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.
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.
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.
Poseida Therapeutics
NASDAQ:PSTXPoseida Therapeutics, Inc., a clinical-stage biopharmaceutical company, focuses on developing therapeutics for patients with high unmet medical needs. The company's development candidates for Heme Malignancies includes P-BCMA-ALLO1, which is in Phase I trial to treat patients with relapsed/refractory multiple myeloma; P-CD19CD20-ALLO1, which is in Phase I trial for treating B cell malignancies and other autoimmune diseases; P-BCMACD19-ALLO1, an allogeneic, off-the-shelf CAR-T product candidate in preclinical development for treating multiple myeloma; and P-CD70-ALLO1 under preclinical development to treat hematological indications. It is also involved in the development of P-MUC1C-ALLO1 that is in Phase I trial for treating a range of solid tumors, including breast, colorectal, lung, ovarian, pancreatic, and renal cancers; P-PSMA-ALLO1, an autologous chimeric antigen receptor T cell (CAR-T) product candidate that is in preclinical development for the treatment of patients with metastatic castrate resistant prostate cancer (mCRPC); and P-PSMA-101, an allogeneic CAR-T product candidate under Phase 1 clinical trial for treating mCRPC. In addition, the company engages in the development of P-FVIII-101, a clinical stage liver-directed gene therapy for the in vivo treatment of hemophilia A; P-OTC-101, a clinical stage liver-directed gene therapy for the in vivo treatment of ornithine transcarbamylase deficiency; and P-PAH-101, a clinical stage liver-directed gene therapy for the in vivo treatment of phenylketonuria. It has a research collaboration and license agreement with F. Hoffmann-La Roche Ltd, and Hoffmann-La Roche Inc. The company was incorporated in 2014 and is headquartered in San Diego, California.