Amylyx Pharmaceuticals, Inc. Common Stock
Amylyx Pharmaceuticals, Inc., a clinical-stage pharmaceutical company, engages in the discovery and development of treatment options for neurodegenerative diseases and endocrine conditions in the United States. Its lead products candidate includes avexitide, an investigational, first-in-class glucagon-like peptide-1, or GLP-1 receptor antagonist, which is in phase 3 clinical trial to treat post-bariatric hypoglycemia and congenital hyperinsulinism. The company also develops AMX0035, an oral, fixed-dose combination of sodium phenylbutyrate and taurursodiol which is phase 2 clinical trial for the treatment of wolfram syndrome, as well as in phase 2b/3 clinical trial to treat progressive supranuclear palsy; AMX0114, which is in phase 1 clinical trial for the treatment of amyotrophic lateral sclerosis; and long-acting GLP-1 receptor antagonist to treat post-bariatric hypoglycemia and other rare diseases. Amylyx Pharmaceuticals, Inc. was founded in 2013 and is headquartered in Cambridge, Massachusetts.
Open AMLX dashboard ↗CRISPR Therapeutics AG
CRISPR Therapeutics AG, a gene editing company, focuses on developing gene-based medicines for serious human diseases using its Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/CRISPR-associated protein 9 (Cas9) platform. The company's CRISPR/Cas9 is a revolutionary technology for gene editing which is the process of precisely altering specific sequences of genomic DNA. It has a portfolio of therapeutic programs across a range of disease areas, including hemoglobinopathies, CAR T cell therapies, in vivo, and type 1 diabetes, as well as develops investigational CAR T programs, including an autologous, gene-edited CAR T program targeting glypican-3 (GPC3) for the potential treatment of solid tumors. The company's lead product candidate is CASGEVY, an ex vivo CRISPR/Cas9 gene-edited cell therapy for treating patients suffering from transfusion-dependent beta-thalassemia, severe sickle cell disease (SCD), and hemoglobinopathies in which a patient's hematopoietic stem and progenitor cells are edited to produce high levels of fetal hemoglobin in red blood cells. It also develops CAR T cell therapies, including CTX112 targeting cluster of differentiation 19 (CD19) and CTX131 targeting CD70 for oncology and autoimmune indications; CTX310 and CTX320, in vivo gene editing to address the cardiovascular disease by disrupting the validated targets angiopoietin-like protein 3 and lipoprotein; and CTX211, an allogeneic, gene-edited, hypoimmune stem cell-derived product candidate for the treatment of T1D. It has strategic partnerships with Vertex Pharmaceuticals Incorporated, Nkarta, Inc., and Capsida Biotherapeutics. CRISPR Therapeutics AG was incorporated in 2013 and is headquartered in Zug, Switzerland.
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