Immitra Bio GmbH has completed a CHF2.4 million (US$ 3 million) pre-seed financing round as it advances development of a platform for scalable, one-time curative in vivo gene editing therapies based on its mutation-agnostic gene editing technology and digital target identification platform.
The field of cell and gene therapy is moving toward a new phase of scalability and clinical durability, with in vivo chimeric antigen receptor (CAR) T-cell therapeutics emerging as the dark horse in what speakers at BIO Asia-Taiwan 2026 described as CAR T’s second revolution.
Investigators at the German Cancer Research Center (Deutsches Krebsforschungszentrum, DKFZ) have demonstrated that in the liver, some endothelial cells (ECs) acted as antigen-presenting cells of sorts. These lipoprotein lipase (LPL)-expressing cells cross-presented tumor antigens on their surface, alerting T cells to the presence of metastases.
Excessive fibrinolysis is a major driver of bleeding across multiple clinical settings, including heavy menstrual bleeding and other hemorrhagic disorders. Researchers from Hemab ApS presented preclinical data on HMB-003, a long-acting plasmin inhibitor that directly targets the enzyme’s active site to achieve sustained antifibrinolytic activity.
The prophylaxis of bleeding disorders usually requires frequent intravenous infusions that result in excessive treatment burden and residual bleeding risk. TGM-148 is a siRNA developed by Tangram Therapeutics plc designed to repress the hepatocyte expression of a gene target to rebalance hemostasis, and has shown efficacy in murine models of hemophilia and von Willebrand disease.
Prelude Therapeutics Inc. has identified new tyrosine-protein kinase JAK2 inhibitors potentially useful for the treatment of leukemia, polycythemia vera, myelofibrosis, essential thrombocythemia and graft-vs.-host disease.
The Advanced Research Projects Agency for Health (ARPA-H), an agency within the U.S. Department of Health and Human Services, has announced the teams for the THRIVE (Treating Hereditary Rare diseases with In Vivo prEcision genetic medicines) program. With a commitment of up to $160 million over 5 years, THRIVE aims to accelerate solutions for rare genetic pediatric diseases across multiple technological approaches, clinical trial designs and deployment models.
The alternative pathway (AP) of the complement system is crucial for innate immunity and its dysregulation may lead to several diseases, including paroxysmal nocturnal hemoglobinuria (PNH). Alnylam Pharmaceuticals Inc. and Regeneron Pharmaceuticals Inc. recently presented data on ALN-CFB, a siRNA targeting hepatic complement factor B (CFB) that inhibits the production of CFB in the liver.
Clonal hematopoiesis (CH), where few blood stem cells produce a significant fraction of mature blood cells that are genetically identical, is partly an inevitable feature of aging. Certainly, it is near universal in those older than 60. CH is not itself a disease, but 1%-2% of CH cases progress to acute myeloid leukemia, and it raises the risk of some other types of cancer as well. A total of eight genes are responsible for 95% of CH cases, George Vassiliou told the audience in Saturday’s plenary session at the 2026 Annual Congress of the European Hematology Association (EHA 2026).