Adverum Biotechnologies
NASDAQ:ADVMAdverum Biotechnologies, Inc., a clinical-stage company, develops gene therapy product candidates to treat ocular diseases. Its lead product candidate is ixoberogene soroparvovec (ADVM-022), a single intravitreal injection gene therapy candidate used for the treatment of patients with wet age-related macular degeneration and diabetic macular edema which is in phase 2 clinical trials. The company is developing ADVM-062 (AAV.7m8-L-opsin), a novel gene therapy product candidate for the treatment of blue cone monochromacy via a single IVT injection. Adverum Biotechnologies, Inc. has license and collaboration agreements with University of California; GenSight Biologics; Lexeo Therapeutics; and Virovek, Inc. The company was formerly known as Avalanche Biotechnologies, Inc. and changed its name to Adverum Biotechnologies, Inc. in May 2016. Adverum Biotechnologies, Inc. was incorporated in 2006 and is headquartered in Redwood City, California.
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.
Elevation Oncology
NASDAQ:ELEVElevation Oncology, Inc., an oncology company, focuses on the discovery and development of cancer therapies to treat patients across a range of solid tumors with significant unmet medical needs. The company's lead candidate is EO-3021, an antibody-drug conjugate designed to target Claudin 18.2, a clinically validated molecular target. Its EO-3021 selectively delivers a cytotoxic payload directly to cancer cells expressing Claudin 18.2. Elevation Oncology, Inc. has a license agreement with CSPC Megalith Biopharmaceutical Co., Ltd. to develop and commercialize EO-3021. The company was formerly known as 14ner Oncology, Inc. and changed its name to Elevation Oncology, Inc. in February 2020. The company was incorporated in 2019 and is based in New York, New York.
Orchard Therapeutics
NASDAQ:ORTXOrchard Therapeutics plc, a gene therapy company, research, develops, and commercialize hematopoietic stem cell and gene therapies in the United Kingdom, Italy, France, and Germany. It offers OTL-200, an ex vivo autologous hematopoietic stem cell gene therapy for the treatment of patients with metachromatic leukodystrophy under the Libmeldy brand; and Strimvelis, a gammaretroviral vector-based gene therapy for the treatment of adenosine deaminase deficiency. The company's program for neurodegenerative disorders comprises clinical proof of concept-stage program, which includes OTL-203 for the treatment of mucopolysaccharidosis type I and OTL-201 for mucopolysaccharidosis type IIIA, or MPS-IIIA; and pre-clinical program, OTL-204 for the treatment of frontotemporal dementia with progranulin mutations. In addition, it develops program for immunological disorders consist of pre-clinical programs, which includes OTL-104 for Crohn's disease with mutations in the nucleotide-binding oligomerization domain-containing protein 2; and OTL-105 for the treatment of hereditary angioedema. The company was formerly known as Orchard Rx Limited. Orchard Therapeutics plc was founded in 2015 and is headquartered in London, the United Kingdom. As of January 24, 2024, Orchard Therapeutics plc operates as a subsidiary of Kyowa Kirin Co., Ltd..
TScan Therapeutics
NASDAQ:TCRXTScan Therapeutics, Inc., a clinical-stage biopharmaceutical company, develops T cell receptor-engineered T cell therapies for the treatment of patients with cancer. It is developing TSC-100 and TSC-101 that is in Phase I of clinical trial for the treatment of patients with hematologic malignancies to eliminate residual leukemia and prevent relapse after hematopoietic stem cell transplantation; and TSC-204 that is in Phase I for the treatment of solid tumors. The company also developing TSC-200, TSC-201, TSC-202, TSC-203, and TSC-205 for the treatment of solid tumors. In addition, it engages in the developing vaccines for infectious diseases, such as SARS-CoV-2. The company has a collaboration and license agreement with Novartis Institutes for BioMedical Research, Inc., to identify novel cancer antigens from the T cells of patients with a certain specific type of cancer; and a collaboration agreement with Amgen to identify novel targets in Crohn's disease. TScan Therapeutics, Inc. was incorporated in 2018 and is headquartered in Waltham, Massachusetts.