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Fulcrum Therapeutics, Inc., a clinical-stage biopharmaceutical company, focuses on developing products for improving the lives of patients with genetically defined diseases in the areas of high unmet medical need in the United States. Its product candidates are losmapimod, a small molecule for the treatment of facioscapulohumeral muscular dystrophy is under phase III clinical trial; and pociredir, a fetal hemoglobin inducer for the treatment of sickle cell disease and beta-thalassemia is under phase I clinical trial. The company is also discovering drug targets for the treatments of rare neuromuscular, muscular, central nervous system, and hematologic disorders, as well as cardiomyopathies and pulmonary diseases. Fulcrum Therapeutics, Inc. has collaboration and license agreement with Acceleron Pharma Inc. to identify biological targets to modulate specific pathways associated with a targeted indication within the pulmonary disease space; and MyoKardia, Inc. to discover and develop therapies for the treatment of genetic cardiomyopathies. Fulcrum Therapeutics, Inc. was Incorporated in 2015 and is headquartered in Cambridge, Massachusetts.

KALA BIO, Inc., a clinical-stage biopharmaceutical company, focuses on the discovery, development, and commercialization of therapies for the treatment of eye diseases. The company's product candidates include KPI-012, which is in Phase 2b clinical trial for the treatment of persistent corneal epithelial defects; EYSUVIS for the short-term treatment of the signs and symptoms of dry eye disease; and INVELTYS, a topical twice-a-day ocular steroid for the treatment of post-operative inflammation and pain following ocular surgery. Its preclinical development product, including KPI-014 for the treatment of rare inherited retinal diseases. The company was formerly known as Kala Pharmaceuticals, Inc. and changed its name to KALA BIO, Inc. in August 2023. KALA BIO, Inc. was incorporated in 2009 and is headquartered in Arlington, Massachusetts.

ProQR Therapeutics N.V., a biotechnology company, focuses on the discovery and development of novel therapeutic medicines. The company's products pipeline includes AX-0810 for cholestatic diseases targeting Na-taurocholate cotransporting polypeptide (NTCP); and AX-1412 for cardiovascular diseases (CVDs) targeting Beta-1,4-galactosyltransferase 1 (B4GALT1). It also develops various other early-stage research programs, including AX-1005 for undisclosed targets in CVDs; AX-2402, which focuses on Rett syndrome; AX-2911 for nonalcoholic steatohepatitis (NASH); AX-0601 for obesity and Type 2 diabetes; and AX-9115 for rare metabolic condition, as well as various other targets. In addition, the company develops Axiomer RNA base-editing platform technology. It has a license agreement with Radboud University Medical Center; Inserm Transfert SA; Ionis Pharmaceuticals, Inc.; Vico Therapeutics B.V.; University of Rochester; and Leiden University Medical Center, as well as license and research collaboration with Eli Lilly and Company for the discovery, development, and commercialization of potential new medicines for genetic disorders in the liver and nervous system. The company was incorporated in 2012 and is headquartered in Leiden, the Netherlands.

Syros Pharmaceuticals, Inc., a biopharmaceutical company, focuses on the development of treatment for hematologic malignancies. The company's lead product candidates are Tamibarotene, a selective retinoic acid receptor alpha agonist, which is in Phase III clinical trial for genomically defined subset of patients with myelodysplastic syndrome and Phase II clinical trial for patients with acute myeloid leukemia; SY-2101, a novel oral form of arsenic trioxide for treating patients with acute promyelocytic leukemia; and SY-5609, a cyclin-dependent kinase 7 inhibitor, which is in a Phase I clinical trial in patients with select advanced solid tumors. It has target discovery, research collaboration, and option agreement with Incyte Corporation to identify therapeutic targets with a focus on myeloproliferative neoplasms; and a license agreement with TMRC Co. Ltd. for the development and commercialization of tamibarotene. The company was formerly known as LS22, Inc. and changed its name to Syros Pharmaceuticals, Inc. in August 2012. Syros Pharmaceuticals, Inc. was incorporated in 2011 and is headquartered in Cambridge, Massachusetts.

Theratechnologies Inc., a biopharmaceutical company, focuses on the development and commercialization of various therapies to address the unmet medical needs in the United States, Canada, and Europe. The company offers EGRIFTA SV for the reduction of excess abdominal fat in human immunodeficiency virus (HIV)-infected patients with lipodystrophy; and Trogarzo for the treatment of HIV-1 infection in heavily treatment-experienced adults with multidrug-resistant HIV-1 infection failing their current antiretroviral regimen. Its pipeline products include F8 Formulation that is in Phase 2b/3 clinical trials for studying tesamorelin for the treatment of nonalcoholic steatohepatitis; multi -dose pen injector for the administration of tesamorelin; SORT1+ Technology Platform for the development of proprietary peptides for cancer drug development targeting SORT1 receptors; Sudocetaxel Zendusortide, which is in Phase 1 Clinical Trial for various solid tumor types, including HR+ breast, triple negative breast, ovarian, endometrial, melanoma, thyroid, small cell lung, and prostate cancers. The company was incorporated in 1993 and is headquartered in Montreal, Canada.