https://www.bioworld.com/rss en-us Although antiretroviral therapy (ART) can suppress HIV to undetectable levels, a growing body of evidence suggests that the virus leaves a lasting biological imprint. At the 26th International AIDS Conference (AIDS 2026), researchers reported persistent abnormalities affecting the gut, cardiovascular system, metabolism and immune function despite effective viral control. http://www.bioworld.com/articles/732999 Wed, 29 Jul 2026 09:00:00 -0400 https://www.bioworld.com/articles/732999-aids-2026-new-targets-for-metabolic-dysfunction-despite-art HIV virus 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. http://www.bioworld.com/articles/732825 Wed, 22 Jul 2026 09:00:00 -0400 https://www.bioworld.com/articles/732825-financing-at-immitra-bio-to-advance-in-vivo-gene-editing-therapies DNA double helix under a magnifying glass The decision by Agios Pharmaceuticals Inc. to end work with next-generation oral pyruvate kinase activator tebapivat in sickle cell disease (SCD) put pressure on the company’s first-generation Pyrukynd (mitapivat) and left Novo Nordisk A/S in pole position as the firm advances etavopivat toward an approval bid in the second half of this year. http://www.bioworld.com/articles/732751 Tue, 21 Jul 2026 12:00:00 -0400 https://www.bioworld.com/articles/732751-agios-phase-ii-tebapivat-splats-in-scd-pyrukynd-top-of-mind Sickle cell disease 3D illustration 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. http://www.bioworld.com/articles/732840 Tue, 21 Jul 2026 11:00:00 -0400 https://www.bioworld.com/articles/732840-to-viral-or-not-to-viral-in-vivo-car-t-debates-delivery-tech Red CAR T cell on blue blackground 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. http://www.bioworld.com/articles/732839 Tue, 21 Jul 2026 09:00:00 -0400 https://www.bioworld.com/articles/732839-to-viral-or-not-to-viral-in-vivo-car-t-debates-delivery-tech Red CAR T cell on blue blackground 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. http://www.bioworld.com/articles/732779 Tue, 21 Jul 2026 09:00:00 -0400 https://www.bioworld.com/articles/732779-vascular-endothelial-cells-can-present-tumor-antigens Illustration of tumor 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. http://www.bioworld.com/articles/732736 Mon, 20 Jul 2026 12:00:00 -0400 https://www.bioworld.com/articles/732736-to-viral-or-not-to-viral-in-vivo-car-t-debates-delivery-tech Red CAR T cell on blue blackground 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. http://www.bioworld.com/articles/732673 Thu, 16 Jul 2026 09:00:00 -0400 https://www.bioworld.com/articles/732673-hmb-003-outperforms-tranexamic-acid-in-fibrinolysis-models 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. http://www.bioworld.com/articles/732643 Wed, 15 Jul 2026 09:00:00 -0400 https://www.bioworld.com/articles/732643-preclinical-data-support-continued-development-of-tgm-148-for-bleeding-disorders Takeda Pharmaceutical Co. Ltd.’s anti-CD38 antibody mezagitamab (TAK-079) is showing benefits beyond platelet restoration, with new data suggesting the candidate may improve quality of life for patients with chronic immune thrombocytopenia (ITP) and sustain those gains after treatment ends. http://www.bioworld.com/articles/732669 Tue, 14 Jul 2026 12:00:00 -0400 https://www.bioworld.com/articles/732669-takedas-mezagitamab-adds-quality-of-life-gains-to-itp-responses Antibodies and red blood cells Takeda Pharmaceutical Co. Ltd.’s anti-CD38 antibody mezagitamab (TAK-079) is showing benefits beyond platelet restoration, with new data suggesting the candidate may improve quality of life for patients with chronic immune thrombocytopenia (ITP) and sustain those gains after treatment ends. http://www.bioworld.com/articles/732586 Mon, 13 Jul 2026 12:00:00 -0400 https://www.bioworld.com/articles/732586-takedas-mezagitamab-adds-quality-of-life-gains-to-itp-responses Antibodies and red blood cells 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. http://www.bioworld.com/articles/732577 Mon, 13 Jul 2026 09:00:00 -0400 https://www.bioworld.com/articles/732577-jak2-inhibitors-reported-in-prelude-therapeutics-patent 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. http://www.bioworld.com/articles/732544 Fri, 10 Jul 2026 09:00:00 -0400 https://www.bioworld.com/articles/732544-arpa-h-program-supports-advances-for-rare-genetic-diseases Orca Bio Inc. is bringing to market a precision-engineered cell therapy approach designed to improve outcomes in patients with hematologic malignancies undergoing allogeneic hematopoietic stem cell transplantation (HSCT) following U.S. FDA approval of Tregzi, a first-of-its-kind, personalized cell therapy. http://www.bioworld.com/articles/732255 Wed, 01 Jul 2026 12:00:00 -0400 https://www.bioworld.com/articles/732255-orcas-t-cell-therapy-approach-to-hsct-wins-fda-nod-as-tregzi FDA approval seal on blue glittering background Following an initial rebuff in February 2026, the EMA’s Committee for Medicinal Products for Human Use (CHMP) is recommending approval of Acadia Pharmaceuticals Inc.’s Daybu (trofinetide) for treating neurobehavioral symptoms of Rett syndrome, after it took another look at the file. http://www.bioworld.com/articles/732227 Mon, 29 Jun 2026 12:00:00 -0400 https://www.bioworld.com/articles/732227-ema-reverses-its-stance-on-acadias-rett-syndrome-therapy-daybu EU flags at European Commission building Vascular-type bleeding disorder (BDVAS) is a rare, autosomal dominant disorder mainly caused by impaired vascular integrity. http://www.bioworld.com/articles/732118 Tue, 23 Jun 2026 09:00:00 -0400 https://www.bioworld.com/articles/732118-case-report-links-emapold1-em-variant-to-vascular-type-bleeding-disorder Illustration of red and white blood cells in an artery 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. http://www.bioworld.com/articles/732096 Mon, 22 Jun 2026 09:00:00 -0400 https://www.bioworld.com/articles/732096-preclinical-data-on-aln-cfb-for-paroxysmal-nocturnal-hemoglobinuria-presented Photo of pipette with test tube Treatment with first-generation CAR T cells regularly sent patients to the intensive care unit. Now, investigators are envisioning a future where CAR T treatment could occur on an outpatient basis. http://www.bioworld.com/articles/731988 Tue, 16 Jun 2026 11:00:00 -0400 https://www.bioworld.com/articles/731988-viva-in-vivo-at-eha-in-vivo-car-t-data-continues-to-impress CAR T and red blood cells 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). http://www.bioworld.com/articles/731978 Tue, 16 Jun 2026 09:00:00 -0400 https://www.bioworld.com/articles/731978-for-clonal-hematopoiesis-epigenetics-can-be-in-drivers-seat Red blood cells, DNA 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). http://www.bioworld.com/articles/731932 Mon, 15 Jun 2026 09:00:00 -0400 https://www.bioworld.com/articles/731932-for-clonal-hematopoiesis-epigenetics-can-be-in-drivers-seat Red blood cells, DNA Treatment with first-generation CAR T cells regularly sent patients to the intensive care unit. Now, investigators are envisioning a future where CAR T treatment could occur on an outpatient basis. At Sunday’s late-breaking oral session of the 2026 Annual Congress of the European Hematology Association (EHA2026), Lei Fan told his audience that the first-in-human data “support further development of LB-2501 as a potential first-in-class, off-the-shelf, single infusion, no lymphodepletion, outpatient use CAR T therapy.” Fan is a professor of hematology at the First Affiliated Hospital of Nanjing Medical University. http://www.bioworld.com/articles/731976 Sun, 14 Jun 2026 11:00:00 -0400 https://www.bioworld.com/articles/731976-viva-in-vivo-at-eha-in-vivo-car-t-data-continues-to-impress CAR T and red blood cells 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.” http://www.bioworld.com/articles/731957 Fri, 12 Jun 2026 09:00:00 -0400 https://www.bioworld.com/articles/731957-eha-2026-with-new-lenses-a-changing-view-on-pediatric-cancers Genome sequence map Tolerability is a key factor in the appeal of Enliven Therapeutics Inc.’s ELVN-001 for previously treated chronic myeloid leukemia (CML), which yielded positive updated phase I data from the Enable trial. Dennis Kim, hematologist from Princess Margaret Cancer Center in Toronto, said he would use the drug for a patient “who doesn’t have any other options.” http://www.bioworld.com/articles/731799 Thu, 11 Jun 2026 12:00:00 -0400 https://www.bioworld.com/articles/731799-enlivens-phase-i-cml-data-enable-phase-iii-with-bcr-abl1-bid Microscopic image of chronic myeloid leukemia 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. http://www.bioworld.com/articles/731851 Wed, 10 Jun 2026 09:00:00 -0400 https://www.bioworld.com/articles/731851-columbia-researchers-use-base-editing-to-modify-human-embryo-genome DNA double helix under a magnifying glass Agios Pharmaceuticals Inc.’s potential $665 million deal for Oscotec Inc.’s cevidoplenib, a phase III-ready oral SYK inhibitor to treat immune thrombocytopenia, marks a new chapter of development focused on cancer therapy resistance for Oscotec, CEO Yoon Tae-young said. http://www.bioworld.com/articles/731828 Tue, 09 Jun 2026 12:00:00 -0400 https://www.bioworld.com/articles/731828-disciplined-665m-deal-moves-oscotecs-cevidoplenib-to-agios Deal handshake with coin, chart background China’s National Medical Products Administration (NMPA) on June 4 granted conditional approvals to two oncology drugs from Lupeng Pharmaceutical Co. Ltd. and Vcare Pharmatech Co. Ltd., as well as one hemophilia drug developed by Staidson Biopharmaceutical Co. Ltd. http://www.bioworld.com/articles/731712 Tue, 09 Jun 2026 12:00:00 -0400 https://www.bioworld.com/articles/731712-nmpa-gives-conditional-nods-to-drugs-by-lupeng-vcare-staidson Red wooden approved stamp 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. http://www.bioworld.com/articles/731708 Tue, 09 Jun 2026 12:00:00 -0400 https://www.bioworld.com/articles/731708-columbia-researchers-use-base-editing-to-modify-human-embryo-genome DNA double helix under a magnifying glass In its largest acquisition to date, Incyte Inc. snagged rights to phase III-stage VGA-039, a monoclonal antibody the firm said could offer a new standard of care in von Willebrand disease (VWD), in a buyout of Vega Therapeutics Inc. that includes a $1.25 billion up-front payment and up to $750 million tied to the achievement of sales milestones. The announcement was followed shortly by news of another buyout, Johnson & Johnson’s deal to acquire Firefly Bio Inc. for $1 billion in cash, both moves continuing this year’s trend of larger, more strategic M&A transactions. http://www.bioworld.com/articles/731689 Mon, 08 Jun 2026 12:00:00 -0400 https://www.bioworld.com/articles/731689-incyte-nabs-potential-vwd-game-changer-in-vega-deal-j-and-j-buys-firefly Collage of businesspeople Agios Pharmaceuticals Inc.’s potential $665 million deal for Oscotec Inc.’s cevidoplenib, a phase III-ready oral SYK inhibitor to treat immune thrombocytopenia, marks a new chapter of development focused on cancer therapy resistance for Oscotec, CEO Yoon Tae-young said. http://www.bioworld.com/articles/731632 Fri, 05 Jun 2026 12:00:00 -0400 https://www.bioworld.com/articles/731632-disciplined-665m-deal-moves-oscotecs-cevidoplenib-to-agios Deal handshake with coin, chart background Shares of Fulcrum Therapeutics Inc. (NASDAQ:FULC) fell 54% after the company said it is discontinuing work on sickle cell disease (SCD) candidate pociredir, its only clinical-stage candidate, and reviewing strategic alternatives in the wake of the U.S. FDA’s safety concerns regarding the drug target. http://www.bioworld.com/articles/731532 Tue, 02 Jun 2026 12:00:00 -0400 https://www.bioworld.com/articles/731532-fulcrum-drops-work-on-scd-drug-pociredir-stock-plummets Sickle cell illustration