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BioWorld - Friday, March 27, 2026
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Hematologic

Epigenetic modifier modulator CS-014 shows good clot prevention profile in vivo

Aug. 30, 2023
Under pathologic conditions such as vascular injury or atherosclerosis, the hyperactivation of platelets may lead to occlusive thrombus formation, myocardial infarction or stroke. Although there are several targets for clot prevention validated clinically, these strategies may present bleeding risk as a limitation. Researchers from the University of Michigan have reported on CS-014, a histone deacetylase (HDAC) inhibitor aimed to reduce clot formation without risk of bleeding.
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DNA double helix with sand hour glass
Biomarkers

Biomarker brainteaser: Aging? Or just changing?

Aug. 30, 2023
By Anette Breindl
“Change is the only constant” is an ageless truth. In the search for age-related biomarkers, it is also a prosaic confounding factor. Age-related biomarkers will be critical for the development of antiaging therapeutics. “Nobody is planning to do a life span study in humans,” Eric Verdin told the audience at the 10th Conference on Aging Research and Drug Development in Copenhagen on Monday. “Hence the need for … surrogate markers.” And “we are not there … we are actually quite far from there.”
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3D illustration of Cryptosporidium oocysts releasing sporozoites
Infection

Novartis discloses a novel soft-drug strategy for cryptosporidiosis

Aug. 29, 2023
Cryptosporidiosis is a parasitic infection caused by Cryptosporidium where infected individuals develop profuse watery diarrhea and enteric symptoms such as abdominal pain. In immunocompromised or malnourished children, the infection may become chronic and lead to significant morbidity and mortality rates.
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Colorized transmission electron micrograph of SARS-CoV-2.
Infection

Next-generation oral SARS-CoV-2 Mpro inhibitor disclosed

Aug. 29, 2023
Viral proteases are well-established therapeutic targets in HIV and hepatitis C virus infections. Following the recent COVID-19 pandemic, one of the strategies in place is SARS-CoV-2 main protease (Mpro) inhibition, given the crucial role of SARS-CoV-2 Mpro in the replication of the virus.
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Central nervous system
Neurology/Psychiatric

Methodological, target breadth begets optimism for movement disorder progress

Aug. 29, 2023
By Anette Breindl
“I am not a fortune teller, nor am I a gambler. I will make no bets,” Lorraine Kalia told the audience at the 2023 International Congress of Parkinson’s Disease and Movement Disorders. “But I am optimistic.” At the meeting, which is being held in Copenhagen this week, Kalia, who is a scientist at Toronto Western Hospital’s Krembil Brain Institute and at the University of Toronto’s Tanz Centre for Research in Neurodegenerative Diseases, was giving an overview of “Emerging targets in the clinic” in a plenary session on “Therapeutic strategies for the future.”
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Cancer cell targeted in crosshairs
Cancer

Highly potent and selective MASTL inhibitors disclosed

Aug. 25, 2023
Microtubule-associated serine/threonine kinase-like (MASTL) is involved in cell proliferation, migration, and invasion and for that reason is considered a relevant therapeutic target for cancer treatment. At the recent ACS Fall meeting, researchers from Pfizer Inc. presented the discovery and characterization of a series of potent and selective MASTL inhibitors for the treatment of cancer.
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Illustration highlighting the brain's reward circuit.
Substance Use & Poisoning

First-in-class RXFP3 negative allosteric modulator RLX-33 prevents alcohol relapse in rats

Aug. 24, 2023
New treatment options are needed for the prevention of relapse to alcohol use, the major problem in the treatment of alcohol dependence in humans. The neuropeptide relaxin-3/RXFP3 plays a relevant role in the motivation for the reward process and is considered a possible therapeutic target in addictive behaviors.
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Brain and encephalography
Neurology/Psychiatric

NS-041, a novel KCNQ2/3 activator for the treatment of epilepsy, presented at ACS meeting

Aug. 23, 2023
Voltage-gated potassium channels (KCNQ) control neural excitability and their dysfunction leads to neuronal channelopathies frequently related to...
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Illustration of clot forming in blood vessel
Cardiovascular

Potent and orally bioavailable pyridine-N-oxide FXIa inhibitor from Janssen presented

Aug. 22, 2023
Protease form factor Xia (FXIa) is a therapeutic target for thrombosis prevention due to its well-known contribution to VTE and ischemic stroke in humans. Researchers from Janssen Pharmaceutica NV presented an antithrombotic target FXIa inhibitor obtained through a non-classical interactions strategy to improve the pharmacokinetic and pharmacological activity for the treatment of thrombosis. In the series, the potency was increased by using water-mediated hydrogen bonds to reduce polar hydrogen bond donors and using methyl to displace high-energy waters.
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Genetic/Congenital

Preclinical characterization of SLC6A19 inhibitor JNT-517 divulged at ACS

Aug. 22, 2023
Phenylketonuria (PKU) is an inborn error of metabolism caused by heritable phenylalanine hydroxylase gene mutations that result in decreased metabolism of phenylalanine (Phe) causing brain damage. The most severe phenotype termed PKU occurs when untreated individuals achieve plasma Phe concentrations of >1200 microM/L, which are neurotoxic.
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