Epigenetic desilence of the paternal allele of the gene that causes Angelman syndrome (AS) could be used to treat this disease for which there are currently no approved therapies.

The big advantage of cell culture to model diseases is its throughput. “You can play the disease over and over again in the dish,” Clive Svendsen told the audience at the International Society of Stem Cell Research (ISSCR) Annual Meeting held in Hamburg last week. That high throughput, however, is not particularly useful if the cell lines themselves do not accurately model the disease. Cancer cell lines are used in many cell culture experiments far beyond cancer for their ability to grow. But they are “highly abnormal,” Bill Skarnes told the audience at an innovation showcase, as well as quite unstable. “I don’t think the [HEK-293] cell line is the same in your lab as it is in the lab next door,” Skarnes said.

The first patenting from South Korea’s Neudive Inc. sees its CEO, SungJa Cho, applying for protection of the company’s mobile digital social therapy device, NDTx-01, which helps build the social skills of neurodiverse individuals, and in particular children and adolescents with autism spectrum disorder and social communication disorder.

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.

Researchers from the Chinese Academy of Sciences evaluated the highly potent and selective inhibitor of the second bromodomain (BD2) of the bromodomain and extra-terminal (BET) family of proteins, ABBV-744, with the aim of assessing its preclinical efficacy and exploring the pathways by which the compound regulates microglia-mediated neuroinflammation.

One of the most common causes of adverse neurological disabilities in newborns is neonatal hypoxic-ischemic encephalopathy. Therapeutic hypothermia (TH) is the standard therapy, but it is not efficient in all cases. Cannabinoids have raised interest as therapeutics as they are neuroprotective.

Satellos Bioscience Inc. has submitted a clinical research proposal to a Human Research Ethics Committee (HREC) in Australia seeking regulatory authorization under the Therapeutic Goods Administration (TGA)’s clinical trial notification scheme to conduct a first-in-human phase I trial of SAT-3247.