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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.

Generation Bio Co. develops non-viral genetic medicines for the treatment of rare and prevalent diseases. The company develops cell-targeted lipid nanoparticle (ctLNP) platform, a modular delivery system for nucleic acids to avoid off-target clearance by the liver and spleen that enables ctLNPs to persist in systemic circulation, which allows for highly selective and potent ligand-driven targeting to specific tissues and cell types; and novel immune-quiet DNA (iqDNA) to enable long-lasting high levels of gene expression from non-integrating episomes and avoids innate immune sensors that have long prevented DNA from use in non-viral systems. It uses its platform for developing a portfolio of programs for treating cancer, autoimmune, hematologic disorders, prioritizing sickle cell, beta-thalassemia, and hemophilia A diseases, as well as for other tissues and cell types, including retina, skeletal muscle, and central nervous system. The company was formerly known as Torus Therapeutics, Inc. and changed its name to Generation Bio Co. in November 2017. Generation Bio Co. was incorporated in 2016 and is headquartered in Cambridge, Massachusetts.

MacroGenics, Inc., a biopharmaceutical company, develops, manufactures, and commercializes antibody-based therapeutics to treat cancer in the United States. Its approved product is MARGENZA (margetuximab-cmkb), a human epidermal growth factor receptor 2 (HER2) receptor antagonist indicated, in combination with chemotherapy, for the treatment of adult patients with metastatic HER2-positive breast cancer who have received two or more prior anti-HER2 regimens. The company's pipeline of immuno-oncology product candidates includes MGC018, an antibody drug conjugate (ADC), which targets solid tumors expressing B7-H3; Enoblituzumab, a monoclonal antibody that targets B7-H3; and MGD024, an investigational bispecific CD123 × CD3 DART molecule to minimize cytokine-release syndrome for patients with hematologic malignancies. In addition, it develops Lorigerlimab, a monoclonal antibody that targets the immune checkpoints PD-1 and cytotoxic T-lymphocyte-associated protein 4; Tebotelimab, an investigational tetravalent DART molecule for PD-1 and lymphocyte-activation gene 3; Retifanlimab, a humanized monoclonal antibody targeting programmed death receptor-1; and IMGC936, an ADC that targets ADAM9, a cell surface protein over-expressed in various solid tumor types. Further, the company develops MGD014 and MGD020, a DART molecule to target the envelope protein of human immunodeficiency virus infected cells and CD3 on T cells; Teplizumab for the treatment of type 1 diabetes; and PRV-3279, a CD32B × CD79B DART molecule for the treatment of autoimmune indications. It has collaborations with Incyte Corporation; Zai Lab Limited; I-Mab Biopharma; and Janssen Biotech, Inc. The company was incorporated in 2000 and is headquartered in Rockville, Maryland.

Stoke Therapeutics, Inc., an early-stage biopharmaceutical company, develops medicines to treat the underlying causes of severe genetic diseases in the United States. The company utilizes its proprietary targeted augmentation of nuclear gene output to develop antisense oligonucleotides to selectively restore protein levels. Its lead clinical candidate is STK-002, which is in preclinical stage for the treatment of autosomal dominant optic atrophy. The company also develops STK-001, which is in phase I/II clinical trial to treat Dravet syndrome; and programs focused on multiple targets, including haploinsufficiency diseases of the central nervous system and eye. The company has a license and collaboration agreement with Acadia Pharmaceuticals Inc. for the discovery, development, and commercialization of novel RNA-based medicines for the treatment of severe and rare genetic neurodevelopmental diseases of the central nervous system. The company was formerly known as ASOthera Pharmaceuticals, Inc. and changed its name to Stoke Therapeutics, Inc. in May 2016. Stoke Therapeutics, Inc. was incorporated in 2014 and is headquartered in Bedford, Massachusetts.