Assembly Biosciences, Inc
Assembly Biosciences, Inc., a biotechnology company, develops therapeutic candidates for the treatment of viral diseases worldwide. Its pipeline includes long-acting helicase-primase inhibitors candidates, including ABI-5336 and ABI-1179, which are in Phase 1a/1b clinical studies for recurrent genital herpes; ABI-4334, a capsid assembly modulator designed to disrupt the replication cycle of hepatitis B virus that is in Phase 1b clinical study; and ABI-6250, an orally bioavailable hepatitis delta virus entry inhibitor that is in Phase 1a clinical study. The company also develops ABI-7423, an oral broad-spectrum non-nucleoside polymerase inhibitor that targets transplant-related herpesviruses. It has an option, license, and collaboration agreement with Gilead Sciences, Inc. for the research and development of virology therapies. The company was formerly known as Ventrus Biosciences, Inc. and changed its name to Assembly Biosciences, Inc. in June 2014. Assembly Biosciences, Inc. was incorporated in 2005 and is headquartered in South San Francisco, California.
Open ASMB 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 ↗