Copper overload within the body may lead to cancer, neurodegenerative diseases, inflammation or fibrosis, among others. Copper chelation is an effective strategy to counteract this potential overload; mitochondria play an important role, as they are the main copper-using organelles in the cells. Copper chelators have been developed, but they have shown some undesired off-target effects and low specificity, which suggests the need for new therapies in the field.

CBL-B is a RING-type E3 ubiquitin ligase that acts as a negative regulator of T-cell activation, contributing to immune homeostasis by limiting excessive immune responses. In cancer, however, this inhibitory role can impair the immune system’s capacity to detect and eradicate tumor cells. Researchers from Genentech Inc. presented the design and optimization of a series of CBL-B-targeting molecular glues.

The N6-methyladenosine (m6A) RNA modification has a role in cancer progression, including renal cell carcinomas (RCCs). Recent work showed that KIAA1429, which serves as a scaffold for the m6A methyltransferase holocomplex, is significantly upregulated in hepatocellular carcinoma tissues and presents oncogenic roles in breast and gastric cancers. However, the potential role of KIAA1429 in clear cell RCC and its underlying regulatory mechanism remain largely unknown.

Ibio Inc. has received clinical trial notification (CTN) acknowledgement from Australia’s Therapeutic Goods Administration (TGA), as well as Human Research Ethics Committee approval, for IBIO-600, enabling the initiation of a first-in-human trial in Australia.

Cue Biopharma Inc. has reached a preclinical milestone in its collaboration and license agreement with Boehringer Ingelheim Pharma GmbH & Co. KG, following the latter’s selection of a first compound for lead optimization. The milestone triggers a $7.5 million payment to Cue.

Proqr Therapeutics NV has highlighted the continued progression and expansion of its early-stage pipeline of RNA editing therapies, including programs in cholestatic diseases, Hurler syndrome, metabolic dysfunction-associated steatohepatitis and Rett syndrome.

Newco Sononeu Inc. has launched to translate more than a decade of basic research in controlling cells with ultrasound into novel, non-invasive therapies for conditions including peripheral and diabetic neuropathy. The underlying technology of sonogenetics provides the ability to manipulate cellular activity via ultrasound-sensitive ion channels that respond to acoustic pressure. These channels may be endogenous, or could be delivered by targeted vectors to specific cell types.