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Calliditas Therapeutics AB (publ), a commercial-stage specialty pharmaceutical company, focused on identifying, developing, and commercializing pharmaceuticals products for treatments in orphan indications with initial focus on renal and hepatic diseases. It offers Nefecon, an oral formulation of budesonide that is an immunosuppressant for the treatment of the autoimmune renal disease immunoglobulin A nephropathy. The company's lead product candidate is Setanaxib, a NOX inhibitor that is in Phase 2b/3 clinical trial for the treatment of primary biliary cholangitis; and in Phase 2 clinical trial for the treatment of squamous cell carcinoma of the head and neck cancer and idiopathic pulmonary fibrosis, as well as for type 1 diabetic kidney disease. It also develops Budenofalk 3 mg oral capsules for the treatment of autoimmune hepatitis. The company was founded in 2004 and is headquartered in Stockholm, Sweden.

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.

Olema Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company, focuses on the discovery, development, and commercialization of therapies for women's cancers. Its lead product candidate is OP-1250, an estrogen receptor (ER) antagonist and a selective ER degrader, which is in Phase 3 clinical trial for the treatment of recurrent, locally advanced, or metastatic estrogen receptor-positive, human epidermal growth factor receptor 2-negative breast cancer; and OP-1250 combine with CDK4/6 inhibitors palbociclib, ribociclib, and alpelisib in Phase 1/2 clinical trial for the treatment of recurrent, locally advanced, or metastatic estrogen receptor-positive human epidermal growth factor receptor 2-negative breast cancer, as well as develops OPERA-01 for the of ER+/HER2- advanced or metastatic breast cancer. The company was formerly known as CombiThera, Inc. and changed its name to Olema Pharmaceuticals, Inc. in March 2009. Olema Pharmaceuticals, Inc. was incorporated in 2006 and is headquartered in San Francisco, California.

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.