The serotonin 5-HT2B receptor is widely expressed in the brain, including on serotoninergic, GABAergic and dopaminergic neurons, on astrocytes and microglia, and neuronal fibers of the frontal cortex and cerebellum.
The FDA has granted orphan drug designation to Modalis Therapeutics Corp.’s MDL-101, a novel epigenetic editing therapy being developed for the treatment of congenital muscular dystrophy type 1a (LAMA2-CMD), a severe, early-onset muscular dystrophy caused by the absence of the LAMA2 protein.
For a company that was running out of money, a missed phase III endpoint for its only development product knelled a death blow for Marinus Pharmaceuticals Inc., tanking its stock by 82%. The Radnor, Pa.-based company will no longer develop oral ganaxolone for seizures associated with tuberous sclerosis complex, or for any other indication, as it reduces its workforce and explores strategic alternatives.
Privately held Dyno Therapeutics Inc. has added another notch to its adeno-associated virus (AAV) vectors development portfolio in a deal with the Roche Group that includes $50 million up front and ultimately could top $1 billion. Dyno will help in developing next-generation AAV vectors, optimized by artificial intelligence, to target neurological diseases.
The first patenting from Neuro-Joy Ltd. details development of a device that electrically stimulates facial nerves and muscles in order to emulate a facial expression configured to ameliorate a mood disorder and improve symptoms of depression, anxiety and sleep disorders.
Aliad Biopharma Co. Ltd. has described compounds acting as autophagy inducers and mTOR complex 2 (mTORC2) inhibitors reported to be useful for the treatment of Alzheimer’s disease, among others.
Scientists from different laboratories around the world have presented the latest advances in research into malignant brain tumors at the 31st Annual Congress of the European Society of Gene and Cell Therapy (ESGCT), which is being held Oct. 22 to 25 in Rome.
Despite positive findings from an earlier trial, Alto Neuroscience Inc.’s BDNF-targeting candidate, ALTO-100, failed to best placebo in a phase IIb study in major depressive disorder, sending shares of the company to their lowest price since going public in a February 2024 IPO, as investors worried about readthrough to Alto’s biomarker-based approach for treating psychiatric disorders.
The U.K. Medicines and Healthcare products Agency has become the third to approve Eli Lilly and Co.’s Kisunla (donanemab), but the drug’s spending watchdog has simultaneously ruled the Alzheimer’s disease treatment is not cost effective.
Humanwell Healthcare (Group) Co. Ltd. has patented compounds acting a type-2 angiotensin II receptor (AGTR2; AT2) antagonists reported to be useful for the treatment of neuropathy and neuropathic pain.
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