Lys Therapeutics SAS is advancing LYS-241 toward the clinic with the support of more than €25 million (US$33 million) in funding raised since the company’s inception in 2021. The financing includes a grant of more than $5 million from The Michael J. Fox Foundation.
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.
Oligomeric forms of α-synuclein are increasingly recognized as the primary neurotoxic species in Parkinson’s disease (PD) and other synucleinopathies, contributing to synaptic dysfunction, mitochondrial impairment and the prion-like propagation of pathology. Targeting these early aggregates represents a promising strategy for disease modification.
Arrowhead Pharmaceuticals Inc. has announced a global licensing and collaboration agreement with Novartis AG for ARO-SNCA, Arrowhead’s preclinical stage siRNA therapy for the treatment of synucleinopathies such as Parkinson’s disease, and for additional targets.
Being able to detect and monitor the aggregation of α-synuclein in situ could lead to more objective, earlier diagnosis of Parkinson’s disease as well as allow real-time monitoring of whether patients are responding to treatment.
Abnormal aggregation of α-synuclein is thought to contribute to the pathology of Parkinson’s disease, the second most frequent neurodegenerative disorder. Therefore, developing drugs that prevent such aggregation could be effective for slowing or even preventing the disease.
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.
At the 2025 International Conference on Alzheimer’s and Parkinson’s Diseases and Related Neurological Disorders (ADPD), Promis Neurosciences Inc. presented its new approach to optimizing the α-synuclein (α-Syn) vaccine composition in order to maximize targeting of toxic α-Syn species.
Researchers from Tridem Bioscience GmbH & Co. KG provided details on the development of TRB-001, an anti-α-synuclein construct consisting of a B-cell epitope derived from human α-synuclein (SNCA), conjugated to CRM197 and C-type lectin (CLEC) β-glucan.
Researchers from Sanofi SA reported the preclinical characterization of SAR-446159 (ABL-301), a bispecific antibody construct comprising an antibody targeting α-synuclein fused to an engineered antibody fragment that targets IGF-1R and functions as a blood-brain barrier (BBB) shuttle, known as the Grabody-B platform.