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Cerebellum, brain stem, spinal cord
Neurology/Psychiatric

AUT-00201 ameliorates cerebellar dysfunction and seizure susceptibility in model of EPM7

Dec. 12, 2023
Researchers from Autifony Therapeutics Ltd. presented preclinical data for the novel potent Kv3 channel positive modulator, AUT-00201, which is currently being evaluated in early clinical studies for the treatment of patients with progressive myoclonus epilepsy type 7 (EPM7).
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Neurology/Psychiatric

HDAC6 inhibitor ACY-738 shows protective effects in axonal degeneration

Dec. 12, 2023
Microtubule acetylation and impaired axonal transport have been proposed as mechanisms causing amyotrophic lateral sclerosis (ALS). Furthermore, histone deacetylase 6 (HDAC6) is known to play a role in acetylation of α-tubulin, a subunit of microtubules. ACY-738 is an HDAC6 inhibitor that has been reported to slow neuron degeneration in Alzheimer’s disease and ALS models by increasing acetylation of α-tubulin.
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Silhouette made of crumpled paper illustrating depression
Neurology/Psychiatric

CV-1238, a PDE4B inhibitor with promising antidepressant and antipsychotic activity in vivo

Dec. 12, 2023
Psychiatric indications, such as depression, schizophrenia and bipolar disorder, share a common feature of elevated expression of pro-inflammatory markers in the periphery and/or central nervous system. Based on this, it is believed that combined immuno- and neuromodulatory activities of phosphodiesterase 4 (PDE4) inhibitors may represent a promising new therapeutic strategy for various psychiatric indications.
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Photomicrograph of bone marrow aspirate showing myeloblasts of acute myeloid leukemia
Immuno-oncology

Novel CD123xCD3 bispecific IgM antibody shows strong efficacy in AML models

Dec. 12, 2023
High expression of CD123, the IL-3 receptor α chain (IL-3α), is observed on both leukemic blasts and leukemic stem cells, thus suggesting it can be considered an attractive therapeutic target in AML.
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Biomarkers

Researchers unveil novel HBB gene mutation involved in β-thalassemia

Dec. 12, 2023
It is known that heterozygous mutations in the HBB gene, which encodes β-globin, are the cause of inherited β-thalassemia. A new case report describes a novel frameshift mutation in the HBB gene leading to a dominant form of β-thalassemia.
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Human NK cell
Hematologic

ASH 2023: NK cells championed as way to trifecta of fast, cheap, good – with engineering help

Dec. 12, 2023
By Anette Breindl
Katy Rezvani received this year’s E. Donnall Thomas Prize for her work on natural killer (NK) cells at the annual meeting of the American Society of Hematology (ASH). It was not love at first sight, though.
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Neurology illustration
Neurology/Psychiatric

NX-210c, a promising disease-modifying drug for neurodegenerative disorders

Dec. 11, 2023
Subcommisural organ (SCO)-spondin is a brain-specific glycoprotein essential for neurogenesis with a high impact on neuronal development; thus, reduction in the production of SCO-spondin may lead to a lack of regeneration in neurological disorders.
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Neurology/Psychiatric

Selective P2X7 pathway activation enhances skeletal muscle regeneration in ALS mice

Dec. 11, 2023
Previous research has demonstrated that systemic administration of a P2X7 agonist improved motor performance in a mouse model of amyotrophic lateral sclerosis (ALS) through enhancing satellite cells and the muscle pro-regenerative activity of infiltrating macrophages.
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Neural network
Neurology/Psychiatric

DLX-001 promotes structural neuroplasticity without hallucinogenic or cardiotoxic side effects

Dec. 11, 2023
Researchers from Delix Therapeutics Inc. recently presented preclinical pharmacology data for DLX-001, a novel orally bioavailable, nonhallucinogenic neuroplastogen being developed for the treatment of neuropsychiatric diseases.
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Neurology/Psychiatric

CAPN2 inhibitor might reduce axonal degeneration in ALS

Dec. 11, 2023
Axonal degeneration is a key early pathogenic driver of amyotrophic lateral sclerosis and other neurodegenerative diseases. Activation of calpain-2 (CAPN2) is thought to be the critical effector behind axonal degeneration. Amylyx Pharmaceuticals Inc. has presented data on their CAPN2 inhibitor antisense oligonucleotide (ASO) AMX-0114 as a potential therapeutic for axonal degeneration.
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