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Sickle cell illustration
Hematologic

ASH 2023: For broad reach, meaningful innovation still means small molecules

Dec. 11, 2023
By Anette Breindl
Spirits were high at the 2023 Annual Meeting of the American Society of Hematology (ASH), buoyed by U.S. FDA approval of the first two gene therapies for sickle cell disease (SCD) the day before the conference kicked off in San Diego.
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Biomarkers

IGFBP7 SNP tied to the reversal phenotype of amyotrophic lateral sclerosis

Dec. 7, 2023
Researchers have defined an amyotrophic lateral sclerosis (ALS) reversal phenotype as having an initial diagnosis of ALS but subsequently showing a progressive and sustained clinical improvement, based on an unusual case they found.
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Neurology/Psychiatric

TRV-045 demonstrates efficacy for both reversal and prevention of CIPN in mice

Dec. 7, 2023
At the recent American College of Neuropsychopharmacology meeting, Trevena Inc. presented preclinical data for the novel selective sphingosine 1-phosphate S1P1 receptor modulator TRV-045, being developed for the treatment of chemotherapy-induced peripheral neuropathy (CIPN).
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Neurology/Psychiatric

Longboard’s 5-HT2C superagonist yields positive results in preclinical models of epilepsy

Dec. 5, 2023
Serotonin 5-HT2 receptor agonists have shown efficacy in treating seizure disorders, such as epilepsy. At the American Epilepsy Society meeting this week, Longboard Pharmaceuticals Inc. presented data on their 5-HT2C receptor superagonist LP-352 for the treatment of epilepsy.
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Neurology/Psychiatric

Favorable preclinical profile of investigational gene therapy for TLE reported

Dec. 4, 2023
Temporal lobe epilepsy (TLE) is the most common form of focal epilepsy characterized by recurrent seizures originating in the hippocampus and with sprouting of mossy fiber axons that contribute to new recurrent synaptic excitability in the dentate gyrus (DG).
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Brain waves
Neurology/Psychiatric

Iama Therapeutics’ IAMA-6 shows promise in preclinical mesial temporal lobe epilepsy

Dec. 4, 2023
Intracellular chloride levels are crucial for physiological brain development and correct functioning. On the other hand, aberrant expression of chloride importer NKCC1 (basolateral Na-K-Cl symporter, SLC12A2) and exporter KCC2 (K-Cl cotransporter 2, SLC12A5) is involved in neurodevelopmental and neurologic conditions, including mesial temporal lobe epilepsy (MTLE), among others, leading to an imbalance between excitatory and inhibitory signals.
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Infection

Bifunctional molecule capsid modulator and cGAS agonist achieves complete cure in AAV-HBV mice

Nov. 30, 2023
LW-231, under development by Shanghai Longwood Biopharmaceuticals Co. Ltd., is a novel bifunctional molecule designed to simultaneously modulate hepatitis B virus (HBV) capsid assembly and activate cGAS-STING pathway. Preclinical data were recently presented.
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Colorized epithelial cells.
Cancer

Stem, immune and nervous cells all forces for tumor progression

Nov. 30, 2023
By Mar de Miguel
One of the difficulties for preventing the evolution of a tumor is that cancer progression can be promoted by undifferentiated or migrating cells whose states could follow different directions. At the 40th edition of Barcelona Biomed conferences at the Institute for Research in Biomedicine (IRB Barcelona), which took place from Nov. 27 to 29, 2023, and is entitled “Cancer in Context: Cellular, Tissue, and Organismal Determinants of Malignant Fates,” Angela Nieto presented her latest data on epithelial-to-mesenchymal transition (EMT).
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Neurons and amyloid plaques
Neurology/Psychiatric

New Alzheimer's disease model includes cholinergic dysfunction

Nov. 29, 2023
Researchers from Nagoya City University presented the development of a new mouse model with cholinergic dysfunction and amyloid pathogenesis for Alzheimer's disease.
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Dermatologic

New pemphigus vulgaris mouse model better mimics human disorder

Nov. 29, 2023
Pemphigus vulgaris is an autoimmune skin disease mediated by autoantibodies to desmoglein-3 (DSG3). Since the adult murine model of this disease is quite limited, researchers aimed to develop a novel active adult pemphigus vulgaris murine model.
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