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Homology Medicines, Inc., a genetic medicines company, focuses on transforming the lives of patients suffering from rare genetic diseases in the United States. The company offers proprietary platform is designed to utilize its human hematopoietic stem cell derived adeno-associated virus vectors (AAVHSCs) to deliver genetic medicines in vivo either through a nuclease-free gene editing mobility, gene therapy or for various genetic disorders. Its earlier stage product candidates comprising HMI-202, an investigational gene therapy which completed IND-enabling studies for the treatment of metachromatic leukodystrophy; and HMI-104 which completed IND-enabling studies. Homology Medicines, Inc. was incorporated in 2015 and is headquartered in Bedford, Massachusetts.

Odonate Therapeutics is a pharmaceutical company dedicated to the development of best-in-class therapeutics that improve and extend the lives of patients with cancer. Our initial focus is on the development of tesetaxel, a novel chemotherapy agent that belongs to a class of drugs known as taxanes, which are widely used in the treatment of cancer. Tesetaxel has several potential therapeutic advantages over currently available taxanes, including: oral administration with a low pill burden and a patient-friendly dosing regimen; a formulation that does not contain solubilizing agents that are known to cause hypersensitivity (allergic) reactions; and improved activity against chemotherapy-resistant tumors. Tesetaxel has been generally well tolerated in clinical studies and has demonstrated robust single-agent antitumor activity in two Phase 2 studies in patients with locally advanced or metastatic breast cancer (''MBC'').

OPKO Health, Inc., a healthcare company, engages in the diagnostics and pharmaceuticals businesses in the United States, Ireland, Chile, Spain, Israel, Mexico, and internationally. The company's Diagnostics segment operates BioReference Laboratories that offers laboratory testing services for the detection, diagnosis, evaluation, monitoring, and treatment of diseases, including esoteric testing, molecular diagnostics, anatomical pathology, genetics, women's health, and correctional healthcare to physician offices, clinics, hospitals, employers, and governmental units; and 4Kscore prostate cancer test. Its Pharmaceutical segment offers Rayaldee to treat secondary hyperparathyroidism in adults with stage 3 or 4 chronic kidney disease, and vitamin D insufficiency. This segment also develops multi-specific immune therapies focused on oncology, infectious diseases, vaccines, and immunology; OPK88004, an orally administered selective androgen receptor modulator; OPK88003, a once-weekly administered peptide for the treatment of type 2 diabetes and related obesity; Somatrogon (hGH-CTP), a once-weekly human growth hormone injection; and Factor VIIa-CTP, a novel long-acting coagulation factor being developed to treat hemophilia. In addition, it develops and commercializes longer-acting proprietary versions of already approved therapeutic proteins; develops and produces specialty APIs; develops, manufactures, markets, and sells pharmaceutical, nutraceutical, veterinary, and ophthalmic products; commercializes food supplements and over the counter products; manufactures and sells products primarily in the generics market; and markets, distributes, and sells pharmaceutical products in a range of indications, including cardiovascular products, vaccines, antibiotics, gastro-intestinal products, hormones, and others. The company also operates pharmaceutical platforms in Ireland, Chile, Spain, and Mexico. The company was founded in 1991 and is headquartered in Miami, Florida.

Rocket Pharmaceuticals, Inc., together with its subsidiaries, operates as a late-stage biotechnology company that focuses on developing gene therapies for rare and devastating diseases. It has three clinical-stage ex vivo lentiviral vector programs for fanconi anemia, a genetic defect in the bone marrow that reduces production of blood cells or promotes the production of faulty blood cells; leukocyte adhesion deficiency-I, a genetic disorder that causes the immune system to malfunction; and pyruvate kinase deficiency, a rare red blood cell autosomal recessive disorder that results in chronic non-spherocytic hemolytic anemia. The company also has a clinical stage in vivo adeno-associated virus program for Danon disease, a multi-organ lysosomal-associated disorder leading to early death due to heart failure; Plakophilin-2 Arrhythmogenic Cardiomyopathy, an inheritable cardiac disorder; and BAG3 Dilated Cardiomyopathy. It has license agreements with Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas, Centro de Investigacion Biomedica En Red, and Fundacion Instituto de investigacion Sanitaria Fundacion Jimenez Diaz; UCL Business PLC; The Regents of the University of California; and REGENXBIO, Inc. Rocket Pharmaceuticals, Inc. was founded in 1999 and is headquartered in Cranbury, New Jersey.