Epilepsygtx Ltd. has raised a $33 million series A to fund a phase I/IIa trial of EPY-201, a gene therapy for treating drug-resistant focal epilepsy. EPY-201 uses an adeno-associated viral vector to deliver KCNA1, the gene encoding Kv1.1, a potassium ion channel that modulates neuronal excitability.
Researchers from Neumirna Therapeutics ApS have presented an anti-miR-134 ASO approach named NMT.001 for the potential treatment of drug-resistant epilepsy.
Mutations in the KCNT1 gene produce gain-of-function effects that lead to overactivation of the potassium channel and consequent disruption of normal neuronal electrical signaling. These alterations give rise to a severe, early-onset developmental and epileptic encephalopathy that is typically associated with a high seizure burden and resistance to standard antiseizure medications.
In an alternative to small molecules, researchers at the University of Ferrara and Università degli Studi di Padova sought to create an NOP agonist based on peptides, specifically dimers of peptides derived from native nociceptin.
The ALS Association has announced the recipients of its 2025 Hoffman ALS Clinical Trial Awards. These research grants, worth up to $1 million each, support early-phase trials that generate critical data to accelerate the development of new amyotrophic lateral sclerosis (ALS) treatments.
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.
Genescience Pharmaceuticals Co. Ltd. has described salts of known melanocortin MC4 receptor antagonists reported to be useful for the treatment of anorexia nervosa, cachexia, muscle atrophy, nausea and vomiting, sarcopenia, anxiety, bone disorders and inflammatory disorders.
LRRK2 plays a key role in the biology of multiple neurodegenerative disorders, including Parkinson’s disease (PD) and progressive supranuclear palsy (PSP), where mutations or altered activity are associated with impaired cellular signaling and neuronal decline.
Arrakis Therapeutics Inc. has announced the presentation of preclinical data demonstrating the progress of its RNA-targeted small molecule (rSM) drug program for the treatment of myotonic dystrophy type 1 (DM1).
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