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. Read More

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. Read More

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. Read More

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. Read More

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. Read More

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. Read More

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. Read More

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. Read More

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. Read More

X-Chem Inc. has synthesized 17-β-hydroxysteroid dehydrogenase 13 (HSD17B13; 17-β-HSD 13) inhibitors reported to be useful for the treatment of nonalcoholic steatohepatitis (NASH). Read More

Merck Sharp & Dohme LLC has identified receptor-interacting serine/threonine-protein kinase 1 (RIPK1; RIP-1) inhibitors reported to be useful for the treatment of amyotrophic lateral sclerosis and inflammatory disorders. Read More

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). Read More

Tarapeutics Science Inc. has divulged tyrosine-protein phosphatase non-receptor type 11 (PTPN11; PTP-2C; SHP-2) inhibitors reported to be useful for the treatment of cancer, fibrosis, immunological, eye and cardiovascular disorders. Read More

Celros Biotech Co. Ltd. has described cytochrome b-245 heavy chain (CYBB; NOX2) and NADPH oxidase 4 (NOX4) inhibitors reported to be useful for the treatment of neuroinflammatory disorders. Read More

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. Read More

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