Hematologic

Hematopoietic stem cell research points to leukemia’s early roots

Thu, 02 Apr 2026 09:00:00 -0400

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.

Five-drug VIPOR regimen shows promise in aggressive blood cancer

Wed, 01 Apr 2026 12:00:00 -0400

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.

Hematopoietic stem cell research points to leukemia’s early roots

Wed, 01 Apr 2026 09:00:00 -0400

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.

Grant supports Epifrontier’s EPF-001 for β-globin disorders

Wed, 25 Mar 2026 09:00:00 -0400

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.

Karyopharm phase III Sentry entry bucks rux in MF; to FDA next

Tue, 24 Mar 2026 12:00:00 -0400

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).

Sanofi licenses Sino Biopharm’s JAK/ROCK drug for $1.4B

Tue, 10 Mar 2026 12:00:00 -0400

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.

Sanofi licenses Sino Biopharm’s JAK/ROCK drug for $1.4B

Wed, 04 Mar 2026 12:00:00 -0500

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.

China clears first JAK/ROCK drug for myelofibrosis

Tue, 03 Mar 2026 12:00:00 -0500

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.

Red blood cells directly control blood glucose level

Fri, 20 Feb 2026 08:00:00 -0500

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.

After thalassemia win, Agios advances mitapivat, tebapivat in SCD

Fri, 13 Feb 2026 12:00:00 -0500

Agios Pharmaceuticals Inc. is preparing to present a mixed bag of phase III Rise Up data to the U.S. FDA in hopes of “full approval” for mitapivat in sickle cell disease (SCD), which would make it its third indication in rare hematology.

Human-relevant models lining up to replace animal tests

Thu, 12 Feb 2026 12:00:00 -0500

The pressure to replace animal testing with human-relevant assays that are more predictive of human-drug responses has now reached a tipping point, and there is a movement toward greater acceptance of these potentially more translatable tests.

Tangram’s TGM-148 demonstrates pan-bleeding disorder therapeutic potential

Fri, 06 Feb 2026 08:00:00 -0500

Researchers at Tangram Therapeutics Inc. have presented preclinical safety and efficacy data for TGM-148 in a model of von Willebrand disease.

Sunshine Lake Pharma discovers PKLR activators

Wed, 28 Jan 2026 08:00:00 -0500

Sunshine Lake Pharma Co. Ltd. has divulged heterocyclic fused ring compounds acting as pyruvate kinase PKLR activators potentially useful for the treatment of anemia, abetalipoproteinemia, hemolysis, hereditary spherocytosis, sickle cell disease and thalassemia.

University of Sydney discloses coagulation factor XIa inhibitors

Tue, 27 Jan 2026 08:00:00 -0500

A University of Sydney patent details new cyclic peptides acting as coagulation factor XIa inhibitors reported to be useful for the treatment of thrombosis.

Linkcure Therapeutics divulges new WIZ degradation inducers

Mon, 12 Jan 2026 08:00:00 -0500

Linkcure Therapeutics has synthesized molecular glue degraders acting as zinc finger protein 803 (ZNF803; WIZ) degradation inducers reported to be useful for the treatment of sickle cell anemia and β-thalassemia.

Preclinical results of anti-ADAMTS13 antibody in acquired von Willebrand syndrome

Mon, 29 Dec 2025 08:00:00 -0500

The acquired von Willebrand syndrome (AVWS) is a rare bleeding disorder with laboratory findings similar to those of inherited von Willebrand disease. Researchers from the Nara Medical University and collaborating institutions presented a potential therapeutic approach for AVWS.

Gene therapy genie back in the bottle?

Wed, 24 Dec 2025 12:00:00 -0500

Gene therapy has had its commercial struggles in the past year. The cost to patients is in the millions and fewer are stepping forward for treatment than companies would like. While development continues in this game-changing field, some have struggled with regulatory authorities during development while others have just stepped away altogether.

AND-017 increases RBC and hemoglobin in sickle cell disease

Wed, 24 Dec 2025 08:00:00 -0500

Sickle cell disease (SCD) is an inherited hemoglobinopathy caused by a mutation in the gene encoding β-globin that results in hemoglobin S polymerization, red blood cell (RBC) sickling and hemolytic anemia, among others.

Hemophilia trial death with Pfizer’s Hympavzi under scrutiny

Tue, 23 Dec 2025 12:00:00 -0500

In a letter to the hemophilia community, Pfizer Inc. reported a death due to cerebellar infarction and subsequent cerebral hemorrhage in a long-term extension trial participant taking the New York-based company’s tissue factor pathway inhibitor antagonist Hympavzi (marstacimab).

LBL-076: a first-in-class trispecific TCE for refractory MM

Tue, 23 Dec 2025 08:00:00 -0500

Despite therapeutic advances, multiple myeloma (MM) remains incurable, with most patients relapsing and developing resistance, especially those refractory to proteasome inhibitors, immunomodulatory drugs and anti-CD38 antibodies. Limited options and poor prognosis highlight the need for new agents with distinct mechanisms and better safety.

DXO-1801 shows promise for inflammation-driven anemia

Mon, 22 Dec 2025 08:00:00 -0500

Dexoligo Therapeutics has presented data for their liver-targeted siRNA DXO-1801 for the potential treatment of inflammation-driven anemia, a serious complication of chronic diseases such as chronic kidney disease, myelofibrosis or advanced solid tumors characterized by elevated pro-inflammatory cytokines where the availability for iron for erythropoiesis is limited due to increased hepcidin levels.

AJ1-11095 outperforms ruxolitinib in myeloproliferative neoplasm models

Mon, 22 Dec 2025 08:00:00 -0500

JAK2 inhibitors (JAK2i) are the standard treatment for myelofibrosis (MF), offering symptom relief and reducing spleen size. However, all FDA-approved JAK2i are type I inhibitors, which fail to eliminate the mutant MPN clone, leading many patients to treatment discontinuation.

EMU-116 performs better than plerixafor in sickle cell disease

Fri, 19 Dec 2025 08:00:00 -0500

Using C-X-C chemokine receptor type 4 (CXCR4) antagonists as cell mobilization agents has resulted in some FDA approved agents, such as Plerixafor, for hematopoietic stem cell transplantation and neutropenia. Oral cell mobilizers could result in using them in conditions such as sickle-cell disease (SCD) and chronic neutropenia. Emory University has developed and presented data for their CXCR4 antagonist EMU-116.

AND-017 ameliorates anemia in MDS mice

Thu, 18 Dec 2025 08:00:00 -0500

Most patients with myelodysplastic syndrome (MDS) exhibit variable degrees of anemia due to impaired erythropoiesis. Ameliorating anemia and reducing the dependence on transfusion may enhance the quality of life of these patients and improve their survival rates.

Taigen in-licenses Insilico Medicine’s PHD inhibitor ISM-4808

Mon, 15 Dec 2025 08:00:00 -0500

Taigen Biotechnology Co. Ltd. and its Taigen Biopharmaceuticals subsidiary have entered into an exclusive in-licensing agreement with Insilico Medicine Inc. for ISM-4808, a prolyl hydroxylase domain (PHD) inhibitor.

ASH 2025: Ianalumab data offer break from chronic ITP therapy

Wed, 10 Dec 2025 12:00:00 -0500

Novartis AG’s monoclonal antibody, ianalumab (VAY-736), when added to standard-of-care eltrombopag, extended disease control of primary immune thrombocytopenia (ITP) by 45%, according to data presented Dec. 9 during a late-breaker abstract session at the 67th American Society of Hematology’s annual meeting in Orlando, Fla.

GB-3226: a first-in-class dual ENL-YEATS/FLT3 inhibitor for AML

Wed, 10 Dec 2025 08:00:00 -0500

ENL-YEATS is an epigenetic reader that sustains transcriptional programs essential for AML, whereas FLT3 mutations, present in approximately 30% of patients, drive malignant proliferation. Dual inhibition of ENL-YEATS and FLT3 may therefore more effectively disrupt complementary drivers of leukemogenesis than FLT3 targeting alone.

Exicure phase II mobilizer burixafor rallies in MM war

Tue, 09 Dec 2025 12:00:00 -0500

Exicure Inc.’s buyout early this year of GPCR Therapeutics Inc. is paying off in a big way with data from the finished phase II trial testing burixafor (GPC-100). The agent is used with propranolol and granulocyte colony-stimulating factor to mobilize hematopoietic progenitor cells in patients with multiple myeloma (MM) undergoing autologous hematopoietic cell transplant.

ASH 2025: Tec-dara shines on ‘functional cure’ Majestec-3 MM data

Tue, 09 Dec 2025 10:00:00 -0500

Impressive results of a potential second-line combination treatment for multiple myeloma from the Majestec-3 trial of teclistamab plus daratumumab raised eyebrows at the American Society of Hematology’s 67th annual meeting, with the combination showing an 83.4% rate of progression-free survival at three years vs. 29.7% for standard of care.

Positive phase Ib SCD data perk Fulcrum stock

Mon, 08 Dec 2025 12:00:00 -0500

On the heels of mixed phase III data from Agios Pharmaceuticals Inc. with mitapivat to treat sickle cell disease (SCD), Fulcrum Therapeutics Inc. wowed investors by way of initial results from the ongoing 20-mg dose cohort in the phase Ib Pioneer trial testing oral, once-daily fetal hemoglobin inducer pociredir.