Duchenne muscular dystrophy (DMD) is a devastating genetic disorder affecting approximately 1 in 3,500-5,000 newborn males worldwide. It is characterized by progressive degeneration of skeletal, cardiac and smooth muscle, leading to early mortality. A team of researchers from the Technion Israel Institute of Technology has developed a novel approach to combat fibrosis in DMD.

Grove Biopharma Inc., a spinout from Northwestern University targeting intracellular protein-protein interactions with a novel synthetic biology-based approach, has closed a $30 million series A financing. Proceeds will be used to further advance Grove Biopharma’s Bionic Biologics platform and drive its lead oncology programs toward the clinic.

Harvard University has filed a lawsuit claiming the Trump administration’s freezing of its federal funding is unlawful and beyond the government’s authority. Announcing the move, Harvard’s president, Alan Garber, highlighted the impact of freezing $2.2 billion in grants – and the threat to freeze a further $1.1 billion – will have on the university’s biomedical research.

α7 nicotinic acetylcholine receptors (α7nAChRs), expressed in neurons and astrocytes at lower levels, have been implicated in neuroinflammatory and neurodegenerative processes associated with Alzheimer’s disease (AD). Evidence suggests that α7nAChRs interact with soluble amyloid-β (Aβ), contributing to reactive astrogliosis and formation of Aβ plaques in AD.

Genes associated with lysosomal dysfunction increase the risk of Parkinson’s disease (PD), according to a study led by scientists at Northwestern University. The discovery also explains why some people who carry a pathogenic variant of the GBA1 gene develop PD or dementia with Lewy bodies (DLB) and others do not. The key lies in the Commander complex, involved in the transport of proteins to this organelle. This discovery raises the need for combinatorial therapies that act on more than one pathway for this type of neurodegenerative disorder.

Researchers from Aarhus University and Synuca Therapeutics recently presented a novel drug-like small-molecule inhibitor of sarco/endoplasmic reticulum calcium ATPase (SERCA), SYN-4569, with superior pharmacological properties and evaluated its efficacy in vitro and in vivo. SYN-4569 is an orally available compound with good potency, pharmacokinetics and brain-penetrant properties.