Neuronos Ltd., a subsidiary of Beyond Air Inc., has announced BA-102 has been awarded U.S. orphan drug designation for the treatment of Phelan-McDermid syndrome.
A large-scale study has revealed the impact of germline variants on proteins in 10 cancer types. Scientists from the National Cancer Institute’s Clinical Proteomic Tumor Analysis Consortium (CPTAC) conducted a precision proteogenomic analysis in a pan-cancer study with data from 1,064 patients, identifying tumor heterogeneity and tumorigenesis associated with heritable genetic alterations. The results provide a broad view of cancer risk that could be useful for patient stratification and the design of prevention strategies.
At the 2025 International Conference on Alzheimer’s and Parkinson’s Diseases and Related Neurological Disorders (ADPD), researchers from Rutgers University investigated the therapeutic potential of CJRB-302, a live biotherapeutic product derived from healthy human gut microbiota, to improve PD motor symptoms and pathology.
At the 2025 International Conference on Alzheimer’s and Parkinson’s Diseases and Related Neurological Disorders (ADPD), researchers from Beijing Joekai Biotechnology LLC presented the discovery process and preclinical results for 50561.
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.
Promis Neurosciences Inc. recently presented the rational design of an Alzheimer’s disease (AD) vaccine candidate maximizing the selective targeting of toxic amyloid-β oligomers.
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).
Dravet syndrome (DS) is a rare and severe form of epilepsy that causes intellectual disability and motor deficits and can lead to premature death. A loss-of-function mutation in one copy of SCN1A, encoding the voltage-gated sodium channel (NaV)1.1 α subunit, is the most frequent alteration found in DS patients and has been linked with inhibitory neuron dysfunction. Despite the potential of gene therapies, AAV-mediated SCN1A gene replacement for DS has not been possible yet due to AAV genome size constraints.