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Aeglea BioTherapeutics, Inc., a clinical-stage biotechnology company, designs and develops human enzyme therapeutics for the treatment of rare metabolic diseases. The company's therapeutic candidates include pegtarviliase, a polyethylene glycol modified, which is in Phase I/II clinical trial for the treatment of patient with homocystinuria; and pegzilarginase, a recombinant human Arginase 1 that is in Phase III PEACE trial to evaluate the safety and efficacy for the treatment of Arginase 1 deficiency. In addition, its preclinical pipeline includes AGLE-325 for the treatment of cystinuria, as well as other research programs. The company was formerly known as Aeglea BioTherapeutics Holdings, LLC and changed its name to Aeglea BioTherapeutics, Inc. in March 2015. Aeglea BioTherapeutics, Inc. was founded in 2013 and is headquartered in Austin, Texas.

Fortress Biotech, Inc., a biopharmaceutical company, develops and commercializes pharmaceutical and biotechnology products. The company markets dermatology products, such as Ximino an oral minocycline drug for the treatment of moderate to severe acne; Targadox an oral doxycycline drug for adjunctive therapy for severe acne; Exelderm cream for antifungal intended for topical use; Qbrexza a medicated cloth towelette for the treatment of primary axillary hyperhidrosis; Amzeeq; Zilxi; and Accutane capsules for severe recalcitrant nodular acne. It also develops late stage product candidates, such as intravenous Tramadol for the treatment of post-operative acute pain; CUTX-101, an injection for the treatment of Menkes disease; MB-107 and MB-207 for the treatment of X-linked severe combined immunodeficiency; Cosibelimab for metastatic cancers; CK-101 for the treatment of patients with EGFR mutation-positive NSCLC; CAEL-101 for the treatment of amyloid light chain amyloidosis; Triplex vaccine for cytomegalovirus; and CEVA101 for the treatment of severe traumatic brain injury in adults and children. The company's early stage product candidates include MB-102 for blastic plasmacytoid dendritic cell neoplasm; MB-101 for glioblastoma; MB-104 for multiple myeloma and light chain amyloidosis; MB-106 for B-cell non-hodgkin lymphoma; MB-103 for GBM & metastatic breast cancer to brain; MB-108; MB-105 for prostate and pancreatic cancers; and BAER-101. Its preclinical product candidates comprise AAV-ATP7A gene therapy; AVTS-001 gene therapy; CK-103 BET inhibitor; CEVA-D and CEVA-102; CK-302, an anti-GITR; CK-303, an anti-CAIX; and ONCOlogues, and oligonucleotide platform. It has collaboration arrangements with universities, research institutes, and pharmaceutical companies. The company was formerly known as Coronado Biosciences, Inc. and changed its name to Fortress Biotech, Inc. in April 2015. Fortress Biotech, Inc. was incorporated in 2006 and is based in Bay Harbor Islands, Florida.

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.

Inozyme Pharma, Inc., a clinical-stage rare disease biopharmaceutical company, focuses on developing novel therapeutics for the treatment of vasculature, soft tissue, and skeleton diseases. The company's lead product candidate is INZ-701, a soluble, recombinant, genetically engineered, and fusion protein for the treatment of ENPP1 and ABCC6 deficiencies, as well as patients with end-stage kidney disease receiving hemodialysis. It has a license agreement with Yale University for specified therapeutic and prophylactic products. The company was incorporated in 2015 and is headquartered in Boston, Massachusetts.