Mustang Bio
NASDAQ:MBIOMustang Bio, Inc., a clinical-stage biopharmaceutical company, focuses on translating medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors, and rare genetic diseases. Its pipeline focuses on gene therapy programs for rare genetic disorders, chimeric antigen receptor (CAR) engineered T cell (CAR T) therapies for hematologic malignancies, and CAR T therapies for solid tumors. The company develops MB-117 and MB-217, a gene therapy program for X-linked severe combined immunodeficiency, a rare genetic immune system condition in which affected patients do not live beyond infancy without treatment. The company also develops MB-106 CAR T cell program for B cell non-hodgkin lymphoma and chronic lymphocytic leukemia; MB-101 CAR T cell program for glioblastoma; MB-108, a next-generation oncolytic herpes simplex virus. It has license agreements with Nationwide Children's Hospital, CSL Behring; Mayo Clinic; Leiden University Medical Centre; SIRION Biotech GmbH. The company was incorporated in 2015 and is headquartered in Worcester, Massachusetts.
Northwest Biotherapeutics
OTCMKTS:NWBONorthwest Biotherapeutics, Inc., a biotechnology company, develops personalized immune therapies for cancer in the United States and internationally. The company develops its products based on DCVax, a platform technology that uses activated dendritic cells to mobilize a patient's own immune system to attack cancer. Its lead product, DCVax-L, which has completed Phase III clinical trials to treat Glioblastoma multiforme brain cancer. The company also develops DCVax-Direct, which is in Phase I/II clinical trials to treat inoperable solid tumors. The company was founded in 1996 and is headquartered in Bethesda, Maryland.
RedHill Biopharma
NASDAQ:RDHLRedHill Biopharma Ltd., a specialty biopharmaceutical company, primarily focuses on gastrointestinal and infectious diseases. The company promotes gastrointestinal drugs, including Movantik for opioid-induced constipation in adults with chronic non-cancer pain; Talicia for the treatment of Helicobacter pylori infection in adults; and Aemcolo for the treatment of travelers' diarrhea in adults. Its clinical late-stage investigational development programs include RHB-204, which is in Phase 3 study for pulmonary nontuberculous mycobacteria infections; opaganib (Yeliva), an SK2 selective inhibitor, which has completed Phase 2 study to treat patients with SARS-CoV-2 severe COVID-19 pneumonia, in Phase 2 study to treat advanced unresectable cholangiocarcinoma, and in investigator-sponsored Phase 2 study to treat prostate cancer; RHB-107, which is in Phase 2/3 study to treat outpatients infected with COVID-19 disease, and preclinical evaluation study to treat advanced unresectable cholangiocarcinoma, as well as has completed Phase 2 study to treat gastrointestinal and other solid tumors; and RHB-104, which is in Phase 3 studies for Crohn's disease; RHB-102 (Bekinda) that is in Phase 3 studies for acute gastroenteritis and gastritis. RedHill Biopharma Ltd. was incorporated in 2009 and is headquartered in Tel Aviv, Israel.
Strongbridge Biopharma
NASDAQ:SBBPStrongbridge Biopharma Plc operates as a commercial-stage biopharmaceutical company, which focuses on the development and commercialization of therapies for rare diseases. Its pipeline is comprised of Keveyis, Macrilen, Recorlev, and Veldoreotide. The company was founded in 1996 and is headquartered in Trevose, PA.
Savara
NASDAQ:SVRASavara Inc., formerly Mast Therapeutics, Inc., is a clinical-stage pharmaceutical company. The Company is focused on the development and commercialization of novel therapies for the treatment of patients with rare respiratory diseases. Its pipeline includes AeroVanc, Molgradex and AIR001. AeroVanc is an inhaled formulation of vancomycin, which the Company is developing for the treatment of persistent methicillin-resistant Staphylococcus aureus, lung infection in cystic fibrosis patients. Molgradex is an inhaled formulation of recombinant human granulocyte-macrophage colony-stimulating factor. It is developing Molgradex for the treatment of autoimmune pulmonary alveolar proteinosis, a rare lung disease. AIR001 is a sodium nitrite solution for inhalation via nebulization. AIR001 is in Phase II clinical development for the treatment of heart failure with preserved ejection fraction, also known as diastolic heart failure or heart failure with preserved systolic function.