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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.

BridgeBio Pharma, Inc., a commercial-stage biopharmaceutical company, discovers, creates, tests, and delivers transformative medicines to treat patients who suffer from genetic diseases and cancers. Its products in development programs include AG10, a next-generation oral small molecule near-complete TTR stabilizer that is in Phase 3 clinical trial for the treatment of TTR amyloidosis, or transthyretin amyloid cardiomyopathy (ATTR-CM); low-dose infigratinib, an oral FGFR1-3 selective tyrosine kinase inhibitor, which is in Phase 3 double-blinded, placebo-controlled pivotal study for the treatment option for children with achondroplasia; and BBP-631, an AAV5 gene transfer product candidate that is in Phase 1/2 clinical trial for the treatment of congenital adrenal hyperplasia, or CAH, driven by 21-hydroxylase deficiency, or 21OHD. The company also develops Encaleret, a small molecule antagonist of the calcium sensing receptor, or CaSR, which is in phase 3 clinical trial for treating autosomal dominant hypocalcemia type 1, or ADH1; and BBP-418, a glycosylation substrate pro-drug that is in Phase 3 clinical trial for treating limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9). In addition, it engages in developing products for mendelian, oncology, and gene therapy diseases. BridgeBio Pharma, Inc. has license and collaboration agreements with the Leland Stanford Junior University; and Leidos Biomedical Research, Inc. The company was founded in 2015 and is headquartered in Palo Alto, California.

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.

Global Blood Therapeutics, Inc., a biopharmaceutical company, engages in the discovery, development, and delivery of treatments for underserved patient communities with sickle cell disease (SCD). The company offers Oxbryta tablets, an oral, once-daily therapy for SCD. It also evaluates the safety and pharmacokinetics of single and multiple doses of Oxbryta in a Phase II a clinical trial of adolescent and pediatric patients with SCD. In addition, the company is developing its lead product candidate inclacumab, novel human monoclonal antibody, which is in Phase III a clinical trial to reduce the incidence of painful vaso-occlusive crises, and resulting hospital admissions; and GBT601, a hemoglobin polymerization inhibitor that is in Phase I development. It has a license and collaboration agreement with Syros Pharmaceuticals, Inc. to discover, develop, and commercialize therapies for SCD and beta thalassemia; and Sanofi S.A. to two early-stage research programs in SCD. Global Blood Therapeutics, Inc. was incorporated in 2011 and is headquartered in South San Francisco, California.

United Therapeutics Corporation, a biotechnology company, engages in the development and commercialization of products to address the unmet medical needs of patients with chronic and life-threatening diseases in the United States and internationally. The company offers Tyvaso DPI, an inhaled dry powder via pre-filled and single-use cartridges; Tyvaso, an inhaled solution via ultrasonic nebulizer; Remodulin (treprostinil) injection to treat patients with pulmonary arterial hypertension (PAH) to diminish symptoms associated with exercise; Orenitram, a tablet dosage form of treprostinil, to delay disease progression and improve exercise capacity in PAH patients; and Adcirca, an oral PDE-5 inhibitor to enhance the exercise ability in PAH patients. It also markets and sells Unituxin (dinutuximab) injection, a monoclonal antibody for treating high-risk neuroblastoma; and Remunity Pump, which contains a pump and separate controller for Remodulin. In addition, the company engages in developing RemoPro and Ralinepag for the treatment of PAH; Aurora-GT, a gene therapy product to rebuild the blood vessels in the lungs; and Nebulized Tyvaso, for the treatment of idiopathic pulmonary fibrosis, as well as xenografts, which are development-stage organ products. It has licensing and collaboration agreements with DEKA Research & Development Corp. to develop a semi-disposable system for the subcutaneous delivery of treprostinil; MannKind Corporation to develop and license treprostinil inhalation powder and the Dreamboat device; and Arena Pharmaceuticals, Inc. to develop Ralinepag. The company was incorporated in 1996 and is headquartered in Silver Spring, Maryland.