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.
Atreca
NASDAQ:BCELAtreca, Inc., a clinical-stage biopharmaceutical company, discovers and develops antibody-based immunotherapeutics to treat a range of solid tumor types. Its lead product candidate is ATRC-101, a monoclonal antibody with a novel mechanism of action and target derived from an antibody identified using its discovery platform. The company's ATRC-101 product candidate reacts in vitro with a majority of human ovarian, non-small cell lung, colorectal, and breast cancer samples from multiple patients; and ATRC-501/MAM01, that targets the circumsporozoite protein of Plasmodium falciparum for the treatment of malaria. Its products in pre-clinical stage include APN-497444, an antibody drug conjugate (ADC) against a novel tumor glycan target; and APN-346958, a CD3 bispecific T-cell engager against an RNA-binding protein target. It has a collaboration and license agreement with Xencor, Inc. for research, development, and commercialization of novel CD3 bispecific antibodies in oncology; licensing agreement with the Bill & Melinda Gates Medical Research Institute for the development and commercialization of MAM01/ATRC-501 for the prevention of malaria. The company was incorporated in 2010 and is based in San Carlos, California.
BELLUS Health
OTCMKTS:BLUSFBELLUS Health, Inc. is a biopharmaceutical development company, which engages in the research and development of products that provide health solutions and address critical unmet medical needs. Its products include BLU-5937, KIACTA, AMO-01, and ALZ-801. The company was founded on June 17, 1993 and is headquartered in Laval, Canada.
Intercept Pharmaceuticals
NASDAQ:ICPTIntercept Pharmaceuticals, Inc., a biopharmaceutical company, focuses on the development and commercialization of therapeutics to treat progressive non-viral liver diseases in the United States, Europe, and Canada. The company markets Ocaliva, a farnesoid X receptor agonist used for the treatment of primary biliary cholangitis in combination with ursodeoxycholic acid in adults. It is also developing Ocaliva for various indications, including nonalcoholic steatohepatitis and NASH; and other product candidates in various stages of clinical and preclinical development. The company has a license agreement with Aralez Pharmaceuticals Canada Inc. to develop and commercialize bezafibrate in the United States. It markets its products through an internal commercial organization and third-party distributors. The company was incorporated in 2002 and is headquartered in Morristown, New Jersey. As of November 8, 2023, Intercept Pharmaceuticals, Inc. operates as a subsidiary of Alfasigma S.p.A..
Magenta Therapeutics
NASDAQ:MGTAMagenta Therapeutics, Inc., a biotechnology company focus on improving stem cell transplantation. Its product candidates are designed to bring the curative power of stem cell transplant to patients with blood cancers, genetic diseases, and autoimmune diseases. The company's product portfolio includes MGTA-117, an anti-CD117 antibody conjugated to an amanitin payload that targets hematopoietic stem cells (HSCs) and leukemia cells; and MGTA-45, an anti-human CD45 antibody conjugated to a DNA-interacting payload for HSCs, leukemia cells, and immune cells. In addition, it focus on development of ADC-based conditioning program that targets a receptor of T cells; and novel conditioning and translation stem cell sciences, as well as develops cell therapy program, E478, which is a small molecule aryl hydrocarbon receptor antagonist for stem cell-based gene therapy and genome editing. In addition, the company has a research and clinical collaboration agreement with AVROBIO, Inc. for the treatment of lysosomal storage disorders; and Beam Therapeutics, Inc. to evaluate the potential utility of MGTA-117 for conditioning of patients with sickle cell disease and beta-thalassemia. Magenta Therapeutics, Inc. was formerly known as HSCTCo Therapeutics, Inc. and changed its name to Magenta Therapeutics, Inc. in February 2016. The company was incorporated in 2015 and is headquartered in Cambridge, Massachusetts.