https://www.bioworld.com/rss en-us 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 Payoff for the November 2023 buyout of Immunogen Inc. came for Abbvie Inc. in the form of U.S. FDA clearance for the CD123-targeting antibody-drug conjugate Decnupaz (pivekimab sunirine) to treat adults with blastic plasmacytoid dendritic cell neoplasm (BPDCN), an ultra-rare, aggressive and quick-killing hematologic malignancy. http://www.bioworld.com/articles/731440 Thu, 28 May 2026 12:00:00 -0400 https://www.bioworld.com/articles/731440-elzonris-this-fda-clears-abbvies-decnupaz-in-bpdcn Blood cells and destruction of cancer cell 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). http://www.bioworld.com/articles/731474 Thu, 28 May 2026 09:00:00 -0400 https://www.bioworld.com/articles/731474-bleednfire-launches-to-advance-bnf-001-for-rare-bleeding-disorders Illustration of blood vessel injury being repaired by red blood cells and platelets 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. http://www.bioworld.com/articles/731234 Thu, 21 May 2026 09:00:00 -0400 https://www.bioworld.com/articles/731234-cereno-scientific-to-study-cs-585-for-antiphospholipid-syndrome Illustration of clot forming in blood vessel 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. http://www.bioworld.com/articles/731267 Thu, 14 May 2026 09:00:00 -0400 https://www.bioworld.com/articles/731267-advancing-the-best-of-both-bms-hengrui-enter-152b-deal Test tubes, dropper and capsules A few months after winning its first regulatory clearance in China, Beone Medicines Inc.’s next-generation BCL2 inhibitor, sonrotoclax, gained the U.S. FDA’s accelerated approval for use in patients with relapsed or refractory mantle cell lymphoma (MCL) following treatment with a BTK inhibitor. The therapy, branded Beqalzi, marks the first BCL2-targeting drug for MCL in the U.S., but the company has pointed to a bigger opportunity in the potential combination with blockbuster BTK drug Brukinsa (zanubrutinib). http://www.bioworld.com/articles/731062 Wed, 13 May 2026 12:00:00 -0400 https://www.bioworld.com/articles/731062-beones-bcl2-drug-beqalzi-wins-fda-nod-in-mantle-cell-lymphoma Mantle cell lymphoma 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. http://www.bioworld.com/articles/731268 Wed, 13 May 2026 09:00:00 -0400 https://www.bioworld.com/articles/731268-asgct-2026-circular-rna-the-new-beast-in-gene-and-cell-therapy Illustration of a glowing circle to represent circRNA 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. The deal includes four oncology/hematology assets from Hengrui, four immunology assets from BMS, and five assets that the two companies will jointly discover and develop. http://www.bioworld.com/articles/731047 Tue, 12 May 2026 12:00:00 -0400 https://www.bioworld.com/articles/731047-advancing-the-best-of-both-bms-hengrui-enter-152b-deal Test tubes, dropper and capsules Continuing biopharma’s IPO resurgence in 2026, Hemab Therapeutics Holdings Inc. priced an upsized offering of 16.75 million shares at $18 per share, the high end of its proposed price range, for gross proceeds of $301.5 million. Another $45 million could come if underwriters exercise their full overallotment. http://www.bioworld.com/articles/730765 Fri, 01 May 2026 12:00:00 -0400 https://www.bioworld.com/articles/730765-hemab-prices-301m-in-latest-upsized-biopharma-ipo IPO and digital hologram with business icons Ferrosa Therapeutics AG has announced a $3.5 million seed financing to support the development of a first-in-class bispecific antibody (FRS-101) to treat anemia of inflammation across chronic kidney disease, autoimmune disease and oncology indications. http://www.bioworld.com/articles/730634 Fri, 24 Apr 2026 09:00:00 -0400 https://www.bioworld.com/articles/730634-seed-funding-at-ferrosa-therapeutics Coins and seedling Minimal residual disease (MRD) has become a central concept in modern oncology, reshaping how clinicians evaluate response, relapse risk and treatment precision. As increasingly sensitive technologies reveal traces of cancer that persist after therapy, MRD is emerging as both a biological challenge and a clinical opportunity, especially as new data illuminate its complexity across hematologic and solid tumors. This topic was addressed at the 2026 American Association for Cancer Research (AACR) annual meeting. http://www.bioworld.com/articles/730633 Fri, 24 Apr 2026 09:00:00 -0400 https://www.bioworld.com/articles/730633-detecting-the-invisible-minimal-residual-disease-at-aacr-2026 Illustration of a tumor Novo Nordisk A/S reported top-line results from the phase III Hibiscus study of its pyruvate kinase-R activator, etavopivat, which was acquired through the 2022 buyout of Forma Therapeutics Holdings Inc. The results set up a potential competition for patients afflicted by sickle cell disease with fellow PKR activator mitapivat from Agios Pharmaceuticals Inc. http://www.bioworld.com/articles/730468 Mon, 20 Apr 2026 12:00:00 -0400 https://www.bioworld.com/articles/730468-novos-forma-acquisition-pays-off-with-mitapivat-competitor Sickle cell illustration Alethio Therapeutics Inc. has unveiled ATX-011, a mutation-agnostic antibody for essential thrombocythemia. ATX-011 is a potential first-in-class monoclonal antibody based on a new target identified on mutant stem cells that drive myeloproliferative neoplasm disease progression. http://www.bioworld.com/articles/730427 Thu, 16 Apr 2026 09:00:00 -0400 https://www.bioworld.com/articles/730427-alethio-unveils-atx-011-for-essential-thrombocythemia 3D illustration of platelets in the boodstream