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.
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.
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.
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.
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.