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BioWorld - Wednesday, March 25, 2026
Home » Topics » Disease categories and therapies » Hematologic

Hematologic
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Red blood cells, DNA
Hematologic

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

March 25, 2026
No Comments
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.
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Lab sample and bone marrow illustration

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

March 24, 2026
By Randy Osborne
No Comments
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).
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Gold dollar sign inside gold cog

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

March 10, 2026
By Tamra Sami
No Comments
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.
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Gold dollar sign inside gold cog

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

March 4, 2026
By Tamra Sami
No Comments
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.
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Primary myelofibrosis (PMF) cells in blood flow

China clears first JAK/ROCK drug for myelofibrosis

March 3, 2026
By Tamra Sami
No Comments
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.
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Red blood cells and glucose illustration
Endocrine/metabolic

Red blood cells directly control blood glucose level

Feb. 20, 2026
By Anette Breindl
No Comments
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.
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After thalassemia win, Agios advances mitapivat, tebapivat in SCD

Feb. 13, 2026
By Marian (YoonJee) Chu
No Comments

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.


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Lab mouse

Human-relevant models lining up to replace animal tests

Feb. 12, 2026
By Nuala Moran
No Comments
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.
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3D illustration of platelets in the boodstream
Hematologic

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

Feb. 6, 2026
No Comments
Researchers at Tangram Therapeutics Inc. have presented preclinical safety and efficacy data for TGM-148 in a model of von Willebrand disease.
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Hematologic

Sunshine Lake Pharma discovers PKLR activators

Jan. 28, 2026
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.
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