In a recent publication in Molecular Therapy, researchers from Drexel University College of Medicine and UMass Chan Medical School presented a silence-and-replace gene therapy strategy aiming to address both the gain-of-toxicity and loss-of-function components of the disease hereditary spastic paraplegia (HSP).

The receptor tyrosine kinase-like orphan 1 (ROR1) is a transmembrane protein expressed during embryonic development. Moreover, it is highly expressed on the surface of several cancer cell types, including B-cell malignancies and solid tumors such as triple-negative breast cancer (TNBC). Researchers from The University of Queensland and collaborating institutions recently reported the development of a single-chain fragment variable (scFvs) specifically targeting the membrane proximal region of ROR1 rather than the Ig-like domain, aiming to reduce cross-reactivity with other surface proteins expressed in normal tissues.

SYNGAP1-related disorders (SRDs) are rare neurodevelopmental conditions characterized by a wide range of symptoms, including intellectual disability, epilepsy, motor deficits and increased risk-taking behavior.

Shanghai Henlius Biotech Inc. has announced acceptance of IND applications by China’s National Medical Products Administration (NMPA) for three differentiated candidates for the treatment of solid tumors.

A review of 2025's noteworthy advances in medical research, including GLP-1 receptor agonists as anti-aging drugs, tumor-agnostic therapies and xenotransplants.