Dicerna Pharmaceuticals
NASDAQ:DRNADicerna Pharmaceuticals, Inc. is a biopharmaceutical company, which engages in discovering, developing and commercializing medicines that are designed to leverage ribonucleic acid interference (RNAi) to selectively silence genes that cause or contribute to disease. The firm uses its proprietary GalXC RNAi technology platform to build a pipeline in these therapeutic areas. The company was founded by Douglas M. Fambrough III, Mark A. Behlke, Roberto Guerciolini, James Craig Jenson, and John J. Rossi in October 2006 and is headquartered in Lexington, MA.
IVERIC bio
NASDAQ:ISEEIVERIC bio, Inc., a biopharmaceutical company, focuses on the discovery and development of novel treatments for retinal diseases with unmet medical needs. The company develops Zimura, an inhibitor of complement factor C5 comprising GATHER1, which has completed Phase 2/3 clinical trial; GATHER2 that is in Phase 3 clinical trial for the treatment of geographic atrophy (GA) secondary to dry age-related macular degeneration (AMD); and STAR or OPH2005 that is in Phase 2b clinical trial for the treatment of autosomal recessive Stargardt disease. Its preclinical product candidatures include IC-500, a high temperature requirement A serine peptidase 1 protein inhibitors for the treatment of GA and other age-related retinal diseases. The company also develops OPH2001 that has completed Phase 1/2a clinical trial of various doses of avacincaptad pegol (ACP) administered as a monotherapy for the treatment of GA; OPH2000, which has completed Phase 1/2a clinical trial of various doses of ACP administered in combination with Lucentis for the treatment of wet AMD; OPH2007 that has completed Phase 2a clinical trial for the treatment of wet AMD; and OPH2002, which has completed Phase 2a clinical trial for the treatment of idiopathic polypoidal choroidal vasculopathy. In addition, its minigene programs comprises miniCEP290 program for LCA10; miniABCA4 Program for STGD1; and miniUSH2A Program for usher syndrome type 2A-Related inherited retinal diseases (IRDs). The company was formerly known as Ophthotech Corporation and changed its name to IVERIC bio, Inc. in April 2019. The company was incorporated in 2007 and is based in Parsippany, New Jersey. As of July 11, 2023, IVERIC bio, Inc. operates as a subsidiary of Astellas US Holding, Inc.
Nuvalent
NASDAQ:NUVLNuvalent, Inc., a clinical stage biopharmaceutical company, engages in the development of therapies for patients with cancer. Its lead product candidates are NVL-520, a novel ROS1-selective inhibitor to address the clinical challenges of emergent treatment resistance, central nervous system (CNS)-related adverse events, and brain metastases that may limit the use of ROS1 tyrosine kinase inhibitors (TKIs) for patients with ROS proto-oncogene 1 (ROS1)-positive non-small cell lung cancer (NSCLC) which is under the phase 2 portion of the ARROS-1 Phase 1/2 clinical trial; NVL-655, a brain-penetrant ALK-selective inhibitor, to address the clinical challenges of emergent treatment resistance, CNS-related adverse events, and brain metastases that might limit the use of first-, second-, and third-generation ALK inhibitors that is under the phase 2 portion of the ALKOVE-1 Phase 1/2 clinical trial; and NVL-330, a brain-penetrant human epidermal growth factor receptor 2 (HER2)-selective inhibitor designed to treat tumors driven by HER2ex20, brain metastases, and avoiding treatment-limiting adverse events including due to off-target inhibition of wild-type EGFR, which is expected to initiate phase 1 trial. The company was incorporated in 2017 and is headquartered in Cambridge, Massachusetts.
uniQure
NASDAQ:QUREuniQure N.V. develops treatments for patients suffering from rare and other devastating diseases. It offers HEMGENIX that has completed Phase III HOPE-B pivotal trial for the treatment of hemophilia B. The company also develops AMT-130, a gene therapy that is in Phase I/II clinical study for the treatment of Huntington's disease; AMT-162, which is in preclinical trial for the treatment of superoxide dismutase 1- amyotrophic lateral sclerosis; AMT-260 that is in preclinical trial to treat temporal lobe epilepsy; AMT-191, which is in preclinical trial for the treatment of fabry disease; AMT-161 that is in preclinical trial to treat amyotrophic lateral sclerosis caused by mutations; AMT-240, which is in preclinical trial to for the treatment of autosomal dominant Alzheimer's disease; and AMT-210 that is in preclinical trial to treat Parkinson's disease. uniQure N.V. was founded in 1998 and is headquartered in Amsterdam, the Netherlands.
Zymeworks
NYSE:ZYMEZymeworks Inc., a clinical-stage biopharmaceutical company, discovers, develops, and commercializes biotherapeutics for the treatment of cancer. The company's lead product candidates include zanidatamab, a human epidermal growth factor receptor 2 (HER2) that is in Phase 1, Phase 2, and Phase 3 clinical trials, including certain ongoing pivotal clinical trials; and zanidatamab zovodotin, a HER2 -targeted antibody-drug conjugate that is in Phase 2 clinical trial for the treatment of advanced or metastatic HER2-expressing tumors. It also develops a pipeline of preclinical product candidates and discovery-stage programs in oncology, including immuno-oncology agents and other therapeutic areas. Zymeworks Inc. has strategic partnerships and collaborations with BeiGene, Ltd.; Celgene Corporation; Celgene Alpine Investment Co. LLC; GlaxoSmithKline Intellectual Property Development Limited; Daiichi Sankyo Co., Ltd.; Janssen Biotech, Inc.; LEO Pharma A/S; Iconic Therapeutics, Inc.; Merck Sharp & Dohme Research GmbH; and Atreca, Inc. Zymeworks Inc. was incorporated in 2003 and is based in Middletown, Delaware.