Sickle cell disease (SCD) is autosomal recessive disorder caused by mutations in the β-globin gene, and induction of fetal γ-globin is considered an established therapeutic strategy for the treatment of this disease. A research team led by scientists at Kyorin Pharmaceutical Co. Ltd. has discovered RK-701, a small-molecule inhibitor of G9a and G9a-like protein (GLP) as a potential therapeutic agent for SCD.
Coming off a tough year for raising money, Cadrenal Therapeutics Inc. is leading the charge into a new year by completing 2023’s first U.S. IPO. The company closed its IPO of 1.4 million shares at $5 each for $7 million. The stock (NASDAQ:CVKD) began trading Jan. 20 and closed 9.37% downward on Jan. 25 at $2.90 per share.
A genome-wide association study (GWAS) from The University of Queensland has linked blood cell traits (BCTs) and neurological and psychiatric disorders (NPDs), providing a tool to improve patient treatments or repurposing different drugs. The researchers also found a cause-effect relationship between Parkinson's and platelet distribution width. In their study, published Jan. 25, 2023, in Cell Genomics, the scientists observed the genetic overlap between common NPDs and 29 BCTs, including functional genes, regulatory elements and new genetic correlations linked to hematological data and for these diseases.
Just in time for the Chinese New Year, Mabwell Bioscience Co. Ltd. announced that its U.S. subsidiary, Mabwell Therapeutics Ltd., closed a licensing deal with Disc Medicine Inc. worth up to $412 million for preclinical candidate 9MW-3011, a humanized monoclonal antibody (MAb) that regulates iron metabolism.
Mabwell Therapeutics Inc., a wholly owned subsidiary of Mabwell (Shanghai) Bioscience Co. Ltd., has entered into a license agreement with Disc Medicine Inc. for 9MW3011 (MWTX-001, MWTX-002 & MWTX-003) for hematologic diseases.
By combining drug sensitivity with genomic profiling of tumor cells, a study from St. Jude Children's Research Hospital with more than 800 patients has shown a wide diversity in drug sensitivity for pediatric acute lymphoblastic leukemia (ALL) and defined six patterns of response to treatment. “This work provides a framework for ‘functional precision medicine’,” corresponding author Jun Yang, vice chair of the Department of Pharmacy and Pharmaceutical Sciences at St. Jude Children's Research Hospital, told BioWorld.
Scientists based at Beijing Institute of Radiation Medicine have reported preclinical evaluation of a novel recombinant activated human factor VII (rFVIIa), GEN-0828, being developed as potential candidate for the treatment of hemophilia and trauma hemorrhage. GEN-0828 is rFVIIa that was mainly produced from Chinese hamster ovary (CHO) cells.
Researchers from Beam Therapeutics Inc. presented the discovery and preclinical evaluation of an engineered stem cell antibody paired evasion (ESCAPE) strategy for antibody (Ab)-mediated autologous hematopoietic stem cell (HSC) therapy conditioning for the treatment of hemoglobinopathies.
The serious adverse event in the first phase I/II patient dosed with nulabeglogene autogedtemcel (nula-cel) – a gene editing, autologous hematopoietic stem cell therapy for sickle cell disease (SCD) – may have involved the quality of the stem cells and not the conditioning regimen or characteristics of the patient at baseline. But researchers won’t know until more work is done.
Jasper Therapeutics Inc. may have found a way around toxicity with current approaches in sickle cell disease (SCD) conditioning approaches, if phase I/II data with briquilimab stay consistent – and the drug already has proved itself across a range of indications. Wall Street liked the prospect, sending the Redwood City, Calif.-based firm’s shares (NASDAQ:JSPR) on a wild ride to close at $2.74, up $2.26, or 476%.
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