AVROBIO
NASDAQ:AVROAVROBIO, Inc., a clinical-stage gene therapy company, develops hematopoietic stem cell gene therapies to treat rare diseases in the United States. 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 and 3; AVR-RD-01, which is in phase 2 clinical trials for the treatment of Fabry disease; and AVR-RD-03 that is in preclinical stage for the treatment of Pompe disease. The company was incorporated in 2015 and is headquartered in Cambridge, Massachusetts.
Codiak BioSciences
NASDAQ:CDAKCodiak BioSciences, Inc., a clinical-stage biopharmaceutical company, focuses on developing exosome-based therapeutics for the treatment of oncology, neuro-oncology, neurology, neuromuscular diseases, infectious diseases, and other diseases. Its lead product candidates are exoSTING and exoIL-12 exosome therapeutic candidates for the treatment of various solid tumors. The company is also developing exoASO-STAT6, an antisense oligonucleotide targeting the STAT6 transcription factor; and exoVACC, a modular platform for constructing precision vaccines. It has a collaboration agreement with the Jazz Pharmaceuticals Ireland Limited, Sarepta Therapeutics, Inc., Kayla Therapeutics S.A.S., Washington University, Ragon Institute of MGH, MIT, and Harvard. The company was incorporated in 2015 and is headquartered in Cambridge, Massachusetts.
Fusion Pharmaceuticals
NASDAQ:FUSNFusion Pharmaceuticals Inc., a clinical-stage oncology company, focuses on developing radiopharmaceuticals as precision medicines in Canada and the United States. The company's lead product candidates include FPI-2265 that is in Phase 2 clinical trial to treat prostate-specific membrane antigens; and FPI-1434 that is in Phase 1 clinical trial as a monotherapy in patients with solid tumors expressing that target insulin-like growth factor 1 receptor. It also conducting additional preclinical studies of FPI-1434 in combination with approved checkpoint inhibitors and DNA damage response inhibitors to further assess the anti-tumor activity, and dosing schedule and pharmacodynamics of the combinations. In addition, the company develops FPI-1966 for the treatment of multiple cancers, including colorectal, ovarian, bladder, and head and neck cancers.; FPI-2068, a bispecific antibody, which is in Phase 1 clinical trial targeting epidermal growth factor receptor, and mesenchymal epithelial transition factor. It has a strategic collaboration agreement with AstraZeneca UK Limited to discover, develop, and commercialize alpha-emitting radiopharmaceuticals and combination therapies for the treatment of cancer. Fusion Pharmaceuticals Inc. was incorporated in 2014 and is headquartered in Hamilton, Canada.
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.
Oyster Point Pharma
NASDAQ:OYSTOyster Point Pharma, Inc., a commercial-stage biopharmaceutical company, focuses on the discovery, development, and commercialization of pharmaceutical therapies to treat ophthalmic diseases in the United States. The company's product candidate is TYRVAYA, a nicotinic acetylcholine receptor agonist for the treatment of signs and symptoms of dry eye disease. It is also developing TYRVAYA that is in Phase II clinical trial for the treatment of for neurotrophic keratopathy. The company was incorporated in 2015 and is headquartered in Princeton, New Jersey.