Researchers from Guangzhou Medical University and affiliated organizations presented data from a study that aimed to investigate the disease-causing mechanism of EP400, a gene that encodes the E1A binding protein p400, which is a core catalytic ATPase subunit of ATP-dependent chromatin remodeling complexes.
A €20 million (US$20.6 million) series A financing round at Neumirna Therapeutics ApS is set to support the company’s development of RNA therapies for epilepsy, Parkinson’s disease and other neurological disorders, and enable the company to advance its lead asset into the clinic.
Gait instability and somnolence are the main hindbrain-related adverse effects associated with the inhibition of AMPA receptors (AMPARs). Transmembrane AMPAR regulatory proteins (TARPs) modulate AMPAR function, with the specific member TARPγ8 being highly expressed in brain regions associated with seizures and expressed at low or negligible levels in the hindbrain.
Biohaven Ltd.’s BHV-7000, a selective Kv7.2/7.3 potassium channel activator, is in phase II/III development for the treatment of focal epilepsy, generalized epilepsy, bipolar disorder and major depressive disorder. The company recently presented findings from preclinical testing assessing the candidate’s potential for the treatment of comorbidities associated with epilepsy.
Researchers form Shanghai Zhimeng Biopharma Inc. presented preclinical data for CB-03, a voltage-gated potassium channel-2/3 (Kv7.2/Kv7.3; KCNQ2/3) opener for the treatment of patients with refractory epilepsy and other neuropsychiatric disorders.
Pathogenic variants in the KCNT1 gene, which encodes potassium channel subfamily T member 1, cause a severe childhood developmental epileptic encephalopathy.
Epileptic encephalopathy due to mutations in the STXBP1 gene, also known as genetic epilepsy, is a rare disease characterized by intellectual disability, speech and motor impairment and behavioral issues, among others, that affects 1 in every 30,000 newborns and which has no approved therapies to date.
Researchers from Bright Minds Biosciences Inc. presented data on the mechanism of action of BMB-101, a novel scaffold 5-HT2C Gq-protein biased designed to treat drug-resistant neurological disorders.
Sea Pharmaceuticals LLC has announced it is advancing two new orally administered neurotherapeutic molecules for neurological disorders. SPM-0404 and SPM-0606 are biologically active as dual AMPA receptor (AMPAR) and kainate receptor (KAINR) antagonists.
The FDA has granted orphan drug designation to Capsida Biotherapeutics Inc.’s CAP-002, an investigational gene therapy for the treatment of developmental and epileptic encephalopathy due to syntaxin-binding protein 1 (STXBP1) mutations.