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.
Galera Therapeutics
NASDAQ:GRTXGalera Therapeutics, Inc., a clinical stage biopharmaceutical company, focuses on the development and commercialization of therapeutics for the transformation of radiotherapy in cancer. The company's lead product candidate is avasopasem manganese (GC4419), a small molecule dismutase mimetic, which has completed Phase III clinical trial for the treatment of radiotherapy induced severe oral mucositis in patients with head and neck cancer (HNC); in Phase IIa clinical trial for the treatment of radiotherapy-induced esophagitis in patients with lung cancer; and in Phase IIa clinical trial for patients with HNC undergoing standard-of-care radiotherapy. It is also developing GC4711, a dismutase mimetic product candidate, which is in Phase I/II clinical trial in combination with stereotactic body radiation therapy in patients with non-small cell lung cancer and locally advanced pancreatic cancer. The company was incorporated in 2012 and is based in Malvern, Pennsylvania.
Osmotica Pharmaceuticals
NASDAQ:OSMTOsmotica Pharmaceuticals Plc engages in the development and commercialization of products for neurology and women's health. Its products include Upneeq, M-72, Arbaclofen ER, OS870, Divigel, OB Complete, Methylphenidate ER, Venlafaxine ER tablets (VERT), Hydromorphone ER, Sodium Benzoate/Sodium Phenylacetate, Oxybutynin ER, Prescription Prenatal Vitamins, Chlorzoxazone (Lorzone AG), Tramadol ER (ConZip AG), Nitrofurantoin and Osmodex ANDAs. The company was founded in 1986 and is headquartered in Bridgewater, NJ.
Ovid Therapeutics
NASDAQ:OVIDOvid Therapeutics Inc., a biopharmaceutical company, engages in the development of impactful medicines for patients and families with epilepsies and seizure-related neurological disorders in the United States. The company is developing soticlestat, a novel cholesterol 24 hydroxylase inhibitor, which is in Phase 3 clinical trials for the potential treatment of patients with resistant epilepsies; OV329, a GABA aminotransferase inhibitor which is in Phase 1 clinical trials for the treatment of seizures associated with tuberous sclerosis complex and infantile spasms; and OV350, a small molecule direct activator of the KCC2 transporter, which is in Phase 1 clinical trials for treating epilepsies. It also develops OV815, that focuses on the mutations associated with KIF1A-associated neurological disorder (KAND); OV888 (GV101), a highly selective rock2 inhibitor which is in Phase 1 double-blind multiple-ascending dose trial; OV825, has advanced to potential candidate lead identification for the rare neurodevelopmental condition HNRNPH2 (Bain Syndrome); and OV882, a short hairpin RNA gene therapy for the treatment of Angelman syndrome. The company has license and collaboration agreements with Healx, AstraZeneca AB, H. Lundbeck A/S, Northwestern University, and Graviton, as well as Marinus Pharmaceuticals, Inc. The company was incorporated in 2014 and is headquartered in New York, New York.
Selecta Biosciences
NASDAQ:SELBSelecta Biosciences, Inc., a clinical-stage biopharmaceutical company, engages in the research and development of nanoparticle immunomodulatory drugs for the treatment and prevention of human diseases. The company's lead therapeutic gene therapy program is SEL-302 that is in Phase I clinical trial to enhance the treatment of methylmalonic acidemia. It is also developing biologic therapies, such as SEL-212 that is in Phase III clinical trials for the treatment of chronic refractory gout; and product candidates to treat IgA-mediated diseases, including IgA nephropathy, linear IgA bullous dermatitis, IgA pemphigus, and Henoch-Schonlein purpura. In addition, the company is developing gene therapies comprising for the treatment of pompe disease, duchenne muscular dystrophy, limb-girdle muscular dystrophy, lysosomal storage disorder, and other autoimmune diseases. Further, it develops tolerogenic therapies to treat autoimmune diseases. The company has license and collaboration agreements with Ginkgo Bioworks Holdings, Inc.; Genovis AB (publ.); Cyrus Biotechnology, Inc.; IGAN Biosciences, Inc.; Astellas Therapeutics, Inc.; Takeda Pharmaceuticals USA, Inc.; Swedish Orphan Biovitrum AB (publ.); Sarepta Therapeutics, Inc.; Asklepios Biopharmaceutical, Inc.; Massachusetts Institute of Technology; and Shenyang Sunshine Pharmaceutical Co., Ltd. Selecta Biosciences, Inc. was incorporated in 2007 and is headquartered in Watertown, Massachusetts.