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Curis, Inc., a biotechnology company, engages in the discovery and development of drug candidates for the treatment of human cancers in the United States. Its clinical stage drug candidates include Emavusertib, an oral small molecule IRAK4 kinase inhibitor, which is in a Phase 1/2 open-label, single arm expansion trial in patients with relapsed or refractory, or R/R, AML and high-risk myelodysplastic syndromes. The company's pipeline also includes Fimepinostat, an oral dual inhibitor of HDAC and PI3K enzymes for the treatment of patients with relapsed or refractory diffuse large B-cell lymphoma; CA-170, an oral, small molecule antagonist designated as CA-170 that selectively targets PD-L1 and VISTA; and CA-327, an oral, small molecule, TIM3/PD-L1, which is a molecule antagonist of PD-L1 and TIM3. It has collaboration agreement with Genentech Inc., or Genentech and F. Hoffmann-La Roche Ltd, or Roche, for the commercialization of Erivedge, an orally-administered small molecule hedgehog signaling pathway antagonist for the treatment of advanced basal cell carcinoma, or BCC; Aurigene Discovery Technologies Limited for the discovery, development, and commercialization of small molecule compounds in the areas of immuno-oncology and precision oncology; and also licensed four programs under the Aurigene collaboration, including emavusertib. Curis, Inc. was incorporated in 2000 and is headquartered in Lexington, Massachusetts.

Precision BioSciences, Inc., an advanced gene editing company, develops in vivo gene editing therapies for gene edits, including gene elimination, insertion, and excision in the United States. The company offers ARCUS, a genome editing platform to DNA genome insertion, deletion, and repair. It also provides PBGENE-HBV for the treatment of chronic hepatitis B virus (HBV) to eliminate covalently closed circular DNA with direct cuts and edits as well as to inactivate integrated HBV DNA with the goal of long-lasting reductions in hepatitis B surface antigen; PBGENE-PMM for the treatment of m.3243 associated primary mitochondrial myopathy (PMM) which is expected to submit an IND and/or CTA. In addition, it develops PBGENE-NVS for sickle cell disease/beta thalassemia for insertion; PBGENE-DMD (excision) for duchenne muscular dystrophy; PBGENE-LL2 (insertion), a liver directed target; PBGENE-LL3, a central nervous system directed target; and iECURE-OTC (insertion) for ornithine transcarbamylase deficiency. The company has license and collaboration agreement with Caribou Biosciences, Inc.; license agreement with TG Cell Therapy, Inc. to develop, manufacture, and commercialize azer-cel for autoimmune diseases and other indications outside of cancer; development and license agreement with Eli Lilly and Company for the research and development of potential in vivo therapies for genetic disorders; Cellectis S.A.; iECURE, Inc. to develop ARCUS-based gene-insertion therapies; Duke University; and Novartis Pharma AG to discover and develop in vivo gene editing products. Precision BioSciences, Inc. was incorporated in 2006 and is headquartered in Durham, North Carolina.

Mesoblast Limited engages in the development of regenerative medicine products in Australia, the United States, Singapore, and Switzerland. The company offers products in the areas of cardiovascular, spine orthopedic disorder, oncology, hematology, and immune-mediated and inflammatory diseases. Its proprietary regenerative medicine technology platform is based on specialized cells known as mesenchymal lineage cells. The company offers Remestemcel-L that is in Phase III clinical trials for the treatment of systemic inflammatory diseases, including steroid refractory acute graft versus host disease, acute respiratory distress syndrome, and biologic refractory inflammatory bowel disease; and Remestemcel-L, which is in Phase III clinical trials to treat chronic heart failure and chronic low back pain due to degenerative disc disease. It is also developing MPC-300-IV to treat biologic refractory rheumatoid arthritis diabetic nephropathy; and MPC-25-IC for the treatment or prevention of acute myocardial infarction. It has strategic partnerships with Tasly Pharmaceutical Group to offer MPC-150-IM for heart failure and MPC-25-IC for heart attacks in China; JCR Pharmaceuticals Co. Ltd. to treat wound healing in patients with epidermolysis bullosa; and Grünenthal to develops and commercializes cell therapy for the treatment of chronic low back pain. The company was incorporated in 2004 and is headquartered in Melbourne, Australia.

Tenaya Therapeutics, Inc., a biotechnology company, discovers, develops, and delivers therapies for heart disease in the United States. It develops its products through gene editing, cellular regeneration, and gene addition. The company is developing TN-201, a gene therapy for myosin binding protein C3-associated hypertrophic cardiomyopathy which is in phase 1 clinical trial; TN-301, a small molecule for heart failure with preserved ejection fraction which is in phase 1 clinical trial; and TN-401, a gene therapy for plakophilin 2-associated arrhythmogenic right ventricular cardiomyopathy which is in preclinical stage. It also develops an adeno-associated virus-based gene therapy designed to deliver the dworf gene for patient with dilated cardiomyopathy; and reprogramming program for heart failure due to prior myocardial infarction. Tenaya Therapeutics, Inc. was incorporated in 2016 and is headquartered in South San Francisco, California.

Taysha Gene Therapies, Inc., a gene therapy company, focuses on developing and commercializing adeno-associated virus-based gene therapies for the treatment of monogenic diseases of the central nervous system. It primarily develops TSHA-120 for the treatment of giant axonal neuropathy; TSHA-102 for the treatment of Rett syndrome; TSHA-121 for the treatment of CLN7 disease; TSHA-118 for the treatment of CLN1 disease; TSHA-105 for the treatment of for SLC13A5 deficiency; TSHA-113 for the treatment of tauopathies; TSHA-106 for the treatment of angelman syndrome; TSHA-114 for the treatment of fragile X syndrome; and TSHA-101 for the treatment of GM2 gangliosidosis. Taysha Gene Therapies, Inc. has a strategic partnership with The University of Texas Southwestern Medical Center. Taysha Gene Therapies, Inc. was incorporated in 2019 and is headquartered in Dallas, Texas.