Suzhou Genassist Therapeutic Co. Ltd. has announced its pre-IND application of its first base-editing product, GEN-6050, and acceptance by the FDA. GEN-6050 is an in vivo base-editing drug that targets exon 50 skipping in the Duchenne muscular dystrophy (DMD) gene.
Biosplice Therapeutics Inc. has described 1H-pyrrolo[2,3-b]pyridines acting as dual-specificity tyrosine-(Y)-phosphorylation regulated kinase 1A (DYRK1A) inhibitors reported to be useful for the treatment of cancer, diabetes and neurological disorders.
F. Hoffmann-La Roche Ltd. and Hoffmann-La Roche Inc. have synthesized GABA-A receptor γ1 subunit positive allosteric modulators (PAMs) reported to be useful for the treatment of cognitive and neurological disorders.
Patients suffering from neuropathic pain, a chronic condition, have few treatment options and often develop tolerance to existing pain therapy that decreases its effectiveness. Now, a group of scientists from the University of Alabama at Birmingham (UAB) and the Baylor College of Medicine have described the pathophysiological mechanism of initiation, transmission and maintenance of neuropathic pain and identified a potential therapeutic target to treat it efficiently.
The University of Vermont has identified ADCYAP receptor type I (PAC1 receptor) antagonists reported to be useful for the treatment of pain, eating, neurological and stress disorders, substance abuse and dependency.
Nerve growth factor (NGF) actions are involved in pain perception and mediated via the tyrosine kinase TrkA (tropomyosin receptor kinase A) in neural cells. Mutations in the gene encoding for TrkA cause hereditary sensory and autonomic neuropathy type IV (HSAN IV), a rare autosomal recessive disorder characterized by loss of responses to noxious stimuli, anhidrosis and cognitive impairment. Until now, appropriate animal models to study the mechanisms underlying HSAN IV were missing.
The map of the genetic activity of the risk genes that affect the central nervous system (CNS) reveals the molecular signatures associated with the neurological pathologies in this organ. A study by researchers at McGill University in Canada and the Allen Institute for Brain Science in Washington compared 40 brain diseases with this technology and classified them into five groups whose members shared the same transcriptional pattern.
Recent findings in murine studies have unveiled a circuit linking the basolateral amygdala to the anterior cingulate cortex (BLA-ACC circuit) as a key for chronic pain-induced depression symptoms; researchers aimed to clarify the mechanisms behind this phenotype. Stimulation of this circuit led to the upregulation of semaphorin 4A (SEMA4A), which was the focus of the study.
Therini Bio Inc. has closed a US$36 million series A financing round that will support its work on developing fibrin-targeted therapies for diseases driven by chronic inflammation, including Alzheimer’s disease, multiple sclerosis and retinal diseases such as diabetic macular edema.
Researchers from Athira Pharma Inc. presented preclinical efficacy data for ATH-1105, a positive modulator of hepatocyte growth factor (HGF)/MET, being evaluate for the treatment of amyotrophic lateral sclerosis (ALS).
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