At the Clinical Trials on Alzheimer’s Disease 2025 meeting, a panel of experts discussed the need for developing combination therapies for the complex diseases that result in Alzheimer's disease and other dementias.
Brainomix Ltd. reported the publication of a prospective real-world study demonstrating that its AI imaging platform, Brainomix 360 Stroke, significantly increased rates of endovascular thrombectomy (EVT) treatment, a minimally invasive surgical procedure for patients with large vessel occlusion stroke, and reduced delays in patient triage and transfer.
Lynk Pharmaceuticals Co. Ltd. has received IND clearance from the FDA for LNK-01006, an allosteric TYK2 inhibitor with potential to treat neurodegenerative diseases.
Vectory Therapeutics BV has obtained IND clearance from the FDA for a phase I/II trial (PIONEER-ALS) of VTX-002, a first-in-class vectorized antibody targeting TDP-43 pathology in amyotrophic lateral sclerosis (ALS). VTX-002 delivers an engineered antibody designed to selectively target toxic species of TDP-43.
Latus Bio Inc. has reported IND clearance by the FDA for LTS-101, a gene therapy candidate to treat the CNS manifestations of late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease. The FDA has also granted orphan drug, rare pediatric disease and fast track designations to LTS-101.
Shenzhen Icarbonx Intelligent Peptide Pharmaceutical Technology Co. Ltd. has disclosed peptides targeting IQ motif and SEC7 domain-containing protein 1 (IQSEC1; BRAG2) reported to be useful for the treatment of stroke, major depression, substance abuse and dependence, and Alzheimer’s disease.
Inhibiting transient receptor potential melastatin 2 (TRPM2) may mitigate injury arising from ischemic stroke. Researchers at Zhejiang University and its medical school previously reported a small-molecule inhibitor of TRPM2, which also inhibited the human ether-a-go-go-related gene (hERG) channel, implying high risk of cardiotoxicity.
Brenig Therapeutics Inc. has described leucine-rich repeat kinase 2 (LRRK2; dardarin) and (LRRK2; dardarin) (G2019S mutant) inhibitors reported to be useful for the treatment of Parkinson’s disease, among others.
The number of deaths caused by prion diseases reaches about 30,000 annually. Only 5 months pass from the diagnosis of seemingly healthy patients to the fatal outcome of this neurodegenerative condition, and just 1 month until quality of life is completely lost. Removing the brain protein that causes this genetic or infectious disorder could be achieved thanks to new gene-silencing techniques. At a special meeting of the American Society of Gene & Cell Therapy, in “AAV-mediated epigenetic editing for prion disease,” Sonia Vallabh presented not just the data of her research, but the impact of this disease on her family and on herself.
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