Aldeyra Therapeutics
NASDAQ:ALDXAldeyra Therapeutics is a clinical-stage biotechnology company focused on the development of novel therapies with the potential to improve the lives of patients with immune-mediated diseases. Two of the company's lead compounds, reproxalap and ADX-629, target reactive aldehyde species (RASP), which are elevated in ocular and systemic inflammatory disease, leading to elevated levels of cytokine release via activation of a broad array of inflammatory factors, including NF-κB, inflammasomes, and Scavenger Receptor A. Reproxalap is being evaluated in Phase 3 clinical trials in patients with dry eye disease and allergic conjunctivitis. The company's clinical pipeline also includes ADX-2191, a dihydrofolate reductase inhibitor in Phase 3 testing for proliferative vitreoretinopathy, and ADX-1612, a chaperome inhibitor in Phase 2 testing for COVID-19 and ovarian cancer.
Fulcrum Therapeutics
NASDAQ:FULCFulcrum Therapeutics, Inc., a clinical-stage biopharmaceutical company, focuses on developing products for improving the lives of patients with genetically defined diseases in the areas of high unmet medical need in the United States. Its product candidates are losmapimod, a small molecule for the treatment of facioscapulohumeral muscular dystrophy is under phase III clinical trial; and pociredir, a fetal hemoglobin inducer for the treatment of sickle cell disease and beta-thalassemia is under phase I clinical trial. The company is also discovering drug targets for the treatments of rare neuromuscular, muscular, central nervous system, and hematologic disorders, as well as cardiomyopathies and pulmonary diseases. Fulcrum Therapeutics, Inc. has collaboration and license agreement with Acceleron Pharma Inc. to identify biological targets to modulate specific pathways associated with a targeted indication within the pulmonary disease space; and MyoKardia, Inc. to discover and develop therapies for the treatment of genetic cardiomyopathies. Fulcrum Therapeutics, Inc. was Incorporated in 2015 and is headquartered in Cambridge, Massachusetts.
Generation Bio
NASDAQ:GBIOGeneration 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.
I-Mab
NASDAQ:IMABI-Mab, a clinical stage biopharmaceutical company, discovers, develops, and commercializes biologics to treat cancer and autoimmune disorders. It is developing Felzartamab, a CD38 antibody that is in Phase 1b/2a for patients with membranous nephropathy; Eftansomatropin alfa, a long-acting growth hormone, which has completed Phase 3 clinical trials to treat pediatric growth hormone deficiency; Efineptakin alfa, a recombinant human IL-7 that is in Phase 2 for cancer treatment-related lymphopenia and cancer immunotherapy; and Lemzoparlimab, a CD47 monoclonal antibody that has completed Phase 2 clinical trial. Its product candidates also includes TJ210, a monoclonal antibody against human C5aR1 that is in Phase 1 for the treatment of cancers and autoimmune; Uliledlimab, a CD73 antibody, which is in Phase 2 clinical trials for treating solid tumors and oncology; TJ-L14B, a PD-L1-based tumor-dependent T-cell engager for solid cancers; Givastomig, a tumor-dependent T cell engager for gastric and other cancers; TJ-L1IF, a PD-L1/IFN-a antibody-cytokine fusion protein for PD(L)-1 resistant tumors; and TJ-C64B, a bispecific antibody for ovarian and other cancers. The company has strategic collaboration agreement with PT Kalbe Genexine Biologics; AbbVie Ireland Unlimited Company; Jumpcan Pharmaceutical Group; Sinopharm Group Co. Ltd.; and Roche Diagnostics. I-Mab was founded in 2014 and is headquartered in Shanghai, the People's Republic of China.