uniQure N.V.
uniQure N.V. develops treatments for patients suffering from rare and other devastating diseases in the United States. The company offers HEMGENIX that allows people living with hemophilia B to produce factor IX, which can lower the risk of bleeding. Its lead product candidate is AMT-130, a gene therapy candidate, which is in Phase I/II clinical study for the treatment of Huntington's disease. The company also develops AMT-260, which is in Phase I/IIa clinical trial for the treatment of mesial temporal lobe epilepsy; AMT-162, which is in Phase I/IIa clinical trial to treat superoxide dismutase enzyme-amyotrophic lateral sclerosis; and AMT-191, an investigational gene therapy candidate which is in phase I/IIa clinical trial for the treatment of fabry disease. It has a licensing agreement with Apic Bio to develop, manufacture, and commercialize intrathecally administered investigational gene therapy for ALS caused by mutations in SOD-1; and development and commercial supply agreement with CLS Bhering. uniQure N.V. was founded in 1998 and is headquartered in Amsterdam, the Netherlands.
Open QURE dashboard ↗Sana Biotechnology, Inc. Common Stock
Sana Biotechnology, Inc., a biotechnology company, focuses on utilizing engineered cells as medicines in the United States. It develops ex vivo and in vivo cell engineering platforms for various therapeutic areas with unmet treatment needs, including type 1 diabetes, B cell mediated autoimmune diseases, and oncology. The company's product pipeline consist of UP421, a HIP-modified allogeneic primary islet cell product, which is in phase 1 clinical trial to treat type 1 diabetes; SC451, a HIP-modified, stem cell derived pancreatic islet cell therapy, which is in preclinical trial for the treatment of type 1 diabetes; SC291, a HIP-modified allogeneic CD19-dirtected allogeneic CAR T cell product candidate which is in phase 1 clinical trial to treat systemic lupus erythematosus and ANCA-associated vasculitis, as well as in preclinical trial to treat other indications and b-cell mediated autoimmune disease; and SG299, a CD8-targeted fusosome, which is in preclinical trial to treat patients with hematologic malignancies and B cell mediated autoimmune diseases. It also develops SC262, a hypoimmune-modified CD22-directed allogeneic CAR T program, which is in phase 1 to treat non-Hodgkin's lymphoma; and SG299, a CD8-targeted fusosome, which is in preclinical trial to treat patients with hematologic malignancies and B cell mediated autoimmune diseases. The company has an option and license agreement with Beam Therapeutics Inc. for use of Beam's proprietary CRISPR Cas12b nuclease editing technology to research, develop, and commercialize engineered cell therapy products; and a license agreement with Harvard College to access certain intellectual property for the development of hypoimmune-modified cells. The company was formerly known as FD Therapeutics, Inc. and changed its name to Sana Biotechnology, Inc. in September 2018. Sana Biotechnology, Inc. was incorporated in 2018 and is headquartered in Seattle, Washington.
Open SANA dashboard ↗