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.
Harpoon Therapeutics
NASDAQ:HARPHarpoon Therapeutics, Inc., a clinical-stage immunotherapy company, engages in the development of a novel class of T cell engagers that harness the power of the body's immune system to treat patients suffering from cancer and other diseases in the United States. The company develops tri-specific T cell activating construct (TriTAC) product candidate, including HPN328, which is in Phase I/II clinical trials for the treatment of small cell lung cancer and other Delta-like canonical Notch ligand 3-expressing tumors; and HPN217 that is in Phase I clinical trials for the treatment of multiple myeloma. Its preclinical stage product is HPN601 for the treatment of multiple solid tumor indications. It has a discovery collaboration and license agreement with AbbVie Biotechnology Ltd. to develop and commercialize products that incorporate its proprietary TriTAC platform technology together with soluble T cell receptors. The company was incorporated in 2015 and is headquartered in South San Francisco, California. As of March 11, 2024, Harpoon Therapeutics, Inc. operates as a subsidiary of Merck Sharp & Dohme LLC.
LogicBio Therapeutics
NASDAQ:LOGCLogicBio Therapeutics, Inc., a genetic medicine company, focuses on developing and commercializing genome editing and gene therapy treatments using its GeneRide and sAAVy platforms. The company's GeneRide technology is a new approach to precise gene insertion harnessing a cell's natural deoxyribonucleic acid; and gene delivery platform, sAAVy is an adeno-associated virus, which is designed to optimize gene delivery for treatments in a range of indications and tissues. Its lead product candidate is LB-001 that is in Phase I/II clinical trials for the treatment of methylmalonic acidemia. The company has a collaboration with Children's Medical Research Institute to develop next-generation capsids for gene therapy and gene editing applications in the liver, as well as additional tissues; and a collaboration agreement with Takeda Pharmaceutical Company Limited to develop LB-301, an investigational therapy for the treatment of Crigler-Najjar syndrome. The company also has a research collaboration, license, and option agreement with CANbridge Care Pharma Hong Kong Limited; and collaboration agreement with Daiichi Sankyo Company. The company was incorporated in 2014 and is headquartered in Lexington, Massachusetts.
PDL BioPharma
NASDAQ:PDLIPDL BioPharma, Inc. manages various patents in the United States and internationally. The company's patents cover humanization of antibodies. It also offers notes and other long-term receivables services, as well as engages in the equity investment activities. The company was formerly known as Protein Design Labs, Inc. and changed its name to PDL BioPharma, Inc. in 2006. PDL BioPharma, Inc. was founded in 1986 and is headquartered in Incline Village, Nevada.