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AVROBIO, Inc., a clinical-stage gene therapy company, develops hematopoietic stem cell gene therapies to treat rare diseases in the United States. Its gene therapies employ hematopoietic stem cells that are harvested from the patient and then modified with a lentiviral vector to insert the equivalent of a functional copy of the gene that is mutated in the target disease. The company's pipeline includes AVR-RD-02, which is in phase 1/2 clinical trial for the treatment of Gaucher disease type 1 and 3; AVR-RD-01, which is in phase 2 clinical trials for the treatment of Fabry disease; and AVR-RD-03 that is in preclinical stage for the treatment of Pompe disease. The company was incorporated in 2015 and is headquartered in Cambridge, Massachusetts.

Cidara Therapeutics, Inc., a biotechnology company, focuses on the discovery, development, and commercialization of long-acting anti-infectives for the treatment and prevention of infectious diseases and oncology in the United States. The company's lead product candidate is rezafungin acetate, a novel molecule in the echinocandin class of antifungals for the treatment and prevention of invasive fungal infections, including candidemia and invasive candidiasis, which are fungal infections associated with high mortality rates. It also advances its Cloudbreak platform to develop conjugates for the prevention and treatment of influenza and other viral infections, such as RSV, HIV, and the SARS-CoV-2 strains causing COVID-19. The company was formerly known as K2 Therapeutics, Inc. and changed its name to Cidara Therapeutics, Inc. in July 2014. The company was incorporated in 2012 and is based in San Diego, California.

Entera Bio Ltd., a clinical-stage company, focuses on the development and commercialization of orally delivered peptide and protein therapeutics for unmet medical needs. The company's product candidates include the EB612, which is in Phase II clinical trials for the treatment of hypoparathyroidism; and EB613 that has completed Phase II clinical trials for the treatment of osteoporosis, as well as is in Phase I clinical trials for the treatment of stress fractures. Its other product candidate is GLP-2, which is in preclinical trial for the treatment of short bowl syndrome; and OXM for the treatment of obesity and metabolic diseases. The company has a research collaboration and license agreement with the Amgen Inc. for the development and discovery of clinical candidates in the field of inflammatory disease and other serious illnesses. Entera Bio Ltd. was incorporated in 2009 and is based in Jerusalem, Israel.

Genenta Science S.p.A., a clinical-stage biotechnology company, engages in the development of hematopoietic stem cell gene therapies for the treatment of solid tumors in Italy. Its lead product candidate is Temferon, which is in Phase 1/2a clinical trials for use in the treatment of glioblastoma multiforme in patients with unmethylated MGMT gene promoter. The company is also developing Temferon for use in the treatment of other solid tumor indications, locally advanced hepatocellular carcinoma, and intra-hepatic cholangiocarcinoma. In addition, it develops biologic platform to deliver immunomodulatory molecules directly to the tumor by infiltrating monocytes/macrophages. The company was incorporated in 2014 and is headquartered in Milan, Italy.