Scientists at Ghent University have created a mouse model that incorporates human versions of the receptors that recognize the fragment crystallizable (Fc) region of immunoglobulin G (IgG), one of the most abundant antibodies in the blood and a key mediator of essential immune functions such as cellular activation, pathogen elimination and the regulation of inflammatory responses. These human Fcγ receptors allow the humanized mouse to more accurately reproduce IgG-driven biology, enabling more reliable and safer preclinical assays before evaluating monoclonal antibodies in clinical trials with people. Read More
Krabbe disease, also called globoid-cell leukodystrophy, is caused by a deficiency of the lysosomal enzyme β-galactosylceramidase (GALC), resulting from pathogenic variants in both copies of the GALC gene. The GALC deficiency leads to increased levels of the primary galactosphingolipid substrates, galactosylceramide (GalCer) and psychosine (galactosylsphingosine, PSY), in the nervous system. Read More
Prelude Therapeutics Inc. has received IND clearance from the FDA for PRT-12396, a mutant-selective JAK2 V617F inhibitor being developed for the treatment of patients with certain myeloproliferative neoplasms. Read More
Zipbio has signed an exclusive license agreement with Meiragtx Holdings plc to advance an AAV gene therapy for geographic atrophy. Under the agreement, Meiragtx will receive exclusive rights to Zipbio’s first-in-class therapies targeting the complement pathway for geographic atrophy. Read More
Acute ischemic stroke still represents a public health challenge due to its high incidence and mortality rate. Current treatments may effectively restore the blood flow, but recanalization by opening occluded vessels does not guarantee a favorable prognosis. Read More
IL-10-based approaches have shown promise in cancer immunotherapy by activating exhausted CD8+ T cells, but severe hematological toxicities have limited their clinical use. Recent strategies aim to harness IL-10’s antitumor effects while reducing these toxicities. Read More
Alterity Therapeutics Ltd. has divulged salts of ATH-434, the company's oral agent designed to inhibit the aggregation of pathological proteins implicated in neurodegeneration. Read More
Interleukin-12 (IL-12) is a pro-inflammatory cytokine produced by antigen-presenting cells that plays a central role in shaping cell-mediated immune responses. It promotes the activation and effector function of natural killer cells and cytotoxic CD8+ T lymphocytes and drives the differentiation of CD4+ T cells toward a T helper 1 phenotype. Read More
A patent from Hansoh Bio LLC, Jiangsu Hansoh Pharmaceutical Group Co. Ltd. and Shanghai Hansoh Biomedical Co. Ltd. describes new multi-membered macrocyclic compounds acting as GTPase KRAS G12C, G12V and/or G13D mutant inhibitors. Read More
Haisco Pharmaceutical Group Co. Ltd. has disclosed new muscarinic M4 receptor agonists described as potentially useful for the treatment of substance abuse and dependence, Alzheimer’s disease, pain, schizophrenia and sleep disorders. Read More
Researchers from China hypothesized that Takeda G protein-coupled receptor 5 (TGR5), also known as G-protein coupled bile acid receptor 1, could have therapeutic potential in acute kidney injury by inhibiting ferroptosis. Read More
Anew Therapeutics Pte Ltd. has synthesized heterocyclic compounds acting as interleukin-17A (IL-17A)/interleukin-17 receptor A (IL-17RA) interaction inhibitors. As such, they are reported to be useful for the treatment of Crohn’s disease, type 1 diabetes, dry eye, multiple sclerosis, myositis, psoriasis, rheumatoid arthritis and vitiligo, among others. Read More
Kinoteck Therapeutics Co. Ltd. has discovered nitrogen-containing heterocyclic derivatives acting as Werner syndrome ATP-dependent helicase (WRN; RECQ3; RECQL2) inhibitors potentially useful for the treatment of cancer. Read More
Researchers from Nirma University in India aimed to discover and develop novel small-molecule telomerase inhibitors derived from known scaffolds with improved efficacy and safety for lung cancer therapy. Read More