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BioWorld - Thursday, December 25, 2025
Home » Topics » Disease categories and therapies » Neurology/psychiatric

Neurology/psychiatric
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Glass globe with documents and pen

Zenas gets global rights for Innocare BTK inhibitor in $2B+ deal

Oct. 14, 2025
By Lee Landenberger
No Comments
In a deal that could top $2 billion, China-based Innocare Pharma Ltd. licensed the exclusive worldwide development and commercialization rights to the BTK inhibitor orelabrutinib to Zenas Biopharma Inc. for multiple sclerosis and other indications aside from oncology.
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Concept art for adeno-associated viral-based gene therapy.
Neurology/psychiatric

AAV-delivered PPT1 gene therapy shows promise for CLN1 disease

Oct. 14, 2025
No Comments
Neuronal ceroid lipofuscinosis type 1 (CLN1) disease, also known as infantile neuronal ceroid lipofuscinosis, is a rare and fatal neurodegenerative condition. CLN1 disease is caused by a deficiency in the enzyme palmitoyl-protein thioesterase 1 (PPT1) due to biallelic loss-of-function mutations in the gene encoding the lysosomal enzyme, leading to widespread neurological dysfunction and premature death.
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Illustration of motor neuron connecting to muscle fiber
Neurology/psychiatric

Revir’s RTX-117 designated orphan drug for Charcot-Marie-Tooth disease

Oct. 14, 2025
No Comments
Revir Therapeutics Inc.’s RTX-117 has been awarded orphan drug designation by the FDA for Charcot-Marie-Tooth disease. RTX-117 is a small-molecule therapy designed to activate eIF2B to restore translation of cap-dependent mRNAs to normalize protein expression.
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Neurology/psychiatric

Highly selective HDAC6 inhibitor against neurodegenerative disease

Oct. 14, 2025
No Comments
Inhibiting histone deacetylase 6 (HDAC6) has therapeutic potential against several neurodegenerative disorders. A collaboration including researchers from Eikonizo Therapeutics Inc., spanning the U.K., U.S. and France, developed EKZ-438, which has shown strong potential against amyotrophic lateral sclerosis and frontotemporal dementia in preclinical studies.
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3D rendering of 2-Arachidonoylglycerol (2-AG) endocannabinoid neurotransmitter molecule
Neurology/psychiatric

ECNP 2025: Endocannabinoids have metabolic role in mental health

Oct. 14, 2025
By Anette Breindl
No Comments
At the 2025 European College of Neuropsychopharmacology (ECNP) Congress in Amsterdam this week, researchers presented new findings on the role of endocannabinoids in stress, addiction … and the integrity of the blood-brain-barrier.
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Dollar sign and Euro symbol in cogwheels

Kneu secures $5.6M for neurological disorders platform

Oct. 13, 2025
By Shani Alexander
Who knew? In addition to adopting a new name, Kneu Health Ltd. recently raised $5.6 million in a seed funding round for its smart-phone-based platform which helps to monitor neurological disorders to enable timely intervention. The funds secured will be used to expand commercial operations in the U.S., scale deployments for Parkinson's disease and advance the monitoring capabilities of the platform for dementia.
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Digital 3-D illustration of Japan's flag, light effects

Japan reimagines mental health care through AI

Oct. 13, 2025
By Tamra Sami
Japan is reimagining how mental health care can be delivered digitally and proactively, with local governments investing in digital technology to create data-driven safety nets that aim to detect distress and deliver help, presenters said during the Bio Japan 2025 conference in Yokohama, Oct. 8 to 10.
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3D illustration of a nerve cell
Neurology/psychiatric

Targeting EP2 in Schwann cells blocks pain without inhibiting inflammation

Oct. 13, 2025
No Comments
Nonsteroidal anti-inflammatory drugs (NSAIDs) are commonly used to treat inflammatory pain, but they can have severe side effects, including potentially life-threatening gastrointestinal, renal and cardiac toxicity. Their analgesic effect is driven by inhibition of prostaglandin (PG) biosynthesis and subsequent inflammation, but this inhibitory effect on inflammation could delay pain resolution. An optimal approach to managing PG-mediated pain would selectively relieve pain while preserving the PGs’ essential inflammatory and protective functions.
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Neurology/psychiatric

CSM-101 shows potential utility for Gaucher and Parkinson’s diseases

Oct. 13, 2025
No Comments
Lysosomal homeostasis is crucial to the metabolism of certain proteins and lipids that would otherwise accumulate, thus leading to cellular stress and pathology. This is common in diseases such as Parkinson’s disease and Gaucher disease. Researchers set out to find a brain-penetrant small-molecule agonist of the lysosomal channel TRPML1, also known as mucolipin-1.
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Transmission electron microscopy images of the nanoparticles.
Neurology/psychiatric

Putting the BBB back on track: supramolecular polymersomes for Alzheimer’s

Oct. 13, 2025
By Coia Dulsat
No Comments
Researchers at the Institute for Bioengineering of Catalonia (IBEC) and collaborators have introduced a novel therapeutic strategy for Alzheimer’s disease (AD) that leverages the multivalency of supramolecular nanomedicines to reprogram blood-brain barrier (BBB) function, facilitating efficient amyloid-β (Aβ) clearance and restoring cognitive function in animal models.
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