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.
Cellectis
NASDAQ:CLLSCellectis S.A., a clinical stage biotechnological company, develops immuno-oncology products based on gene-edited T-cells that express chimeric antigen receptors to target and eradicate cancer cells. The company is developing UCART19, an allogeneic T-cell product candidate for the treatment of CD19-expressing hematologic malignancies, such as acute lymphoblastic leukemia; ALLO-501 and ALLO-501A to treat relapsed or refractory for non-hodgkin lymphoma (NHL); and ALLO-715 for the treatment of multiple myeloma. It also develops UCART22 to treat B-cell acute lymphoblastic leukemia; UCARTCS1 and ALLO-605 for the treatment of multiple myeloma; ALLO-316 for renal cell carcinoma; UCART123 for the treatment of acute myeloid leukemia; and UCART 20x22 for relapsed or refractory B-Cell NHL. The company has strategic alliances with Allogene Therapeutics, Inc. and Les Laboratoires Servier; research collaboration and exclusive license agreement with Iovance Biotherapeutics; and collaboration and license agreement with Cytovia, as well as a collaboration agreement with AstraZeneca to develop novel cell and gene therapy candidate products. Cellectis S.A. was founded in 1999 and is headquartered in Paris, France.
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.
Mesoblast
NASDAQ:MESOMesoblast Limited engages in the development of regenerative medicine products in Australia, the United States, Singapore, and Switzerland. The company offers products in the areas of cardiovascular, spine orthopedic disorder, oncology, hematology, and immune-mediated and inflammatory diseases. Its proprietary regenerative medicine technology platform is based on specialized cells known as mesenchymal lineage cells. The company offers Remestemcel-L that is in Phase III clinical trials for the treatment of systemic inflammatory diseases, including steroid refractory acute graft versus host disease, acute respiratory distress syndrome, and biologic refractory inflammatory bowel disease; and Remestemcel-L, which is in Phase III clinical trials to treat chronic heart failure and chronic low back pain due to degenerative disc disease. It is also developing MPC-300-IV to treat biologic refractory rheumatoid arthritis diabetic nephropathy; and MPC-25-IC for the treatment or prevention of acute myocardial infarction. It has strategic partnerships with Tasly Pharmaceutical Group to offer MPC-150-IM for heart failure and MPC-25-IC for heart attacks in China; JCR Pharmaceuticals Co. Ltd. to treat wound healing in patients with epidermolysis bullosa; and Grünenthal to develops and commercializes cell therapy for the treatment of chronic low back pain. The company was incorporated in 2004 and is headquartered in Melbourne, Australia.
Telesis Bio
NASDAQ:TBIOTelesis Bio, Inc., a synthetic biology company, manufactures and sells synthetic biology instruments, reagents, and associated products and related services, primarily to pharmaceutical and academic laboratories worldwide. Its solutions include BioXp system, including BioXp 3250 system, BioXp 9600 system, and BioXp DBC, which are empowers researchers to go from a digital DNA sequence to endpoint-ready synthetic DNA; BioXp portal, an online portal that offers an intuitive guided workflow and design tools for building new DNA sequences and assembling them into vectors of choice; BioXp kits, such as BioXp De Novo kits, BioXp select kits, BioXp next generation sequencing kits, and BioXp DBC kits, which contains building blocks and reagents, including its Gibson Assembly branded reagents, for specific synthetic biology workflow applications; Benchtop reagents that contain all the reagents necessary to proceed with a specific synthetic biology workflow on the benchtop using products generated on the BioXp system; and short oligo ligation assembly enzymatic DNA synthesis designs to significantly reduce timelines for constructing synthetic DNA, RNA, and proteins compared to traditional chemical synthesis, paving the way for more efficient and effective development of mRNA-based vaccines, diagnostics, therapeutics, and personalized medicines. It also serves government institutions, contract research organizations, and synthetic biology companies. The company was formerly known as Codex DNA, Inc. and changed its name to Telesis Bio Inc. in November 2022. Telesis Bio, Inc. was incorporated in 2011 and is headquartered in San Diego, California.