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BioWorld - Monday, April 20, 2026
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BioWorld Science, Conferences
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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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Brain illustration
Neurology/Psychiatric

Merck & Co. reports CNS-penetrant macrocyclic LRRK2 inhibitor for Parkinson’s disease

April 3, 2023
Merck & Co. Inc. presented the discovery, SAR-driven optimization and characterization of leucine-rich...
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Genetic/Congenital

Novel AAV-based gene therapy for liver glycogen storage disease type IX presented

March 31, 2023
Liver glycogen storage disease type IX (GSD IX) accounts for 25% for all GSD cases, with a prevalence of 1 out of 100,000 patients. GSD IX is caused by deficiency in phosphorylase kinase (PhK), which is comprised of four subunits (α2, β, δ and γ2), with γ2 being the catalytic domain.
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