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Amphastar Pharmaceuticals, Inc., a bio-pharmaceutical company, develops, manufactures, markets, and sells generic and proprietary injectable, inhalation, and intranasal products in the United States, China, and France. It offers BAQSIMI, a nasal spray for the treatment of severe hypoglycemia; Primatene Mist, an over-the-counter epinephrine inhalation product for the temporary relief of mild symptoms of intermittent asthma; Enoxaparin, to prevent and treat deep vein thrombosis; REXTOVY and Naloxone for opioid overdose; Glucagon for injection emergency kit; and Cortrosyn, for use as a diagnostic agent in the screening of patients with adrenocortical insufficiency. The company provides Amphadase, an injection to absorb and disperse other injected drugs; Epinephrine injection for allergic reactions; Lidocaine jelly, an anesthetic product for urological procedures; Lidocaine topical solution for various procedures; Phytonadione injection, a vitamin K1 injection for newborn babies; emergency syringe products; morphine injection for use with patient-controlled analgesia pumps; and Lorazepam injection for surgery and medical procedures. In addition, it offers Neostigmine methylsulfate injection to treat myasthenia gravis and to reverse the effects of muscle relaxants; Isoproterenol hydrochloride injection for mild or transient episodes of heart block; Ganirelix Acetate injection for the inhibition of premature luteinizing hormone surges; Vasopressin to increase blood pressure; and Regadenoson, a stress agent for radionuclide myocardial perfusion imaging. Further, the company distributes recombinant human insulin APIs and porcine insulin API. Additionally, it develops generic product candidates, such as injectable, inhalation, and analytical technologies; biosimial product candidates; and intranasal epinephrine for the treatment of allergic reactions. The company was founded in 1996 and is headquartered in Rancho Cucamonga, California.

Design Therapeutics, Inc. a biopharmaceutical company, researches, designs, develops, and commercializes small molecule therapeutic drugs for the treatment of genetic diseases in the United States. The company utilizes its GeneTAC platform to design and develop therapeutic candidates for inherited diseases caused by nucleotide repeat expansion. Its lead product candidates for potentially disease-modifying treatment comprises Friedreich Ataxia, a monogenic, autosomal recessive, progressive multi-system disease that affects organ systems dependent on mitochondrial function that brings to neurological, cardiac, and metabolic dysfunction; Myotonic Dystrophy Type-1, a dominantly-inherited, monogenic progressive neuromuscular disease affecting skeletal muscle, heart, brain, and other organs; Fuchs Endothelial Corneal Dystrophy, a genetic eye disease characterized by bilateral degeneration of corneal endothelial cells and progressive loss of vision; and Huntington's Disease, a dominantly inherited, monogenic neurodegenerative disease characterized by movement, cognitive, and psychiatric disorders. Design Therapeutics, Inc. was incorporated in 2017 and is headquartered in Carlsbad, California.

GH Research PLC, together with its subsidiary GH Research Ireland Limited, engages in developing various therapies to treat psychiatric and neurological disorders. It is involved in the development of 5-Methoxy-N,N-Dimethyltryptamine (5-MeO-DMT) therapies for the treatment of patients with treatment-resistant depression (TRD). The company's lead program is GH001, an inhalable mebufotenin product candidate, currently under Phase 2b clinical trial for the treatment of patients with TRD, as well as Phase 2a clinical trials for the treatment of Bipolar II disorder and postpartum depression. It is also involved in the development of GH002, an intravenous mebufotenin product candidate for IV administration, currently under Phase 1 clinical trials for the treatment of psychiatric or neurological disorder; and GH003, an intranasal mebufotenin product candidate for nasal administration, which is currently in preclinical development with a focus on psychiatric and neurological disorders. The company was founded in 2018 and is based in Dublin, Ireland.

Repare Therapeutics Inc., a clinical-stage precision oncology company, engages in the discovery and development of therapeutics by using its synthetic lethality approach in Canada and the United States. It uses its SNIPRx, a proprietary, genome-wide, and CRISPR-enabled platform, to discover, validate, and build a pipeline of SL-based therapeutics that focuses on genomic instability, including DNA damage repair. The company's lead product candidate is Camonsertib (RP-3500), an oral small molecule inhibitor under Phase ½ development for the treatment of solid tumors with specific DNA damage repair-related genomic alterations. It is also developing Lunresertib (RP-6306), a PKMYT1 Inhibitor, which is under Phase 1 clinical trial for tumors with genetic alterations characterized by CCNE1 amplification; RP-1664, an oral PLK4 inhibitor, under Phase 1 clinical trial designed to harness the synthetic lethal relationship with TRIM37 amplification or overexpression in solid tumors; and RP-3467, a polymerase theta adenosinetriphosphatase (ATPase) inhibitor, a SL target associated with BRCA mutations and other genomic alterations. The company has license and collaboration agreement with Hoffmann-La Roche Inc. and F. Hoffmann-La Roche Ltd; Bristol-Myers Squibb Company; New York University; and Ono Pharmaceutical Co., as well as a clinical study and collaboration agreement with Debiopharm to explore the synthetic lethal combination of PKMYT1 and WEE1 inhibition in cancer. Repare Therapeutics Inc. was incorporated in 2016 and is headquartered in Montréal, Canada.

Stoke Therapeutics, Inc., an early-stage biopharmaceutical company, develops medicines to treat the underlying causes of severe genetic diseases in the United States. The company utilizes its proprietary targeted augmentation of nuclear gene output to develop antisense oligonucleotides to selectively restore protein levels. Its lead clinical candidate is STK-002, which is in preclinical stage for the treatment of autosomal dominant optic atrophy. The company also develops STK-001, which is in phase I/II clinical trial to treat Dravet syndrome; and programs focused on multiple targets, including haploinsufficiency diseases of the central nervous system and eye. The company has a license and collaboration agreement with Acadia Pharmaceuticals Inc. for the discovery, development, and commercialization of novel RNA-based medicines for the treatment of severe and rare genetic neurodevelopmental diseases of the central nervous system. The company was formerly known as ASOthera Pharmaceuticals, Inc. and changed its name to Stoke Therapeutics, Inc. in May 2016. Stoke Therapeutics, Inc. was incorporated in 2014 and is headquartered in Bedford, Massachusetts.