Akouos
NASDAQ:AKUSAkouos, Inc., a precision genetic medicine company, developing gene therapies to restore, improve, and preserve physiologic hearing for individuals. The company's precision genetic medicine platform incorporates a proprietary adeno-associated viral vector library and a novel delivery approach. Its lead product candidate is AK-OTOF, a gene therapy for the treatment of hearing loss due to mutations in the OTOF gene. The company is also developing AK-CLRN1 for the auditory manifestations of Usher syndrome 3A, or USH3A; and AK-antiVEGF for vestibular schwannoma. In addition, its precision genetic medicine platform addresses hearing loss related to genes needed for supporting cell function. The company was incorporated in 2016 and is based in Boston, Massachusetts.
Geron
NASDAQ:GERNGeron Corporation, a late-stage clinical biopharmaceutical company, focuses on the development and commercialization of therapeutics for myeloid hematologic malignancies. It develops imetelstat, a telomerase inhibitor that is in Phase 3 clinical trials, which inhibits the uncontrolled proliferation of malignant stem and progenitor cells in myeloid hematologic malignancies for the treatment of low or intermediate-1 risk myelodysplastic syndromes and intermediate-2 or high-risk myelofibrosis. The company was incorporated in 1990 and is headquartered in Foster City, California.
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.
Spectrum Pharmaceuticals
NASDAQ:SPPISpectrum Pharmaceuticals, Inc., a biopharmaceutical company, develops and commercializes oncology drug products. It offers ROLVEDON(Eflapegrastim), a novel long-acting granulocyte colony-stimulating factor for chemotherapy-induced neutropenia. The company develops Poziotinib, a novel irreversible tyrosine kinase inhibitor for non-small cell lung cancer tumors with various mutations. It has co-development, commercialization, and in-license agreements with Hanmi Pharmaceutical Co. Ltd.; a patent and technology license agreement with The University of Texas M.D. Anderson Cancer Center; and license agreement with Therapyx, Inc. The company was formerly known as NeoTherapeutics, Inc. and changed its name to Spectrum Pharmaceuticals, Inc. in December 2002. Spectrum Pharmaceuticals, Inc. was incorporated in 1987 and is headquartered in Boston, Massachusetts.
Verastem
NASDAQ:VSTMVerastem, Inc., a development-stage biopharmaceutical company, focuses on developing and commercializing drugs for the treatment of cancer in the United States. Its product candidates are Avutometinib, an orally available small molecule RAF/MEK clamp that inhibits the ras sarcoma RAF/MEK, ERK mitogen activated pathway kinase pathway which is involved in cell proliferation, migration, transformation, and survival of tumor cells; and Defactinib, an oral small molecule inhibitor of FAK and proline-rich tyrosine kinase for various solid tumors. The company is involved in clinical studies, including RAMP 301, a randomized global confirmatory trial to evaluate the combination of Avutometinib and Defactinib for the treatment of patients with recurrent low-grade serous ovarian cancer; RAMP 201, an adaptive two-part multicenter, parallel cohort, randomized open label trial to evaluate the efficacy and safety of Avutometinib and in combination with Defactinib; and FRAME, an investigation of Avutometinib and Defactinib in patients with KRAS mutant cancers and subsequent analyses; and RAMP 204 and 205. It has license agreements with Chugai Pharmaceutical Co., Ltd. for the development, commercialization, and manufacture of products containing Avutometinib; and Pfizer Inc. to research, develop, manufacture, and commercialize products containing Pfizer's inhibitors of FAK for therapeutic, diagnostic, and prophylactic uses in humans. In addition, it has a clinical collaboration agreement with Amgen, Inc. to evaluate the combination of Avutometinib with Amgen's KRAS-G12C inhibitor LUMAKRAS which in Phase 1/2 trial entitled RAMP 203; and a discovery and development collaboration with GenFleet Therapeutics to advance new programs targeting RAS pathway-driven cancers. Verastem, Inc. was incorporated in 2010 and is headquartered in Needham, Massachusetts.