New research shows base and prime editing can correct some forms of phenylketonuria (PKU) in mice and human cell lines, raising the prospect that this gene-editing approach could allow children born with the inherited metabolic disorder to have a treatment that would avoid the need for dietary restrictions and medication.
Two new polio vaccine candidates designed to prevent the emergence of vaccine-derived virulent polioviruses have been shown to induce immune responses in mice, raising the possibility of eradicating the virus. For that to happen, the transmission of all poliovirus serotypes must be blocked. However, the vaccine used to control polio prevents disease but does not stop transmission, enabling the virus to mutate and regain virulence.
MD Biopharm Co. Ltd. has described protein-glutamine γ-glutamyltransferase 2 (TGM2) inhibitors reported to be useful for the treatment of cancer, celiac disease, neurological, inflammatory and cardiovascular disorders.
Edgewise Therapeutics Inc. has divulged pyridazinone compounds acting as myosin-2 (MYH2) (ATPase activity) inhibitors reported to be useful for the treatment of Duchenne muscular dystrophy, amyotrophic lateral sclerosis, encephalitis, spinal cord injury, phenylketonuria, cerebral palsy, Alzheimer’s disease and Parkinson’s disease, among others.
Wigen Biomedicine Technology (Shanghai) Co. Ltd. has identified kinesin-like protein KIF18A inhibitors reported to be useful for the treatment of cancer.
Researchers from F. Hoffmann-La Roche Ltd. and Hoffmann-La Roche Inc. have synthesized NLRP3 inflammasome inhibitors reported to be useful for the treatment of asthma, chronic obstructive pulmonary disease, Parkinson’s disease and Alzheimer’s disease.
Colorectal cancer (CRC) is one of the digestive tract cancers with the highest prevalence. Cancer immunotherapy for CRC brings substantial benefit to patients, but the development of resistance mechanisms and relapsing processes along with immune escape call for new treatment options.
La Crosse virus (LACV) is one of the most common causes of arboviral encephalitis in children, which causes CNS damage owing to infection in children, but not adults. LACV pathology indicates significant breakdown of the blood-brain barrier, which is strengthened during developmental transition from child to adult.
Researchers from Imperial College of Science, Technology and Medicine have reported results of preclinical evaluation of THEO-260, a novel oncolytic virus for the treatment of stromal-rich tumors. The candidate is currently in development for late-stage relapsed refractory ovarian cancer.
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