By pairing the expression of an inhibitory ion channel with an activity-dependent promoter, researchers have developed the first on-demand gene therapy that specifically silenced hyperactive cells and prevented epileptic seizures.

Compass Pathways plc is poised to start the first ever phase III trial of the psychedelic drug psilocybin, after getting U.S. FDA backing for a study in treatment-resistant depression.

Emalex Biosciences Inc. closed an upsized and oversubscribed $250 million series D funding round intended to support a soon-to-start phase III trial and preparations for potential commercialization of ecopipam, its first-in-class drug for Tourette syndrome, a neurological disorder characterized by involuntary motor and vocal tics.

Alkermes plc’s decision to explore separating its commercial-stage neuroscience business from earlier-stage oncology efforts – forming a distinct, publicly traded company to investigate cancer therapies –  drove speculation about the launch of the firm’s Lybalvi (olanzapine and samidorphan), approved by the U.S. FDA in the middle of 2021 for schizophrenia and bipolar I disorder.

Circuit dysfunction is clearly recognized as a driver of neuropsychiatric disease, and some neurodegenerative diseases such as Parkinson’s disease. And at the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) 2022 Congress, researchers made an argument that the same is true in multiple sclerosis (MS). Such a lens could explain the radiological-clinical paradox between the amount of structural damage and clinical severity.

There is little doubt that progress in many brain diseases is being hampered because many, maybe most, diagnostic categories do not reflect underlying brain processes. In other disease areas, modern genetic and genomic methods have arrived in the form of approved drugs, from KRAS inhibitors in cancer to PCSK9 inhibitors to lower cholesterol. But brain diseases are different. Psychiatry is simultaneously the most personal area of medicine, and the least precise.

Astellas Pharma Inc. has invested $50 million in Taysha Gene Therapies Inc. in exchange for 15% of the company and exclusive options to in-license Taysha’s lead gene therapy candidates, TSHA-102 for Rett syndrome and TSHA-120 for giant axonal neuropathy.

Approval of a new depression drug, stellar phase III data for both a schizophrenia therapy and an Alzheimer’s disease candidate, as well as an accelerated approval filing for a Duchenne muscular dystrophy gene therapy, are all reasons that BioWorld’s Neurological Diseases Index climbed from a negative position throughout much of 2022 to its 11% above-water mark as of Oct. 20.

The U.S. FDA wants more data on PTC-518 before PTC Therapeutics Inc.'s phase II study of Huntington’s disease can continue enrollment. While stopped in the U.S., the study of the oral, small-molecule splicing modifier still is enrolling participants at sites in several European countries and in Australia.

“Epilepsy is really a classical neurological disorder,” Lars Pinborg told the audience at the European College of Neuropsychopharmacology (ECNP) annual conference on Sunday. “Or is it?” Pinborg, of Rigshospitalet's The Neuroscience Center in Denmark, was chairing a session dedicated to an alternative hypothesis, summed up in the session title: “Is epilepsy a psychiatric disorder?”