Centogene NV and Evotec SE have announced the discovery of a new small molecule with potential to treat patients with type 2 and type 3 Gaucher disease, or neuronopathic Gaucher disease.
Rhythm Pharmaceuticals Inc. has patented new Kir6.2/SUR1 activators reported to be useful for the treatment of disorders of sexual function, hyperinsulinemia, cancer, genitourinary, neurological and cardiovascular disorders.
From glaucoma to Stargardt disease, age-related macular degeneration (AMD) to retinitis pigmentosa, or a corneal transplant to Bietti’s crystalline dystrophy, the 27th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) is working to bring some light to patients with age and congenital diseases that affect vision. From May 7-11, 2024, thousands of scientists are gathering in Baltimore to show their advances against the challenges of delivering genes and cells to the correct place, avoiding immunogenicity and improving diseases.
Maze Therapeutics Inc. has described sodium-dependent neutral amino acid transporter B(0)AT1 (SLC6A19) inhibitors reported to be useful for the treatment of phenylketonuria, metabolic syndrome, chronic kidney disease and metabolic diseases.
Researchers at Biogen Inc. and Broad Institute Inc. have divulged glycogen synthase kinase-3α (GSK3A; GSK-3α) inhibitors reported to be useful for the treatment of cancer, neurological and metabolic diseases.
“Prenatal therapies are the next disruptive technologies in health care, which will advance and shape the future of patient care in the 21st century,” said Graça Almeida-Porada, a professor at the Fetal Research and Therapy Center of the Wake Forest Institute for Regenerative Medicine in Winston-Salem, North Carolina. At the American Society of Gene & Cell Therapy (ASGCT) annual meeting in Baltimore on May 5, 2024, Almeida-Porada introduced the first presentation of the scientific symposium “Prospects for Prenatal Gene and Cell Therapy.”
Researchers from Huazhong University of Science and Technology and collaborators published results from a study that aimed to assess the potential role of caveolin 3 (CAV3) in mitochondrial function during diabetic cardiomyopathy (DCM).
Shenzhen Zhongge Biotechnology Co. Ltd. has synthesized tyrosine-protein phosphatase non-receptor type 1 (PTPN1; PTP-1B) and/or PTPN2 (TCPTP) inhibitors reported to be useful for the treatment of cancer, obesity, atherosclerosis, type 1 diabetes, type 2 diabetes, neurodegeneration, metabolic syndrome and inflammatory bowel disease, among others.
RSS
