Akouos
NASDAQ:AKUSAkouos, Inc., a precision genetic medicine company, developing gene therapies to restore, improve, and preserve physiologic hearing for individuals. The company's precision genetic medicine platform incorporates a proprietary adeno-associated viral vector library and a novel delivery approach. Its lead product candidate is AK-OTOF, a gene therapy for the treatment of hearing loss due to mutations in the OTOF gene. The company is also developing AK-CLRN1 for the auditory manifestations of Usher syndrome 3A, or USH3A; and AK-antiVEGF for vestibular schwannoma. In addition, its precision genetic medicine platform addresses hearing loss related to genes needed for supporting cell function. The company was incorporated in 2016 and is based in Boston, Massachusetts.
Applied Molecular Transport
NASDAQ:AMTIApplied Molecular Transport Inc., a clinical-stage biopharmaceutical company, engages in the design and development of a pipeline of oral biologic product candidates to treat autoimmune, inflammatory, metabolic, and other diseases. The company's lead product candidate is AMT-101, a gastrointestinal (GI) selective oral fusion of rhIL-10 that is in Phase II clinical trials to treat patients with chronic pouchitis, as well as is in Phase II development to treat patients with rheumatoid arthritis. It develops AMT-126, a GI-selective oral fusion of interleukin 22, which is in a Phase I clinical trial for diseases related to intestinal epithelium barrier function defects. The company, through its technology platform, designs and develops various oral and respiratory biologic therapeutic modalities, such as peptides, proteins, antibodies, antibody fragments, and ribonucleic acid therapeutics. Applied Molecular Transport Inc. was founded in 2010 and is headquartered in South San Francisco, California. As of December 27, 2023, Applied Molecular Transport Inc. operates as a subsidiary of Cyclo Therapeutics, Inc.
Assembly Biosciences
NASDAQ:ASMBAssembly Biosciences, Inc., a biopharmaceutical company, discovers and develops therapeutic candidates for the treatment of hepatitis B virus (HBV) infection in the United States. The company's next-generation HBV core inhibitors include ABI-4334, which is in Phase 1a studies for the treatment of HBV; and ABI-H3733, which is in Phase 1b clinical study. It is also involved in novel small molecule approaches for HBV and hepatitis delta virus (HDV) comprising an orally bioavailable HBV/HDV entry inhibitor and liver-focused interferon-a receptor agonist. In addition, the company develops Herpesvirus programs, such as ABI-5366 HSV-2, a long-acting helicase inhibitor; and pan-herpes non-nucleoside polymerase inhibitors for transplant-associated infections. Further, Assembly Biosciences, Inc. has collaboration agreement with BeiGene, Ltd. to develop and commercialize the novel core inhibitor product candidates for chronic HBV infection in the People's Republic of China, Hong Kong, Taiwan, and Macau; and clinical trial collaboration agreement with Arbutus Biopharma Corporation to conduct a randomized, multi-center, and open-label clinical trials, as well as agreement with Antios Therapeutics, Inc. for triple combination therapy. The company also has license agreement with Indiana University Research and Technology Corporation for HBV research, as well as a partnership with Gilead Sciences, Inc. to develop next-generation therapeutics for serious viral diseases. The company was formerly known as Ventrus Biosciences, Inc. and changed its name to Assembly Biosciences, Inc. in June 2014. Assembly Biosciences, Inc. was incorporated in 2005 and is headquartered in South San Francisco, California.
Nkarta
NASDAQ:NKTXNkarta, Inc., a clinical-stage biopharmaceutical company, develops and commercializes natural killer cell therapies for cancer and autoimmune disease treatment. The company's lead product candidate is NKX019, a chimeric antigen receptor-natural killer (CAR NK) targeting the CD19 antigen that is in Phase 1 clinical trial for the treatment of relapsed/refractory (r/r) non-hodgkin lymphoma, as well as for lupus nephritis. It also develops NKX101, a CAR NK product candidate targeting cells that display NKG2D ligands, which is in Phase I clinical trial for the treatment of r/r acute myeloid leukemia or higher risk myelodysplastic syndromes, as well as for solid tumors. In addition, the company develops NKX070, targeting the CD70 tumor antigen to treat solid and liquid tumors; and NK+T cell therapy for use in the treatment of oncology, autoimmune disease, or infectious disease. It has a research collaboration agreement with CRISPR Therapeutics AG. Nkarta, Inc. was incorporated in 2015 and is based in South San Francisco, California.
ZIOPHARM Oncology
NASDAQ:ZIOPZIOPHARM Oncology, Inc. is a biopharmaceutical company, which engages in the development, acquisition, and commercialization of immuno-oncology platforms that leverage cell- and gene-based therapies to treat patients with cancer. Its pipeline includes Sleeping Beauty TCR-T Targeting neoantigens; Ad-RTS-hlL-12 + veledimex; and Sleeping Beauty CAR-T. The company was founded on September 9, 2003 and is headquartered in Boston, MA.