Homology Medicines
NASDAQ:FIXXHomology Medicines, Inc., a genetic medicines company, focuses on transforming the lives of patients suffering from rare genetic diseases in the United States. The company offers proprietary platform is designed to utilize its human hematopoietic stem cell derived adeno-associated virus vectors (AAVHSCs) to deliver genetic medicines in vivo either through a nuclease-free gene editing mobility, gene therapy or for various genetic disorders. Its earlier stage product candidates comprising HMI-202, an investigational gene therapy which completed IND-enabling studies for the treatment of metachromatic leukodystrophy; and HMI-104 which completed IND-enabling studies. Homology Medicines, Inc. was incorporated in 2015 and is headquartered in Bedford, Massachusetts.
ProQR Therapeutics
NASDAQ:PRQRProQR Therapeutics N.V., a biotechnology company, focuses on the discovery and development of novel therapeutic medicines. The company's products pipeline includes AX-0810 for cholestatic diseases targeting Na-taurocholate cotransporting polypeptide (NTCP); and AX-1412 for cardiovascular diseases (CVDs) targeting Beta-1,4-galactosyltransferase 1 (B4GALT1). It also develops various other early-stage research programs, including AX-1005 for undisclosed targets in CVDs; AX-2402, which focuses on Rett syndrome; AX-2911 for nonalcoholic steatohepatitis (NASH); AX-0601 for obesity and Type 2 diabetes; and AX-9115 for rare metabolic condition, as well as various other targets. In addition, the company develops Axiomer RNA base-editing platform technology. It has a license agreement with Radboud University Medical Center; Inserm Transfert SA; Ionis Pharmaceuticals, Inc.; Vico Therapeutics B.V.; University of Rochester; and Leiden University Medical Center, as well as license and research collaboration with Eli Lilly and Company for the discovery, development, and commercialization of potential new medicines for genetic disorders in the liver and nervous system. The company was incorporated in 2012 and is headquartered in Leiden, the Netherlands.
Seelos Therapeutics
NASDAQ:SEELSeelos Therapeutics, Inc., a clinical-stage biopharmaceutical company, focuses on the development and commercialization of therapeutics for the treatment of central nervous system, respiratory, and other disorders. The company's lead programs are SLS-002, an intranasal racemic ketamine for the treatment of acute suicidal ideation and behavior in patients with major depressive disorders; SLS-005, a protein stabilizer for the treatment of amyotrophic lateral sclerosis and Sanfilippo syndrome; and SLS-006, a partial dopamine agonist for the treatment of patients with Parkinson's disease (PD). Its preclinical programs include SLS-007, a peptidic inhibitor to treat patients with PD; SLS-008 for the treatment of pediatric indications; SLS-004 for the treatment of PD; SLS-010, an H3 receptor antagonist; and SLS-012. The company was founded in 2016 and is headquartered in New York, New York.
Viracta Therapeutics
NASDAQ:VIRXViracta Therapeutics, Inc., a clinical-stage precision oncology company, focuses on the treatment and prevention of virus-associated cancers that impact patients worldwide. Its lead product candidate is Nana-val, an all-oral combination therapy of its proprietary investigational drug, nanatinostat, and the antiviral agent valganciclovir. Nana-val is in various ongoing clinical trials, including NAVAL-1, an open-label Phase 2 basket trial for the treatment of multiple subtypes of relapsed/refractory Epstein-Barr virus-positive (EBV+) lymphoma, as well as an open-label Phase 1b/2 trial for the treatment of EBV+ recurrent or metastatic nasopharyngeal carcinoma and other EBV+ solid tumors. The company's development pipeline also includes vecabrutinib, a clinical-stage non-covalent ITK/BTK inhibitor; and VRx-510, a preclinical-stage PDK-1 inhibitor. The company was formerly known as Sunesis Pharmaceuticals, Inc. and changed its name to Viracta Therapeutics, Inc. in February 2021. Viracta Therapeutics, Inc. was incorporated in 1998 and is headquartered in Cardiff-by-the-Sea, California.