Autolus Therapeutics
NASDAQ:AUTLAutolus Therapeutics plc, a clinical-stage biopharmaceutical company, develops T cell therapies for the treatment of cancer and autoimmune diseases. The company's clinical-stage programs include obecabtagene autoleucel (AUTO1), a CD19-targeting programmed T cell investigational therapy that is in Phase 1b/2 clinical trial for the treatment of adult ALL; AUTO1/22, which is in a Phase 1 clinical trial in pediatric patients with relapsed or refractory ALL; AUTO4, a programmed T cell investigational therapy for the treatment of peripheral T-cell lymphoma targeting TRBC1 and TRBC2; AUTO6NG, a programmed T cell investigational therapy targeting GD2 in development for the treatment of neuroblastoma; and AUTO8, a product candidate to treat multiple myeloma. It also focuses on developing AUTO5, a preclinical TRBC2 programmed T cell product candidate for the treatment of peripheral T-cell lymphoma. The company was incorporated in 2014 and is headquartered in London, the United Kingdom.
Precision BioSciences
NASDAQ:DTILPrecision BioSciences, Inc., a clinical stage gene editing company, develops ex vivo allogeneic chimeric antigen receptor (CAR) T immunotherapies and in vivo therapies for genetic and infectious diseases in the United States. The company offers ARCUS, a genome editing platform to cure genetic disorders. It also provides Ex vivo Allogeneic CAR T Immunotherapy, a form of immunotherapy in which T cell, a specific type of immune cell is genetically engineered to recognize and kill cancer cells; PBCAR0191, an azercabtagene zapreleucel, for the treatment of acute lymphoblastic leukemia and B-cell precursor acute lymphoblastic leukemia; PBCAR19B, an anti-CD19 CAR T candidate built on the stealth cell platform utilizing a single-step gene edit to minimize the risk of chromosome abnormalities; and PBCAR269A, an investigational allogeneic CAR T cell candidate targeting B-cell maturation antigen for relapsed or refractory (R/R) multiple myeloma in combination with nirogacestat, a gamma secretase inhibitor. The company has development and commercial license agreements with Les Laboratoires Servier to develop allogeneic CAR T cell therapies for antigen targets, hematological cancer targets beyond CD19, and solid tumor targets; Tiziana Life Sciences to evaluate foralumab, a fully human anti-CD3 monoclonal antibody as a lymphodepleting agent for the potential treatment of cancers; iECURE, Inc. to develop ARCUS-based gene-insertion therapies; and Novartis Pharma AG to discover and develop in vivo gene editing products. Precision BioSciences, Inc. was incorporated in 2006 and is headquartered in Durham, North Carolina.
Prevail Therapeutics
NASDAQ:PRVLPrevail Therapeutics Inc., a gene therapy company, focuses on developing and commercializing disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases. The company's lead product candidate is PR001, which is in Phase I/II clinical trial for the treatment of Parkinson's disease with GBA1 mutation and neuronopathic Gaucher disease. It is also developing PR006 for the treatment of frontotemporal dementia with GRN mutation; and PR004 for the treatment of synucleinopathies. Prevail Therapeutics Inc. was founded in 2017 and is based in New York, New York.
Replimune Group
NASDAQ:REPLReplimune Group, Inc., a clinical-stage biotechnology company, focuses on the development of oncolytic immunotherapies to treat cancer. The company's proprietary tumor-directed oncolytic immunotherapy product candidates are designed and intended to activate the immune system against cancer. Its lead product candidate is RP1, a selectively replicating version of HSV-1 that expresses GALV-GP R(-) and human GM-CSF, which is in Phase I/II clinical trials for a range of solid tumors; and that has completed Phase II clinical trials for treating cutaneous squamous cell carcinoma. The company is also developing RP2, which is in Phase I clinical trials to express an anti-CTLA-4 antibody-like protein to block the inhibition of the immune response otherwise caused by CTLA-4; and RP3 that is in Phase I clinical trial to express immune-activating proteins that stimulate T cells. Replimune Group, Inc. was founded in 2015 and is headquartered in Woburn, Massachusetts.
Voyager Therapeutics
NASDAQ:VYGRVoyager Therapeutics, Inc., a biotechnology company, focuses on the treatment of gene therapy and neurology diseases. The company's lead clinical candidate is VY-TAU01, an anti-tau antibody program for the treatment of alzheimer's disease. Its product pipeline includes superoxide dismutase 1 silencing gene therapy, which is in preclinical trial for the treatment of amyotrophic lateral sclerosis; tau silencing gene therapy, which is in preclinical trial for the treatment of alzheimer's disease; and vectorized anti-amyloid antibody, a gene therapy targeting anti-amyloid for the treatment of alzheimer's disease and is in preclinical trial. In addition, the company develops VY-FXN01, which is in preclinical trial to treat friedreich's ataxia; and GBA1 gene replacement to treat parkinson's disease and is in preclinical trial. Further, it provides research program for the treatment of Huntington's disease. The company has collaboration and license agreements with Alexion; AstraZeneca Rare Disease; Novartis Pharma AG; Neurocrine Biosciences, Inc; and Sangamo Therapeutics, Inc. Voyager Therapeutics, Inc. was incorporated in 2013 and is headquartered in Lexington, Massachusetts.