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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.

Eiger BioPharmaceuticals, Inc., a commercial-stage biopharmaceutical company, focuses on the development and commercialization of targeted therapies for rare and ultra-rare diseases in the United States and internationally. Its lead product candidate is Lonafarnib, an orally bioavailable, small molecule, which is in Phase III clinical trials to treat hepatitis delta virus infection. The company's product candidate also include Lambda, which targets type III interferon receptors that has completed Phase II clinical trials; Lonafarnib for the treatment of progeria and progeroid laminopathies; and Avexitide for the treatment of congenital hyperinsulinism, as well as has completed Phase II clinical trials to treat post-bariatric hypoglycemia. Eiger BioPharmaceuticals, Inc. was founded in 2008 and is headquartered in Palo Alto, California.

Harpoon Therapeutics, Inc., a clinical-stage immunotherapy company, engages in the development of a novel class of T cell engagers that harness the power of the body's immune system to treat patients suffering from cancer and other diseases in the United States. The company develops tri-specific T cell activating construct (TriTAC) product candidate, including HPN328, which is in Phase I/II clinical trials for the treatment of small cell lung cancer and other Delta-like canonical Notch ligand 3-expressing tumors; and HPN217 that is in Phase I clinical trials for the treatment of multiple myeloma. Its preclinical stage product is HPN601 for the treatment of multiple solid tumor indications. It has a discovery collaboration and license agreement with AbbVie Biotechnology Ltd. to develop and commercialize products that incorporate its proprietary TriTAC platform technology together with soluble T cell receptors. The company was incorporated in 2015 and is headquartered in South San Francisco, California. As of March 11, 2024, Harpoon Therapeutics, Inc. operates as a subsidiary of Merck Sharp & Dohme LLC.

PDL BioPharma, Inc. manages various patents in the United States and internationally. The company's patents cover humanization of antibodies. It also offers notes and other long-term receivables services, as well as engages in the equity investment activities. The company was formerly known as Protein Design Labs, Inc. and changed its name to PDL BioPharma, Inc. in 2006. PDL BioPharma, Inc. was founded in 1986 and is headquartered in Incline Village, Nevada.

Scholar Rock Holding Corporation, a biopharmaceutical company, focuses on the discovery, development, and delivery of medicines for the treatment of serious diseases in which signaling by protein growth factors plays a fundamental role. The company develops Apitegromab, an inhibitor of the activation of myostatin that is in Phase 3 clinical trial for the treatment of spinal muscular atrophy; and SRK-181, which has completed Phase 1 clinical trials for the treatment of cancers that are resistant to checkpoint inhibitor therapies, such as anti-PD-1 or anti-PD-L1 antibody therapies. It is developing a pipeline of product candidates to deliver novel therapies to treat a range of serious diseases, including neuromuscular disorders, cardiometabolic disorders, cancer, fibrosis, and iron-restricted anemia. Scholar Rock Holding Corporation was founded in 2012 and is headquartered in Cambridge, Massachusetts.