Arrowhead Research Corporation
Arrowhead Pharmaceuticals, Inc. develops medicines for the treatment of intractable diseases in the United States. Its pipeline includes Plozasiran to reduce production of apolipoprotein C-III in Phase 3 studies; Zodasiran to reduce production of angiopoietin-like protein 3 in Phase 3 clinical trials; ARO-DIMER-PA, a dual functional RNAi molecule in a Phase 1/2a clinical trials; and ARO-PNPLA3, an investigational RNAi therapeutic in Phase 1 clinical trials. The company also develops ARO-INHBE, to reduce the hepatic expression of the INHBE gene and its secreted gene product, Activin E in Phase 1/2a clinical trials; ARO-ALK7 to silence adipocyte expression of the ACVR1C gene in Phase 1/2a clinical trials; ARO-RAGE to reduce production of the receptor for advanced glycation end products in Phase 1/2a clinical trials; and ARO-MAPT, an investigational RNAi-based therapy. In addition, it is developing ARO-C3 to reduce production of C3 in Phase 1/2a clinical trials; and ARO-CFB to reduce hepatic expression of CFB in a Phase 1/2a clinical trials. The company has collaboration and license Agreements with Glaxosmithkline Intellectual Property (No. 3) Limited; Takeda Pharmaceutical Company Limited; Amgen Inc.; and Sarepta Therapeutics, Inc. Arrowhead Pharmaceuticals, Inc. was founded in 2001 and is headquartered in Pasadena, California.
Open ARWR dashboard ↗CRISPR Therapeutics AG
CRISPR Therapeutics AG, a gene editing company, focuses on developing gene-based medicines for serious human diseases using its Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/CRISPR-associated protein 9 (Cas9) platform. The company's CRISPR/Cas9 is a revolutionary technology for gene editing which is the process of precisely altering specific sequences of genomic DNA. It has a portfolio of therapeutic programs across a range of disease areas, including hemoglobinopathies, CAR T cell therapies, in vivo, and type 1 diabetes, as well as develops investigational CAR T programs, including an autologous, gene-edited CAR T program targeting glypican-3 (GPC3) for the potential treatment of solid tumors. The company's lead product candidate is CASGEVY, an ex vivo CRISPR/Cas9 gene-edited cell therapy for treating patients suffering from transfusion-dependent beta-thalassemia, severe sickle cell disease (SCD), and hemoglobinopathies in which a patient's hematopoietic stem and progenitor cells are edited to produce high levels of fetal hemoglobin in red blood cells. It also develops CAR T cell therapies, including CTX112 targeting cluster of differentiation 19 (CD19) and CTX131 targeting CD70 for oncology and autoimmune indications; CTX310 and CTX320, in vivo gene editing to address the cardiovascular disease by disrupting the validated targets angiopoietin-like protein 3 and lipoprotein; and CTX211, an allogeneic, gene-edited, hypoimmune stem cell-derived product candidate for the treatment of T1D. It has strategic partnerships with Vertex Pharmaceuticals Incorporated, Nkarta, Inc., and Capsida Biotherapeutics. CRISPR Therapeutics AG was incorporated in 2013 and is headquartered in Zug, Switzerland.
Open CRSP dashboard ↗