Vanda Pharmaceuticals Inc. presented data this week at the annual American Academy of Neurology conference regarding allele-specific antisense oligonucleotides (ASOs) that specifically target the mutant p.A53T allele from the SNCA gene while preserving the expression of the wild-type allele. The mutant allele is associated with increased risk of early-onset Parkinson’s disease (PD) and current ASOs target SNCA regardless of its mutation status.

Mabwell (Shanghai) Bioscience Co. Ltd. has announced that SST-001 (18F-FD4), an α-synuclein-targeted PET tracer developed by Mabwell’s incubated company Synusight Biotech, has received clinical trial clearance from China’s National Medical Products Administration (NMPA). The planned phase I trial will enroll healthy volunteers, patients with multiple system atrophy (MSA), and patients with Parkinson’s disease.

Kinase inhibitors have shown success in disease areas such as oncology, but their application in neurodegenerative diseases is still limited. This is mainly due to several challenges, such as the complexity of kinase networks, limited blood-brain barrier permeability and the lack of biomarkers.

Parkinson’s disease is a progressive neurodegenerative disorder best known for its motor symptoms. However, a proportion of patients also develop dementia as the condition advances. Yet the biological divide between those who experience this cognitive decline and those who do not has remained an open question. Are they different conditions or simply stages of the same disease?

Oryon Cell Therapies, named after the Orion constellation used for navigation at night, emerged from stealth mode to announce a new round of funding and to present data from the phase Ib/IIa study of its neuron replacement therapy in patients with Parkinson’s disease at the 20th International Conference on Alzheimer’s and Parkinson’s Diseases.

A new Danish research project focused on Parkinson’s disease has received funding from Innovation Fund Denmark. The DESYNA (Degradation of Extracellular α-SYNuclein Aggregates) project aims to develop a new therapy targeting α-synuclein, the accumulation of which is a key driver of Parkinson’s disease.

Parkinson’s disease (PD) involves the progressive loss of dopaminergic neurons, particularly in the substantia nigra. This neurodegeneration is linked to the abnormal accumulation of α-synuclein, a protein that forms toxic aggregates and spreads between cells, damaging them. At the 20th International Conference on Alzheimer’s and Parkinson’s Diseases (AD/PD), held from March 17 to 21, 2026, in Copenhagen, several strategies were presented that aim to modify the course of the disease and offer real alternatives to purely symptomatic treatments.

Congruence Therapeutics Inc. has closed a $39.5 million financing to advance into its portfolio of small-molecule correctors for diseases of protein misfolding into the clinic.

Glucocerebrosidase (GCase), encoded by the gene GBA1, is a ubiquitous lysosomal enzyme that breaks down lipid substrates, glucosylceramide (GL-1) and glucosylsphingosine (Lyso-GL1), into glucose and ceramide. Loss-of-function mutations in GBA1 reduce GCase activity, resulting in lipid accumulation within lysosomes and subsequent lysosomal dysfunction.

Casma Therapeutics Inc. has been awarded approximately $7.6 million in funding across two competitive grant programs from The Michael J. Fox Foundation for Parkinson’s Research (MJFF) to support Casma’s lead program, CSM-101.