https://www.bioworld.com/rss en-us 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 Hematopoietic stem cell research over the past century has shown that leukemia may be driven by an invisible hand of inflammation. The bone marrow and inflammation, then, may hold the keys to preventing blood cancers, according to John E. Dick’s plenary session at the 2026 Korean Society of Hematology International Conference, held March 26, 2026. http://www.bioworld.com/articles/730205 Tue, 07 Apr 2026 11:00:00 -0400 https://www.bioworld.com/articles/730205-hematopoietic-stem-cell-research-points-to-leukemias-early-roots Cancer and blood cells Deep molecular advances are enabling precision medicine for the field of hematology, Wyndham Wilson said during a plenary session at the 2026 Korean Society of Hematology International Conference March 26. http://www.bioworld.com/articles/730204 Tue, 07 Apr 2026 11:00:00 -0400 https://www.bioworld.com/articles/730204-five-drug-vipor-regimen-shows-promise-in-aggressive-blood-cancer PET scans showing before and after treatment with VIPOR Before (top) and after (bottom) treatment with VIPOR. Full-body and cross-sectional PET scans of a patient show large lymphoma tumors (circled in red) that have disappeared with treatment. Credit: Center for Cancer Research/National Cancer Institute Hematopoietic stem cell research over the past century has shown that leukemia may be driven by an invisible hand of inflammation. The bone marrow and inflammation, then, may hold the keys to preventing blood cancers, according to John E. Dick’s plenary session at the 2026 Korean Society of Hematology International Conference, held March 26, 2026. http://www.bioworld.com/articles/730162 Thu, 02 Apr 2026 09:00:00 -0400 https://www.bioworld.com/articles/730162-hematopoietic-stem-cell-research-points-to-leukemias-early-roots Cancer and blood cells Deep molecular advances are enabling precision medicine for the field of hematology, Wyndham Wilson said during a plenary session at the 2026 Korean Society of Hematology International Conference March 26. http://www.bioworld.com/articles/729980 Wed, 01 Apr 2026 12:00:00 -0400 https://www.bioworld.com/articles/729980-five-drug-vipor-regimen-shows-promise-in-aggressive-blood-cancer PET scans showing before and after treatment with VIPOR Before (top) and after (bottom) treatment with VIPOR. Full-body and cross-sectional PET scans of a patient show large lymphoma tumors (circled in red) that have disappeared with treatment. Credit: Center for Cancer Research/National Cancer Institute Hematopoietic stem cell (HSC) research over the past century has shown that leukemia may be driven by an invisible hand of inflammation. The bone marrow and inflammation, then, may hold the keys to preventing blood cancers, according to John E. Dick’s plenary session at the 2026 Korean Society of Hematology International Conference (ICKSH 2026), held March 26, 2026. Work in Dick’s lab has found acute myeloid leukemia (AML) HSCs that harbor preleukemic mutations long before any disease diagnosis. These insights have enabled predictive models that could identify individuals at elevated AML risk years before the onset of outright disease, opening the door to new prevention strategies. http://www.bioworld.com/articles/730055 Wed, 01 Apr 2026 09:00:00 -0400 https://www.bioworld.com/articles/730055-hematopoietic-stem-cell-research-points-to-leukemias-early-roots Cancer and blood cells Epifrontier Therapeutics Inc. has been awarded a grant of up to $32 million in nondilutive funding from the Japan Agency for Medical Research and Development (AMED) to advance the clinical development of EPF-001 (RK-701), a first-in-class G9a inhibitor being developed for sickle cell disease and β-thalassemia. http://www.bioworld.com/articles/729877 Wed, 25 Mar 2026 09:00:00 -0400 https://www.bioworld.com/articles/729877-grant-supports-epifrontiers-epf-001-for-globin-disorders Red blood cells, DNA What one analyst called an “intriguing” overall survival signal in phase III results has Karyopharm Therapeutics Inc. planning to meet with the U.S. FDA about a would-be sNDA filing for Xpovio (selinexor) in myelofibrosis (MF). http://www.bioworld.com/articles/729824 Tue, 24 Mar 2026 12:00:00 -0400 https://www.bioworld.com/articles/729824-karyopharm-phase-iii-sentry-entry-bucks-rux-in-mf-to-fda-next Lab sample and bone marrow illustration On the heels of China’s approval of Sino Biopharmaceutical Ltd.’s rovadicitinib, Sanofi SA is now inlicensing the first-in-class dual JAK/ROCK inhibitor in a deal worth more than $1.4 billion. http://www.bioworld.com/articles/729584 Tue, 10 Mar 2026 12:00:00 -0400 https://www.bioworld.com/articles/729584-sanofi-licenses-sino-biopharms-jak-rock-drug-for-14b Gold dollar sign inside gold cog On the heels of China’s approval of Sino Biopharmaceutical Ltd.’s rovadicitinib, Sanofi SA is now inlicensing the first-in-class dual JAK/ROCK inhibitor in a deal worth more than $1.4 billion. http://www.bioworld.com/articles/729302 Wed, 04 Mar 2026 12:00:00 -0500 https://www.bioworld.com/articles/729302-sanofi-licenses-sino-biopharms-jak-rock-drug-for-14b Gold dollar sign inside gold cog China’s National Medical Products Administration has approved Sino Biopharmaceutical Ltd.’s rovadicitinib, branded as Anxu, for first-line treatment of adults with intermediate- or high-risk primary myelofibrosis, as well as post polycythemia vera myelofibrosis or post-essential thrombocythemia myelofibrosis. http://www.bioworld.com/articles/729288 Tue, 03 Mar 2026 12:00:00 -0500 https://www.bioworld.com/articles/729288-china-clears-first-jak-rock-drug-for-myelofibrosis Primary myelofibrosis (PMF) cells in blood flow On the average, humans – and pigs, and deer, and birds – who live at high altitudes have better blood glucose control than their counterparts near sea level. In work published in the Feb. 19, 2026, issue of Cell Metabolism, investigators have linked this phenomenon to red blood cells that directly take up and metabolize glucose from the blood under low oxygen conditions. Cell Metabolism, investigators have linked this phenomenon to red blood cells that directly take up and metabolize glucose from the blood under low oxygen conditions.]]> http://www.bioworld.com/articles/728951 Fri, 20 Feb 2026 08:00:00 -0500 https://www.bioworld.com/articles/728951-red-blood-cells-directly-control-blood-glucose-level Red blood cells and glucose illustration Red blood cells act as a previously unrecognized sink for blood glucose, absorbing sugar from the bloodstream at a scale that can influence whole-body metabolism. Credit: Chiara Ricci-Tam