Congruence Therapeutics Inc. has closed a $39.5 million financing to advance into its portfolio of small-molecule correctors for diseases of protein misfolding into the clinic.
At the ongoing International Conference on Alzheimer’s and Parkinson’s Diseases and Related Neurological Disorders (AD/PD 2026) in Copenhagen, researchers presented details on a new SERCA inhibitor from Synuca Therapeutics ApS – SYN-4569 – as a potential approach to treat multiple system atrophy-cerebellar type (MSA-C).
Shanghai Huilun Pharmaceutical Co. Ltd. has synthesized potassium voltage-gated channel subfamily KQT member 2/3 (KCNQ2/3) activators. They are reported to be useful for the treatment of epilepsy, neuropathic pain, ischemic stroke and neurodegeneration.
Biohaven Therapeutics Ltd. has presented prodrugs of betahistine reported to be useful for the treatment of obsessive-compulsive disorder, trichotillomania, Tourette syndrome, amyotrophic lateral sclerosis, multiple sclerosis and more.
Researchers from the China Pharmaceutical University and Guangdong Pharmaceutical University (China) have unveiled the crucial role of the alternative splicing of E2A in myogenic progression and demonstrated that PTBP1, by controlling E2A alternative splicing, is a critical regulator of myogenesis.
Ovid Therapeutics Inc. has announced plans to initiate a phase I study of OV-4071, an oral, direct activator of potassium-chloride cotransporter 2 (KCC2), having received Australian Human Research Ethics Committee (HREC) approval and clinical trial notification (CTN) acknowledgement from Australia’s TGA.
A new way of understanding Alzheimer’s disease, based on biological inflection points that mark decisive moments in the progression of the disorder, could change how new drugs are developed to achieve more effective therapies. This new perspective could rethink strategies that depend not so much on the target itself, but on the precise moment at which it is addressed.
Exon skipping therapies based on antisense phosphorodiamidate morpholino oligomer (PMO) have great potential to restore dystrophin in the skeletal muscle and treat Duchenne muscular dystrophy (DMD). Entrada Therapeutics Inc. has developed an endosomal escape vehicle conjugated to DMD exon skipping PMOs (exon 51 skipping), ENTR-601-51, for the potential treatment of DMD.
VST Bio Corp. has closed its series A financing designed to support the company’s work in vascular diseases with the development of first-in-class antibodies targeting edema and inflammation for patients with ischemic stroke.
Neurodegenerative disease and cognitive decline cannot be explained by a single process. Beta-amyloid plaques, hyperphosphorylated tau, alpha-synuclein, activated microglia and astrocytes, altered receptors such as TREM2, mitochondrial dysfunction, epigenetic changes and cerebrovascular alterations all seem to contribute to the development of dementia in Alzheimer’s disease (AD). While scientists attempt to address each of these elements, prevention is growing as a primary goal.
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