prophylactic mRNA-based vaccine, which has completed Phase 1 clinical trial for rabies virus glycoprotein; and CVSQIV, which is in Phase 1 clinical trial against influenza, as well as vaccine for lassa fever, yellow fever, respiratory syncytial virus, rotavirus, malaria, and universal influenza. The company also develops RNA-based cancer therapies, including CV8102, which is in Phase 1 clinical trial for treating cutaneous melanoma and adenoidcystic carcinoma, as well as squamous cell cancer of skin, head, and neck. CureVac N.V. was founded in 2000 and is headquartered in Tübingen, Germany.

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.

Jazz Pharmaceuticals plc identifies, develops, and commercializes pharmaceutical products for unmet medical needs in the United States, Europe, and internationally. The company offers Xywav for cataplexy or excessive daytime sleepiness (EDS) with narcolepsy and idiopathic hypersomnia; Xyrem to treat cataplexy or EDS with narcolepsy; Epidiolex for seizures associated with Lennox-Gastaut and Dravet syndromes, or tuberous sclerosis complex; Zepzelca to treat metastatic small cell lung cancer, or with disease progression on or after platinum-based chemotherapy; Rylaze for acute lymphoblastic leukemia or lymphoblastic lymphoma; Enrylaze to treat acute lymphoblastic leukemia and lymphoblastic lymphoma; Defitelio to treat severe hepatic veno-occlusive disease; and Vyxeos for newly-diagnosed therapy-related acute myeloid leukemia. It also develops Zanidatamab to treat HER2-expressing gastroesophageal adenocarcinoma (GEA), and patients with HER2-expressing metastatic GEA; Zepzelca for the treatment of patients with select relapsed/refractory solid tumors based on limited response in three solid tumor cohorts; JZP815, a pan-RAF kinase inhibitor that targets components of the mitogen-activated protein kinase; JZP898, a conditionally-activated interferon alpha molecule; Epidiolex to treat LGS, DS, and TSC; Suvecaltamide to treat parkinson's disease tremor; JZP150, a fatty acid amide hydrolase inhibitor program to treat post-traumatic stress disorder; and JZP441 to treat narcolepsy, IH, and other sleep disorders. The company has licensing and collaboration agreements with XL-protein GmbH to extend the plasma half-life of selected asparaginase product candidates; Redx Pharma plc for preclinical activities Ras/Raf/MAP kinase pathway program; and Autifony Therapeutics Limited on discovering and developing drug candidates targeting two different ion channel targets associated with neurological disorders. The company was incorporated in 2003 and is headquartered in Dublin, Ireland.

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.