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.
Researchers from UCB SA and affiliated organizations presented the discovery and preclinical characterization of novel NUAK family SnF1-like kinase-1 (NUAK1) inhibitors.
Zhongzhi Pharmaceutical Holdings Ltd. made a $3 million investment in a series A financing round of stem cell therapy developer Gabaeron Inc. Dec. 21, expected to help propel Gabaeron’s preclinical Alzheimer’s disease (AD) candidate into phase I testing.
Lario Therapeutics Ltd. has divulged voltage-dependent R-type calcium channel subunit α-1E (Cav2.3) blockers reported to be useful for the treatment of neurodegeneration, neurodevelopmental disorders, epilepsy, Parkinson’s disease, endocrine disorders, cerebral vasospasm, pain and epileptic encephalopathy.
Nico Therapeutics Inc. has described NLRP3 inflammasome inhibitors reported to be useful for the treatment of Alzheimer’s disease and nonalcoholic fatty liver disease (metabolic dysfunction-associated steatotic liver disease [MASLD]).
Deficiencies in the SLC6A1 gene, encoding the γ-aminobutyric acid transporter GAT-1, are associated with infantile encephalopathy with intellectual disability. Like other neurodevelopmental disorders, SLC6A1-related disorders lack effective treatments; therefore, gene therapy using viral vectors has been proposed as a potential therapeutic strategy.
Spiromacrocyclic compounds acting as orexin OX2 receptor agonists have been described in an H. Lundbeck A/S patent and reported to be useful for the treatment of type 1 and type 2 narcolepsy.
Among the most profound results presented at the 2024 European Society for Medical Oncology (ESMO) Congress were the 10-year data from the Checkmate-067 and Keynote-006 trials of Opdivo and Keytruda as first-line agents in advanced or metastatic melanoma in which 10-year overall survival topped 40%. The success of checkpoint blockade, however, has not extended to all tumor types, but in 2024, molecular studies have led to advances in gene therapies and a multitude of approaches that have opened the door to hope.
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.
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