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Centessa Pharmaceuticals plc, a clinical-stage pharmaceutical company, discovers, develops, and delivers medicines to patients. Its pipeline products include SerpinPC, an activated protein C inhibitor, which is in Phase IIa clinical development for the treatment of hemophilia A and B; and ORX750, an orally administered OX2R agonist for the treatment of NT1 with potential expansion into other sleep disorders. The company also develops LB101, a PD-L1xCD47 LockBody, which is designed to selectively drive potent CD47 and CD3 effector function activity while avoiding systemic toxicity; ZF874 in alpha-1 antitrypsin deficiency, a dual-STAT3/5 degrader program in acute myeloid leukemia (AML); MGX292, a protein-engineered variant of human bone morphogenetic protein 9 (BMP9) for the treatment of pulmonary arterial hypertension (PAH); and OX2R Agonists compounds are currently in development for the treatment of narcolepsy, including TAK-861. In addition, its products pipeline comprises CBS001, a neutralizing therapeutic mAb to the inflammatory membrane form of LIGHT for inflammatory / fibrotic diseases; and CBS004, a therapeutic mAb targeting BDCA-2 for the potential treatment of autoimmune diseases, as well as earlier-stage preclinical assets, including ORX750, an orally administered, selective orexin receptor-2 (OX2R) agonist for the treatment of narcolepsy and other sleep disorders; and discovery-stage programs in certain other disease areas. Centessa Pharmaceuticals plc was incorporated in 2020 and is based in Altrincham, the United Kingdom.

Dicerna 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, 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, 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.

Prelude Therapeutics Incorporated, a clinical-stage biopharmaceutical company, focuses on the discovery and development of novel precision cancer medicines to underserved patients. It is developing PRT1419, a myeloid cell leukemia-1 inhibitor, which is in Phase 1 clinical trial for the treatment of selected relapsed/refractory myeloid or B-cell malignancies; PRT2527, a cyclin-dependent kinase, which is in Phase 1 clinical trial for the treatment of advanced solid tumors; PRT3645, a cyclin-dependent kinase 4/6 inhibitor, which is in Phase 1 clinical trial for the treatment of advanced and metastatic solid tumors; and PRT3879, a SMARCA2 selective protein degrader, which is in Phase 2/3 clinical trial for the treatment of advanced and metastatic solid tumors with loss of SMARCA4 due to truncating mutation and/or deletion. The company's CDK9 program is a regulator of cancer-promoting transcriptional programs, including MCL1, MYC and MYB. Prelude Therapeutics Incorporated was incorporated in 2016 and is based in Wilmington, Delaware.