Juvenile neuronal ceroid lipofuscinosis (JNCL) is an inherited neurodegenerative disease caused by mutations in the gene encoding CLN3 lysosomal protein. Contrary to late-infantile neuronal ceroid lipofuscinosis (LINCL), caused by mutations in the lysosomal protein tripeptidyl peptidase 1 (TPP1), no animal models or effective treatment exist for JNCL. Previous research characterized mouse models with either mutated Tpp1 or Cln3, but the phenotype of a double Cln3/Tpp1 mutant, as well as the function of CLN3 protein, remain unclear.

Lung adenocarcinoma (LUAD) represents 35% to 40% of all lung cancers, making it the most common non-small-cell lung cancer in the U.S. Despite continual treatment advances, lung cancer remains the leading cause of cancer-related mortality worldwide. Accordingly, there is an ongoing urgent need to identify targets and associated therapeutics.

Researchers from Nordic Bioscience A/S and Keybioscience AG have published data from a study that aimed to assess the impact of treatment sequencing or combination therapy with the novel long-acting dual amylin and calcitonin receptor agonist (DACRA) KBP-336 and the glucagon-like peptide-1 (GLP-1) analogue semaglutide for the treatment of obesity and associated co-morbidities.

Researchers from Kunming University of Science and Technology and affiliated organizations presented discovery and preclinical evaluation of a novel salinomycin derivative, SAL-98, as a potential therapeutic candidate for the treatment of cancer.

Novaccess Global Inc. has signed a letter of intent and formed a partnership with BCN Biosciences LLC to expand its immunotherapy platform. Utilizing BCN’s small-molecule drug BCN-057, Novaccess Global has begun the development of a fourth generation of its novel brain tumor immunotherapy TLR-AD-1, targeting glioblastoma.

Researchers from Hepagene Therapeutics Inc. have disclosed preclinical data for the novel FXR agonist HPG-1860, which is being developed for the treatment of nonalcoholic steatohepatitis (NASH).

Researchers at the Salk Institute for Biological Studies have shown a possible beneficial role for muscle wasting in mice infected with Trypanosoma brucei, a parasite that causes sleeping sickness in humans. The team assessed the impact of both fat and muscle wasting on survival of mice with T. brucei infection. They found that while fat wasting appeared to have no impact on survival, mice that were unable to undergo muscle wasting died more quickly from the infection than those that experienced muscle loss.