Patients with congenital hearing loss could benefit from a gene therapy currently in development. Although there are approaches that could reverse the process in children and young people before it becomes severe, so far, adults do not have any treatment that prevents the progressive deterioration of auditory sensory cells caused by this disease. Read More

Researchers from the Center for Applied Medical Research (CIMA) in Spain have published preclinical data for two new histone deacetylase (HDAC) inhibitors – CM-444 and CM-1758 – being developed for the treatment of acute myeloid leukemia (AML). Read More

N6-Methyladenosine (m6A) is the most widely distributed RNA modification in eukaryotes and its dysregulation has been tied in gastric cancer protumorigenic processes, as well as drug resistance and metastasis. Vir-like m6A methyltransferase associated, also known as KIAA1429, is a crucial regulator of the m6A complex, and thus hypothesized to be involved in the progression of gastric cancer. Read More

Researchers from Igyxos SA presented the preclinical profile of IGX-12, a humanized IgG4 antibody, in both female and male infertility models. Read More

Zhejiang University of Technology has reported preclinical safety data for the novel multitargeting compound XYY-CP1106, being developed for the treatment of Alzheimer’s disease (AD). XYY-CP1106 is a multitargeted anti-AD compound that has previously demonstrated strong monoamine oxidase B (MAO-B) inhibition (IC50=14.7 nM), high Fe3+ chelation (pFe3+=18.04), no in vitro cytotoxicity and protection against cellular oxidative damage. Read More

Ipsen SA and Foreseen Technology (Beijing) Co. Ltd. have entered into an exclusive global licensing agreement for FS-001, an antibody-drug conjugate (ADC) that targets a novel tumor-associated antigen highly expressed across a range of solid tumors that was identified through the application of Foreseen’s proprietary proteomic platforms. Read More

From no hope to viable treatments, BioWorld  is there to cover the breakthroughs in medicine. Listen to Randy Osborne explain why the BioWorld  team is ‘always on the lookout for what's next.’ Read More

Rgenta Therapeutics Inc. has received IND clearance by the FDA for RGT-61159, which is being developed for adenoid cystic carcinoma, colorectal cancer and other solid tumors, as well as acute myeloid leukemia. Read More

Boehringer Ingelheim Pharma GmbH & Co KG has synthesized D-3-phosphoglycerate dehydrogenase (3-PGDH; PHGDH) inhibitors reported to be useful for the treatment of cancer, autoimmune disease, infections and inflammation. Read More

Haisco Pharmaceutical Group Co. Ltd. has identified phosphatidylinositol 3-kinase α (PI3Kα) (H1047R mutant) inhibitors reported to be useful for the treatment of breast cancer. Read More

The use of artificial intelligence (AI) in drug discovery has shown promise in recent years with a growing number of new compounds moving forward in the pipeline. Read More

Sironax Ltd. has discovered (+) hydrolase SARM1 (SAMD2; MyD88-5) inhibitors reported to be useful for the treatment of amyotrophic lateral sclerosis, diabetic neuropathy, multiple sclerosis, Parkinson's disease, chemotherapy-induced peripheral neuropathy and traumatic brain injury. Read More

Hangzhou Zhongmei Huadong Pharmaceutical Co. Ltd. has described proteolysis targeting chimera (PROTACs) compounds comprising an E3 ubiquitin ligase-binding moiety covalently linked to a mitogen-activated protein kinase kinase kinase kinase 1 (MAP4K1; HPK1; MEKKK1)-targeting moiety. They are reported to be useful for the treatment of cancer. Read More

Ependymoma is a brain cancer that occurs during childhood and is usually resistant to cytotoxic therapy. Molecular profiling has helped in better understanding the mechanisms behind the disease and has unveiled EPH receptor B2 (EPHB2) as a critical player in driving disease. Read More

Gasherbrum Bio Inc. has disclosed GLP-1 receptor agonists reported to be useful for the treatment of type 2 diabetes, dyslipidemia, hyperglycemia, hypertension, obesity, stroke, myocardial infarction and gout, among others. Read More

Additional early-stage research and drug discovery news in brief, from: Lisata Therapeutics, Ovid Therapeutics, Recce Pharmaceuticals, Virpax Pharmaceuticals. Read More