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Home » Topics » Disease categories and therapies » Hematologic

Hematologic
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D-rendered image showing atlas of human embryonic skeletal development
Drug design, drug delivery & technologies

More than 100M cells included in the human cell atlas

Nov. 21, 2024
By Mar de Miguel
An international consortium of thousands of scientists is creating the Human Cell Atlas, a three-dimensional map of all the cells in the body. The goal is to understand all the cells that make up human tissues, organs and systems, which will enable multiple medical applications. This collection of cell maps is openly available for navigation at single-cell resolution, identified through omics analyses that reveal the tridimensional distribution of each cell.
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Global handshake silhouette

Kura, Kyowa Kirin to co-develop ziftomenib in $1.49B deal

Nov. 21, 2024
By Tamra Sami
Kura Oncology Inc. and Kyowa Kirin Co. Ltd. have joined hands in a global strategic collaboration worth $1.49 billion to develop and commercialize ziftomenib, Kura’s selective oral menin inhibitor for treating patients with acute myeloid leukemia (AML) and other hematologic malignancies.
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Hematologic

Kalvista describes new FXIIa inhibitors

Nov. 15, 2024
Kalvista Pharmaceuticals Ltd. has identified coagulation factor XII (FXIIa) inhibitors reported to be useful for the treatment of hereditary angioedema, atherosclerosis, inflammatory disorders, neurodegeneration, diabetic retinopathy, SARS-CoV-2 infection (COVID-19), stroke and thrombotic disorders, among others.
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AI-generated image of blood cells in a bone marrow biopsy

No RARA, Syros trouble: MDS phase III sinks tamibarotene

Nov. 13, 2024
By Randy Osborne
Syros Pharmaceuticals Inc.’s phase III failure with oral retinoic acid receptor alpha agonist tamibarotene in myelodysplastic syndrome (MDS) meant not only severe stock damage but also defaulting on the loan facility with Oxford Finance LLC, which means an obligation may be accelerated for the company of about $43.6 million, including principal, interest, and other amounts, according to an SEC filing.
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Menin suits? Syndax’s revumenib phase II win in AML measured

Nov. 12, 2024
By Randy Osborne
The competitive menin-inhibitor space chalked further data from Syndax Pharmaceuticals Inc., which disclosed positive top-line results from the pivotal phase II portion of the Augment-101 study, designed to test oral small-molecule revumenib for safety and efficacy. But shares of the firm (NASDAQ:SNDX) closed Nov. 12 at $16.21, down $5.57, or 26%, after the Augment-101 numbers were disclosed.
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Hematologic

Hepaitech (Beijing) Biopharma Technology reports new MRGPRX4 inhibitors

Nov. 8, 2024
Work at Hepaitech (Beijing) Biopharma Technology Co. Ltd. has led to the identification of Mas-related G-protein coupled receptor member X4 (MRGPRX4; SNSR5; SNSR6) antagonists reported to be useful for the treatment of anemia, fungal infections, HIV infections, renal disorders, liver diseases, psoriasis, pruritus and urticaria, among others.
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Illustration of sickle-cell and normal red blood cells

SCD phase I/II death shines Beam on ‘very real’ busulfan risk

Nov. 5, 2024
By Randy Osborne
Alongside the release of abstracts related to the American Society of Hematology meeting next month in San Diego, and as part of the firm’s third-quarter update, Beam Therapeutics Inc. disclosed that one patient died in the phase I/II trial testing BEAM-101 in sickle cell disease (SCD).
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Black umbrella open against blue sky with clouds

Disc over the moon as FDA urges Apollo bid with bitopertin in EPP

Nov. 4, 2024
By Randy Osborne
Disc Medicine Inc. found itself after an end-of-phase II meeting with the U.S. FDA in what Wainwright analyst Douglas Tsao called a “best-case scenario” regarding the path forward for bitopertin in erythropoietic protoporphyria (EPP).
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US CRS weighs in on improving gene therapy regulatory landscape

Oct. 28, 2024
By Mari Serebrov
Although more and more gene therapies are getting the FDA stamp of approval, concerns persist about their potential long-term risks. U.S. lawmakers have proposed several pieces of legislation over the past few years to address some of the uncertainties. Now the Congressional Research Service (CRS) is suggesting other requirements Congress may want to consider to improve the regulatory landscape for gene therapies, especially those intended to treat blood disorders.
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Stem cells
Hematologic

Editas Medicine achieves in vivo preclinical proof of concept of HSPC editing

Oct. 23, 2024
Editas Medicine Inc. has achieved in vivo preclinical proof of concept of hematopoietic stem and progenitor cell (HSPC) editing and fetal hemoglobin (HbF) induction in humanized mice engrafted with human hematopoietic stem cells and lacking their own hematopoietic cells.
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