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Biomarkers

Study finds ENPP6 gene behind CAKUT pathogenesis

April 5, 2023
Congenital anomalies of the kidneys and urinary tract (CAKUT) remain the main cause of chronic kidney disease before the age of 25 years, and account for about 40% of childhood end-stage renal diseases. Studies in Xenopus species have shown ENPP6 knockdown to lead to impaired pronephros development, and mutations in the ENPP6 paralogue PIGN gene have been tied to CAKUT.
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Kidneys
Nephrology

PTP4A1 knockout ameliorates renal injury in murine model

April 5, 2023
Protein tyrosine phosphatase 4A1 (PTP4A1) is known to promote tumor growth and migration of tumoral cells, but its role in the kidney has not been deeply explored. Researchers from Korea have aimed to investigate the role of PTP4A1 during kidney fibrosis and suggest it as a potential therapeutic target.
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3D illustration of kidney cross section
Nephrology

Unicycive Therapeutics provides preclinical pharmacokinetic profile of UNI-494

April 5, 2023
Nicorandil is a potent K(ATP) channel activator and a sulfonylurea receptor 2 (SUR2) selective agonist launched for the treatment of angina pectoris. Nicorandil binds to ATP-sensitive KATP channels in the mitochondrial inner membrane to preserve mitochondrial function during ischemia.
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Elderly woman holding illustration of brain with missing puzzle piece
Neurology/Psychiatric

ARKD-104, a novel PGRN-enhancing compound to prevent frontotemporal dementia

April 4, 2023
Frontotemporal dementia (FTG-GRN) is a fatal neurodegenerative disease caused by heterozygous mutations in the GRN gene, encoding progranulin (PRGN). Researchers at Arkuda Therapeutics Inc. reported on ARKD-104, a novel potent orally bioavailable PGRN-enhancing compound that increased PRGN secretion and lysosomal granulins in multiple relevant cell lines.
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Neurology/Psychiatric

Preclinical profile of a novel cholesterol 24-hydroxylase inhibitor reported

April 4, 2023
Researchers from Takeda Pharmaceutical Co. Ltd. have hypothesized that inhibition of CH24H may...
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Artery and plaque
Endocrine/Metabolic

Merck & Co. presents macrocyclic peptide MK-0616 as a potent, oral PCSK9 inhibitor

April 4, 2023
Merck & Co. revealed the structure of an orally active and potent proprotein convertase subtilisin/kexin-type 9 (PCSK9) inhibitor macrocyclic peptide, MK-0616, which is being developed for the potential treatment of hypercholesterolemia and atherosclerosis.
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Illustration representing connection between brain and gut microbiome.
Neurology/Psychiatric

AX-5006: a gut-selective, small-molecule inhibitor of CsgA for Parkinson’s disease

April 3, 2023
The accumulation of α-synuclein fibrils in the brain is the primary pathogenic hallmark in Parkinson’s disease (PD), the second most prevalent neurodegenerative disorder. Current therapies are mainly focused on late-stage symptoms and hence there is a need to find therapies tackling the disease at early stages. Recent studies validate that gut microbiome of PD patients as different from the one of healthy people.
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Tree in the shape of human head losing leaves
Neurology/Psychiatric

Procognitive action of IRL-942 in the preclinical setting reported

April 3, 2023
One of the features associated with early-stage Parkinson’s disease (PD) diagnosis is the presence of cognitive impairment. Other neurological conditions such as dementia, synucleopathies or amyloid disorders share the same impact on mental deterioration where the disruption of frontal-subcortical circuits is involved.
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Cancer

NX-2127: a small-molecule degrader targeting BTK, Ikaros and Aiolos for B-cell malignancies

April 3, 2023
Nurix Therapeutics Inc. presented the structure, discovery, preclinical profile and early phase I...
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Brain and encephalography
Neurology/Psychiatric

Novel Kv7.2/3 activator ETX-123 disclosed by researchers from Eliem Therapeutics

April 3, 2023
First-generation Kv7.2/3 channel openers show significant efficacy but also display relevant...
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