Applied Genetic Technologies
NASDAQ:AGTCApplied Genetic Technologies Corporation, a clinical-stage biotechnology company, develops transformational genetic therapies for patients suffering from rare and debilitating diseases. Its advanced product candidates include three ophthalmology development programs across two targets, including X-linked retinitis pigmentosa, which is in the Phase 1/2 clinical trials; and achromatopsia that is in Phase 1/2 clinical trials. It is also developing an optogenetic product candidate to treat advanced retinal disease. In addition, the company has initiated one preclinical program in otology; preclinical program in dry age-related macular degeneration; and two preclinical programs in targeting central nervous system disorders, including frontotemporal dementia and amyotrophic lateral sclerosis. It has collaboration agreements with University of Florida; Bionic Sight, Inc.; and Otonomy, Inc. The company was incorporated in 1999 and is headquartered in Alachua, Florida.
AVROBIO
NASDAQ:AVROAVROBIO, Inc., a clinical-stage gene therapy company, develops ex vivo lentiviral-based gene therapies to treat rare diseases following a single dose worldwide. Its gene therapies employ hematopoietic stem cells that are harvested from the patient and then modified with a lentiviral vector to insert the equivalent of a functional copy of the gene that is mutated in the target disease. The company's pipeline includes AVR-RD-02, which is in phase 1/2 clinical trial for the treatment of Gaucher disease type 1, as well as to treat Gaucher disease type 3; AVR-RD-03 that is in preclinical stage for the treatment of Pompe disease; AVR-RD-04, which is in phase 1/2 clinical trial for the treatment of cystinosis; and AVR-RD-05 that is in preclinical stage for the treatment of Hunter syndrome. The company was incorporated in 2015 and is headquartered in Cambridge, Massachusetts.
CASI Pharmaceuticals
NASDAQ:CASICASI Pharmaceuticals, Inc., a biopharmaceutical company, develops and commercializes therapeutics and pharmaceutical products in the People's Republic of China, the United States, and internationally. It offers EVOMELA, an intravenous formulation of melphalan for use as a conditioning treatment prior to stem cell transplantation, and as a palliative treatment for patients with multiple myeloma. The company's other hematology/oncology assets in pipeline include CNCT 19, an autologous CD19 CAR-T investigative product; BI-1206, novel anti-Fc?RIIB antibody, for the treatment of solid tumors and relapsed/refractory non-hodgkin lymphoma; CB-5339, a novel VCP/p97 inhibitor that focuses on valosin-containing protein (VCP)/p97 as a novel target in protein homeostasis, DNA damage response, and other cellular stress pathways for therapeutic use in the treatment of patients with various malignancies; and CID-103, a human IgG1 anti-CD38 monoclonal antibody. The company has licensing agreements with Juventas Biotechnology (Tianjin) Co., Ltd.; BioInvent International AB; Black Belt Therapeutics Limited; Cleave Therapeutics, Inc.; and Acrotech Biopharma L.L.C. to develop and commercialize its commercial product EVOMELA. It also has distribution agreements with China Resources Pharmaceutical Commercial Group International Trading Co., Ltd; Pharmathen Global BV; and Riemser Pharma GmbH. CASI Pharmaceuticals, Inc. was incorporated in 1991 and is based in Beijing, the People's Republic of China.
Pluristem Therapeutics
NASDAQ:PSTIPluristem Therapeutics Inc. operates as a bio-technology company. It focuses on the research, development, clinical trial, and manufacture of cell therapeutic products and related technologies for the treatment of various ischemic, inflammatory, and hematologic conditions, as well as autoimmune disorders. The company develops placental expanded (PLX) based cell therapy products, including PLX-PAD that is in Phase III clinical trial for the recovery after surgery for hip fracture; in Phase II clinical trail for the treatment of acute respiratory distress syndrome associated with COVID-19; and in Phase I/II clinical trial for treatment of steroid-refractory graft versus host disease. It also develops PLX-R18, which has completed Phase I clinical trial incomplete hematopoietic recovery following hematopoietic cell transplantation, as well as conducts various animal studies for the evaluation of PLX-R18 for the treatment of acute radiation syndrome. The company has a license agreement for conducting clinical trials of PLX-PAD product in South Korea. It also has a collaborative project nTRACK, that examines gold nano particles labeling of stem cells; and has collaboration agreement with the NASA's Ames Research Center to evaluate the potential of PLX cell therapies in preventing and treating medical conditions caused during space missions. Pluristem Therapeutics Inc. was incorporated in 2001 and is based in Haifa, Israel.
TRACON Pharmaceuticals
NASDAQ:TCONTRACON Pharmaceuticals, Inc., a biopharmaceutical company, focuses on the development and commercialization of novel targeted therapeutics for cancer in the United States. Its clinical stage products include envafolimab (KN035), a PD-L1 single-domain antibody for the treatment of refractory soft tissue sarcoma; and YH001, an investigational humanized cytotoxic T-lymphocyte-associated protein 4 IgG1 monoclonal antibody that is in Phase I clinical trial for the treatment of various cancer indications. The company's clinical stage products also include TRC102, a small molecule that is in Phase II clinical trial for the treatment of mesothelioma, and in Phase I clinical trial to treat solid tumors and lymphomas, lung cancer, and glioblastoma; and TJ004309, a CD73 antibody that is in Phase I clinical development for the treatment of solid tumors. In addition, it is developing bispecific antibodies, which are in preclinical stage. It has collaboration and license agreements with 3D Medicines Co., Ltd. and Jiangsu Alphamab Biopharmaceuticals Co., Ltd. for the development of envafolimab; I-Mab Biopharma for the development of CD73 antibody TJ004309 and bispecific antibodies; and cooperative research and development agreement with National Cancer Institute. The company was formerly known as Lexington Pharmaceuticals, Inc. and changed its name to TRACON Pharmaceuticals, Inc. in March 2005. TRACON Pharmaceuticals, Inc. was incorporated in 2004 and is headquartered in San Diego, California.