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Athira Pharma, Inc., a late clinical-stage biopharmaceutical company, focuses on developing small molecules to restore neuronal health and slow neurodegradation. Its lead product candidate is Fosgonimeton (ATH-1017), a small molecule designed to modulate the neurotrophic hepatocyte growth factor (HGF) system and its receptor, MET, for a healthy nervous system that is in LIFT-AD Phase 2/3 and ACT-AD Phase 2 clinical trials for the treatment of Alzheimer's disease, as well as is in Phase 2 clinical trials to treat Parkinson's disease dementia and Dementia with Lewy bodies. The company's product pipeline includes ATH-1020, an orally available brain-penetrant small molecule designed to enhance the HGF/MET system that is in Phase 1 clinical trials to treat neuropathic pain and neurodegenerative diseases; and ATH-1105, an oral small molecule drug candidate, which is a preclinical model for the treatment of Amyotrophic Lateral Sclerosis. In addition, it has a license agreement with Washington State University to offer for sale products covered by certain licensed patents, including dihexa, the chemical compound into which fosgonimeton metabolizes following administration; and collaboration and grant agreement with National Institutes of Health Grant to support ACT-AD Phase 2 clinical trial for fosgonimeton. The company was formerly known as M3 Biotechnology, Inc. and changed its name to Athira Pharma, Inc. in April 2019. Athira Pharma, Inc. was incorporated in 2011 and is headquartered in Bothell, Washington.

Aura Biosciences, Inc., a clinical-stage biotechnology company, develops precision immunotherapies to treat a range of solid tumors. The company's proprietary platform enables the targeting of a range of solid tumors using virus-like particles conjugated with drugs or loaded with nucleic acids to create virus-like drug conjugates. Its lead candidate is bel-sar, which is in late-stage clinical development for the treatment of patients with primary choroidal melanoma and other ocular oncology indications, as well as in early-stage clinical development in bladder cancer. The company also focuses on assessing the safety and efficacy of bel-sar in treating a range of other solid tumors, including bladder cancer as an alternative to bacillus calmetteguérin therapy. Aura Biosciences, Inc. was incorporated in 2009 and is headquartered in Boston, 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.

Graphite Bio, Inc., a clinical-stage gene editing company, engages in developing therapies for serious and life-threatening diseases in the United States. The company offers GPH102 for the treatment of beta-thalassemia; and GPH201 for the treatment of XSCID, a life-threatening disease for multiple mutations in a single gene. It also offers GPH301, a product candidate for the treatment of Gaucher disease, a genetic disorder that results in a deficiency in the glucocerebrosidase enzyme. The company was formerly known as Integral Medicines, Inc., and changed its name to Graphite Bio, Inc. in August 2020. Graphite Bio, Inc. was incorporated in 2017 and is based 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.