Embryonal tumor with multilayered rosettes (ETMR) is a rare but aggressive brain cancer that affects very young children, typically under the age of 3 years. Survival rates for ETMR patients remain low, with a median survival of just over a year and only about 20% of patients living beyond 5 years.

Pancreatic ductal adenocarcinoma (PDAC) is among the most lethal cancers and needs innovative therapeutic solutions. Patients with PDAC have a 5-year survival rate of about 12%. To address this need, researchers have developed a Drosophila PDAC model harboring the genetic alterations in the KRAS, CDKN2A, TP53 and SMAD4 genes (4-hit model), which accounts for the worst and most aggressive PDAC with lower survival rates.

Capsida Biotherapeutics Inc. has obtained IND clearance from the FDA for CAP-003, an intravenously administered gene therapy, for Parkinson’s disease associated with GBA mutations (PD-GBA). A phase I/II trial will begin dosing in the third quarter of this year.

Octant Inc. has received a $4.9 million grant from the Gates Foundation to support a new therapeutics program focused on human papillomavirus (HPV)-related cancers. The initiative will develop affordable, broad-spectrum, small-molecule antiviral drugs designed to disrupt the interaction between hundreds of variants of the HPV E6 protein and the tumor suppressor protein p53.

Regulatory T cells (Tregs) maintain immune homeostasis by inhibiting excessive immune responses. Dysregulation of Tregs, characterized by reduced cell numbers or impaired suppressive function, is implicated in the pathogenesis of autoimmune diseases. Low-dose interleukin-2 (IL-2) therapy expands Tregs and enhances their suppressive capacity while minimizing the activation of T cells and natural killer (NK) cells.

4M Therapeutics Inc. has released promising results from completed GLP safety studies for its lead asset, 4MT-2001, supporting its potential to offer a safer treatment option to patients who are underserved by lithium and atypical antipsychotics.