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).
In the most simplistic view, adult cancers occur because “immature cells are exposed to mutagens, accumulate mutations, and across life ultimately transform into cancer cells,” Franck Bourdeaut told his audience at the 2026 Annual Congress of the European Hematology Association (EHA 2026). “On the contrary, in pediatric cancers, it is assumed that very few mutations are responsible for a maturation block, make these cells derail from their normal differentiation trajectory and ultimately result in an early onset typical pediatric cancer.”
Scientists at Columbia University have used base editing to make precise changes in the genomes of human embryos, avoiding the damage to chromosomes that occurs following two-stranded DNA cuts with conventional CRISPR/Cas9 editing.
Bleednfire Therapeutics has launched, having secured nondilutive funding from Innosuisse, Venture Kick, the Gebert Rüf Foundation Innobooster program and Kickfund. It is co-founded by Landmark Bioventures AG and leading KOLs from the University of Bern and Inselspital (Bern University Hospital).
Cereno Scientific AB plans to initiate preclinical disease model studies evaluating CS-585 in antiphospholipid syndrome, a rare autoimmune disease associated with recurrent blood clots and serious cardiovascular complications. The studies will be initiated this year under an ongoing research collaboration with the University of Michigan.
In a deal potentially worth up to $15.2 billion, Jiangsu Hengrui Pharmaceuticals Co. Ltd. is joining efforts with Bristol Myers Squibb Co. to advance 13 early development programs in the fields of oncology, hematology and immunology. Shanghai-based Hengrui will hold exclusive rights in mainland China, Hong Kong and Macau, while Princeton, N.J.-based BMS will hold exclusive rights in the rest of the world.
Circular RNA (circRNA) is not a new concept, but it is a novel strategy in the field of gene and cell therapy. While mRNA vaccines have revolutionized medicine, this RNA fragment without free ends surpasses their performance in both efficacy and durability, bringing it to the attention of several pioneering companies. The latest advances in circRNA presented at the 29th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) clearly surpass the performance achieved with linear mRNA.
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