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.
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.
Shaanxi Micot Technology Co. Ltd. has described theophylline derivatives acting as anticoagulant reversal agents reported to be useful for the treatment of hemorrhagic disorders.
Metagenomi Inc. has reported data from an ongoing preclinical study designed to provide evidence supporting the potential durability and safety of the company’s hemophilia A gene editing investigational therapy, MGX-001.
Shwachman-Diamond syndrome is an inherited bone marrow failure syndrome characterized by exocrine pancreatic insufficiency, cytopenia, growth restriction and skeletal abnormalities, and for which primary treatment is allogeneic hematopoietic stem cell transplantation, which is associated with significant toxicity.
Recent studies have shown that up-regulation of peroxisome proliferator-activated receptor γ coactivator-1α (PGC-1α) was able to induce fetal hemoglobin synthesis in human primary erythroblasts.
Oxford University Innovations Ltd. has divulged Egl nine homolog 1 (EGLN1; HPH-2; PHD2) inhibitors reported to be useful for the treatment of anemia, ischemia, cardiovascular disorders, inflammatory disorders, irritable bowel syndrome, chronic kidney disease, Parkinson’s disease and Alzheimer’s disease, among others.
RNA Therapeutics Inc. has received a written response from the FDA to its pre-IND application questions regarding the development of its lead asset, RNAT-89 (BLA-761423), an mRNA LNP formulation to express darbepoetin.
A recent study in PLoS One by researchers from Cincinnati Children’s Hospital Medical Center has evaluated the preclinical long-term safety of human A gamma-globin gene-carrying GbGM LV in wild-type mice.
Scientists at the Novartis Institutes for Biomedical Research (NIBR) in Cambridge have discovered a small molecule that could be used as a therapy for sickle cell disease (SCD). The molecular glue oral degraders of the WIZ transcription factor called dWIZ-1 and dWIZ-2, bind to cereblon (CRBN) and WIZ, marking it for degradation and inducing the expression of fetal hemoglobin (HbF).
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