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.
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.
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.
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.
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.
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.
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.
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.
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.
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.