Researchers from Violet Therapeutics Inc. presented the discovery of VTT-001, a novel EPHB3 inhibitor designed to target astrocyte-mediated disease mechanisms.

BHV-8000 from Biohaven Pharmaceuticals Inc. is a dual, brain-penetrant, oral small molecule that is highly selective against TYK2 and JAK1 enzymes, which are essential enzymes to the inflammatory response driving the progression of many neuroinflammatory and neurodegenerative diseases, such as Parkinson’s disease (PD).

Stem cell implantation is a step closer to becoming the next strategy against Parkinson's disease. Two clinical trials, one in phase I and the other in phase I/II, have demonstrated their safety and potential to restore dopamine production in the brains of patients with this currently incurable neurodegenerative condition. The number of participants in the study is still small, and further research is needed to demonstrate the clinical benefits of these cell therapies.

Xellsmart Biopharmaceutical (Suzhou) Co. Ltd. has announced IND clearances by the FDA for its XS-411 and XS-228 cell therapies, for Parkinson’s disease and amyotrophic lateral sclerosis (ALS), respectively.

Biohaven Pharmaceuticals Inc. has presented preclinical data on their novel bispecific degrader BHV-1310 for the potential treatment of autoimmune disorders. To date, about 10% of the global population is affected by autoimmune diseases. IgG-lowering agents have suboptimal pharmacology and pharmacodynamic effects and have safety and tolerability challenges.

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.

Remegen Co. Ltd. emerged as a surprise challenger in the generalized myasthenia gravis space, unveiling positive phase III data of its China-approved lupus drug, telitacicept (RCT-18; Tai’ai), in the rare autoimmune neuromuscular disorder at the 2025 American Academy of Neurology conference.