Athira Pharma
NASDAQ:ATHAAthira 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.
Precision BioSciences
NASDAQ:DTILPrecision 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
NASDAQ:GRPHGraphite 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.
Ikena Oncology
NASDAQ:IKNAIkena Oncology, Inc. operates as an oncology company that develops differentiated therapies for patients in need that target nodes of cancer growth, spread, and therapeutic resistance in the United States. Its lead program is IK-930, an internally discovered, oral, TEAD1-selective, small molecule inhibitor of the Hippo pathway. The company also develops IK-595, a molecular glue designed to trap MEK and RAF in an inactive complex. Ikena Oncology, Inc. was incorporated in 2016 and is headquartered in Boston, Massachusetts.
Talaris Therapeutics
NASDAQ:TALSTalaris Therapeutics, Inc. operates as a late-clinical stage cell therapy company in the United States. The company engages in developing a method of allogeneic hematopoietic stem cell transplantation to transform the standard of care in solid organ transplantation and severe autoimmune diseases, as well as severe blood, immune, and metabolic disorders. Its lead product candidate is FCR001, a novel allogeneic cell therapy that is in Phase II trial for living donor kidney transplant patients. The company is also developing FCR001 for the patients with a severe form of scleroderma. Talaris Therapeutics, Inc. was founded in 1988 and is headquartered in Wellesley, Massachusetts.