Investigators from Proqr Therapeutics NV have tested their product QR-1011, an antisense oligonucleotide designed to correct splicing abnormalities within the ABCA4 gene that affect protein expression levels, in Stargardt disease.

Basilea Pharmaceutica Ltd. has entered into an asset purchase agreement with Spexis AG for a preclinical program of antibiotics from a novel class, targeting gram-negative bacteria, including multidrug-resistant strains.

The most comprehensive analysis of gene dependencies in cancer cells to date has identified 370 “highly enriched” drug targets in defined molecular backgrounds. This latest iteration of the Cancer Dependency Map, published in Cancer Cell, Jan. 11, 2024, builds on an earlier version published in 2019, which was based on 324 cell lines.

Researchers have reported that the predictive abilities of a machine learning algorithm trained using best practices on a large clinical dataset did not generalize beyond the data that was used to train it. The algorithm was able to predict, to a degree, which individual patients would benefit from the medication when the patients were from the dataset the algorithm was trained on. But when it was supposed to predict who would benefit in clinical cohorts that were not part of the training, it performed no better than chance.