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.
Compugen
NASDAQ:CGENCompugen Ltd., a clinical-stage therapeutic discovery and development company, researches, develops, and commercializes therapeutic and product candidates in Israel, the United States, and Europe. The company's immuno-oncology pipeline consists of COM701, an anti-PVRIG antibody that is in Phase I clinical study used for the treatment of solid tumors; COM902, a therapeutic antibody targeting TIGIT, which is in Phase I monotherapy clinical study in patients with advanced malignancies through sequential dose escalations; Bapotulimab, a therapeutic antibody targeting ILDR2 that is in Phase I clinical study in patients with naïve head and neck squamous cell carcinoma; and Rilvegostomig, a novel anti-TIGIT/PD-1 bispecific antibody, which is in Phase II clinical study in patients with advanced or metastatic non-small cell lung cancer. Its therapeutic pipeline also includes early-stage immuno-oncology programs focused to address various mechanisms of immune resistance; and COM503, high affinity antibody, which blocks the interaction between IL-18 binding protein and IL-18. The company has collaboration agreement with Bayer Pharma AG for the research, development, and commercialization of antibody-based therapeutics against the company's immune checkpoint regulators; Bristol-Myers Squibb to evaluate the safety and tolerability of COM701 in combination with Bristol-Myers Squibb's PD-1 immune checkpoint inhibitor Opdivo in patients with advanced solid tumors; and Johns Hopkins School of Medicine to evaluate novel T cell and myeloid checkpoint targets. It has license agreement with AstraZeneca for the development of bi-specific and multi-specific immuno-oncology antibody products; and research collaboration with Johns Hopkins University for myeloid. Compugen Ltd. was incorporated in 1993 and is headquartered in Holon, Israel.
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.
Osiris Therapeutics
OTCMKTS:OSIROsiris Therapeutics, Inc. researches, develops, manufactures, markets, and distributes regenerative medicine products in the United States. Its products include Grafix and Stravix for treating chronic wounds of diabetic foot ulcers, venous leg ulcers, pressure ulcers, arterial ulcers, and severe burns, as well as surgical and trauma wounds; BIO4 for bone repair and regeneration in spine, trauma, extremity, cranial, and foot and ankle surgeries; and Cartiform for treating articular cartilage lesions in the knee and other joints. The company also develops and markets Menvivo for the repair of meniscus; and TruSkin for treating chronic wounds. It markets and distributes its products directly to physicians, hospitals, and other healthcare providers, as well as through agents and distributors. Osiris Therapeutics, Inc. was founded in 1992 and is headquartered in Columbia, Maryland.
Solid Biosciences
NASDAQ:SLDBSolid Biosciences Inc., a life science company, develops therapies for neuromuscular and cardiac diseases in the United States. The company's lead product candidate is SGT-003, a gene transfer candidate for the treatment of Duchenne muscular dystrophy; and SGT-501 to treat Catecholaminergic polymorphic ventricular tachycardia. It is also developing AVB-401 for the treatment of BAG3-mediated dilated cardiomyopathy; SGT-601 to treat a rare cardiac disease characterized by mutations in the gene that codes for cardiac troponin T protein, which helps coordinate contraction of the heart muscle; SGT-701 for treatment of rare inherited disease characterized by mutations in the RBM20 gene, a cardiac splicing factor that regulates alternative splicing, and codes for RNA binding motif protein 20; and AVB-202-TT to treat Friedreich's ataxia, as well as other drugs for the treatment of cardiac and other diseases. In addition, the company is involved in developing platform technologies, including capsid libraries and dual gene expression, a technology for packaging multiple transgenes into one capsid. The company has collaboration and license agreement with Ultragenyx Pharmaceutical Inc. to develop and commercialize new gene therapies for Duchenne; and a license agreement with the University of Washington, University of Missouri, and University of Florida. Solid Biosciences Inc. was incorporated in 2013 and is headquartered in Charlestown, Massachusetts.