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.
Black Diamond Therapeutics
NASDAQ:BDTXBlack Diamond Therapeutics, Inc., a clinical-stage precision oncology medicine company, focuses on the discovery and development of therapies for patients with genetically defined cancers. The company's lead product candidate, BDTX-1535, a brain-penetrant, mutant selective, irreversible EGFR MasterKey inhibitor, designed to inhibit a family of oncogenic mutations, currently under Phase 1 clinical trial. It also develops BDTX-4933, a brain penetrant RAF MasterKey inhibitor, designed to be a brain penetrant and highly selective and potent inhibitor of oncogenic BRAF Class I, II, III and active RAF dimers, expected to initiate Phase 1 clinical trial. The company was formerly known as ASET Therapeutics, Inc. and changed its name to Black Diamond Therapeutics, Inc. in January 2018. Black Diamond Therapeutics, Inc. was incorporated in 2014 and is headquartered in Cambridge, Massachusetts.
INmune Bio
NASDAQ:INMBINmune Bio, Inc., a clinical-stage immunology company, focuses on reprogramming the patient's innate immune system to treat cancer and neurodegenerative diseases. It intends to develop and commercialize product candidates to treat hematologic malignancies, solid tumors, and chronic inflammation. The company's development programs include INKmune, which focuses on treating women with relapse refractory ovarian carcinoma solid tumor and patients with high-risk myelodysplastic syndrome; INB03 to treat patients with cancers that express MUC4, a mucinous polyglucan on the surface of some epithelial cancer cells that appears to predict resistant to immunotherapy, including women with MUC4 expressing HER2+ breast cancer and other MUC4 resistant cancers; and XPro1595 for the treatment of Alzheimer's disease and Treatment Resistant Depression. It has license agreements with Xencor, Inc.; Immune Ventures, LLC; and University of Pittsburg. The company was incorporated in 2015 and is headquartered in Boca Raton, Florida.
Opthea
NASDAQ:OPTOpthea Limited, a clinical stage biopharmaceutical company, engages in the development and commercialization of therapies primarily for eye disease in Australia. The company's development activities are based on the intellectual property portfolio covering Vascular Endothelial Growth Factors (VEGF) VEGF-C, VEGF-D, and VEGF Receptor-3 for the treatment of diseases associated with blood and lymphatic vessel growth, as well as vascular leakage. Its lead product candidate is Sozinibercept (OPT 302), a soluble form of vascular endothelial growth factor receptor-3 VEGFR-3, currently under Phase 3 clinical development as a novel therapy for wet age-related macular degeneration and diabetic macular edema. The company was formerly known as Circadian Technologies Limited and changed its name to Opthea Limited in December 2015. Opthea Limited was incorporated in 1984 and is based in South Yarra, Australia.