Researchers at Admare Bioinnovations, Lifearc and Provincial Health Services Authority (PHSA) have disclosed DNA-dependent protein kinase (DNA-PK) inhibitors reported to be useful for the treatment of cancer.
University of Oxford scientists have presented data from deep proteomics of cerebrospinal fluid (CSF) in search of proteins with diagnostic or prognostic value in amyotrophic lateral sclerosis (ALS). Analysis was performed using CSF samples from 40 ALS patients, 15 controls (healthy individuals) and 8 mimicking conditions.
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).
Astrivax NV has been awarded a €3 million grant by Flanders Innovation & Entrepreneurship (VLAIO) to advance development of the company’s therapeutic vaccine targeting chronic hepatitis B.
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.
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.
DNA topoisomerase I (TOP1) is an enzyme that has a direct role in transcriptional regulation, and thus has an impact on cell growth and may be considered a therapeutic target for cancer treatment. Previous research showed that high concentration release of nitric oxide (NO) together with TOP1 inhibition may be an effective strategy in promoting tumor cell apoptosis.
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.
Pasithea Therapeutics Corp. has reported promising preclinical results from two in vivo studies evaluating the antitumor efficacy of PAS-004 (CIP-137401), a small-molecule allosteric inhibitor of mitogen-activated protein kinase kinase 1 and 2 (MEK 1/2), in NRAS mutation cancer xenograft models.
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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