The current treatment strategies for neurodegenerative diseases focus on targeting Aβ in Alzheimer’s disease (AD), α-synuclein aggregates in Parkinson’s disease (PD) and anti-tau therapies, which are primarily used in AD but are also being explored for PD. At the 2025 International Conference of Alzheimer’s & Parkinson’s Disease and Related Neurological Disorders, Aditya Iyer, senior RD scientist from Amyl Therapeutics Srl, presented data on an option which could potentially serve as a pan-amyloid therapeutic.
Nissan Chemical Corporation and Sanwa Kagaku Kenkyusho Co. Ltd. (SKK) have entered an agreement to codevelop SK-2407/SN-001 for the treatment of dentatorubral-pallidoluysian atrophy (DRPLA) in Japan.
Parkinson’s disease (PD) is a neurodegenerative disease of the central nervous system characterized by the loss of dopaminergic neurons in the substantia nigra and the abnormal aggregation of α-synuclein. UCB Pharma Inc. has presented new preclinical data on their anti-α-synuclein antibody UCB-7853 for the potential treatment of PD.
Insilico Medicine Ltd. has identified postsynaptic density-95 (PSD-95) protein inhibitors acting as neuroprotective agents reported to be useful for the treatment of epilepsy, anxiety disorders, neurodegeneration, neuropathic pain, stroke and cerebral infarction.
Neurodegenerative pathologies, once primarily associated with protein alterations, should be revisited in the context of lipidopathies, researchers argued at the 2025 International Conference of Alzheimer’s & Parkinson’s Disease and Related Neurological Disorders (AD/PD 2025).
The survival and plasticity of neurons depends on the signaling of the nerve growth factors BDNF and NGF acting through TRK receptors, which is crucial in neurological disorders such as Alzheimer’s disease (AD). Alzecure Pharma’s ACD-856 is a positive allosteric modulator (PAM) of TRK receptors that is in phase I trials for AD, and has shown good safety, pharmacokinetics and target engagement in the central nervous system.
Innovstone Therapeutics Ltd. has synthesized leucine-rich repeat kinase 2 (LRRK2; Dardarin) inhibitors reported to be useful for the treatment of Parkinson’s disease.
Sangamo Therapeutics Inc. has entered into a license agreement with Eli Lilly and Co., allowing Lilly to use Sangamo’s novel proprietary neurotropic AAV capsid, STAC-BBB, to deliver intravenously administered genomic medicines to treat certain diseases of the central nervous system.
Spark Therapeutics Inc. has presented a proprietary adeno-associated viral (AAV) vector expressing an artificial miRNA targeting human α-synuclein (α-Syn) mRNA. Accumulation of misfolded and insoluble α-Syn causes neuronal toxicity in preclinical models and has been identified as the underlying cause of synucleinopathies.
Dual-specificity tyrosine phosphorylation-regulated kinase 1A (DYRK1A) is an attractive therapeutic target due to its involvement in cancer and neurodegenerative diseases. Researchers from the National Health Research Institutes and their collaborators have presented a series of DYRK1A inhibitors for reducing neurofibrillary tangle formation in Alzheimer’s disease.