Abeona Therapeutics
NASDAQ:ABEOAbeona Therapeutics Inc., a clinical-stage biopharmaceutical company, focuses on developing and delivering gene therapy products for severe and life-threatening rare diseases. The company's lead programs are EB-101 (gene-corrected skin grafts) for recessive dystrophic epidermolysis bullosa (RDEB); ABO-102, which are AAV based gene therapies for Sanfilippo syndrome type A; and ABO-101, an adeno-associated virus (AAV) based gene therapies for Sanfilippo syndrome type B. It is also developing ABO-201 gene therapy for juvenile Batten disease; ABO-202 gene therapy for treatment of infantile Batten disease; EB-201 for for epidermolysis bullosa (EB); ABO-301 for Fanconi anemia disorder; and ABO-302 using a novel CRISPR/Cas9-based gene editing approach to gene therapy program for rare blood diseases. Further, it is involved in marketing MuGard, a mucoadhesive oral wound rinse for the management of mucositis, stomatitis, aphthous ulcers, and traumatic ulcers. Abeona Therapeutics Inc. has collaborations with EB Research Partnership and Epidermolysis Bullosa Medical Research Foundation that focus on gene therapy treatments for EB; and Brammer Bio for commercial translation of ABO-102. The company was formerly known as PlasmaTech Biopharmaceuticals, Inc. and changed its name to Abeona Therapeutics Inc. in June 2015. Abeona Therapeutics Inc. was incorporated in 1989 and is based in Dallas, Texas.
Eton Pharmaceuticals
NASDAQ:ETONEton Pharmaceuticals, Inc., a specialty pharmaceutical company, focuses on developing, acquiring, and commercializing pharmaceutical products for rare diseases. The company offers ALKINDI SPRINKLE, a replacement therapy for adrenocortical insufficiency in children under 17 years of age; Carglumic Acid for the treatment of acute and chronic hyperammonemia due to N-acetylglutamate Synthase deficiency; Betaine Anhydrous for the treatment of homocystinuria; and Nitisinone for the treatment of tyrosinemia type 1. It also provides Zeneo hydrocortisone autoinjector for the treatment of adrenal crisis. Eton Pharmaceuticals, Inc. was incorporated in 2017 and is based in Deer Park, Illinois.
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-107 and MB-207, 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-102 CAR T therapies for blastic plasmacytoid dendritic cell neoplasm, acute myeloid leukemia, and myelodysplastic syndrome; MB-106 CAR T cell program for B cell non-hodgkin lymphoma and chronic lymphocytic leukemia; MB-104 CAR T for multiple myeloma and light chain amyloidosis; MB-101 CAR T cell program for glioblastoma; MB-103 CAR T for glioblastoma multiforme (GBM) and metastatic breast cancer to brain; MB-105 CAR T for prostate and pancreatic cancers; and MB-108, a next-generation oncolytic herpes simplex virus. It has license agreements with Nationwide Children's Hospital, CSL Behring; Mayo Clin Mayo Clinic; Leiden University Medical Centre; SIRION Biotech GmbH, and Minaris Regenerative Medicine GmbH. The company was incorporated in 2015 and is headquartered in Worcester, Massachusetts.
Oasmia Pharmaceutical AB (publ)
NASDAQ:OASMOasmia Pharmaceutical AB engages in the research and development of drugs. It formulates pharmaceutical products for ovarian and breast cancer for humans and cancer for animals. The company was founded by Julian Aleksov and Bo Cederstrand on April 15, 1988 and is headquartered in Uppsala, Sweden.