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Liver illustration
Endocrine/metabolic

Liver-targeted siRNA improves body composition in mouse models

July 8, 2024
Large-scale human genetic studies have revealed that loss-of-function INHBE variants are associated with a lower abdominal to gluteofemoral fat ratio, improved metabolic profile, and reduced fasting glucose levels.
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Dollar sign droplet above test tube
Immuno-oncology

Myricx Bio closes series A to advance novel ADCs

July 8, 2024
Myricx Bio (Myricx Pharma Ltd.), a spin out from Imperial College London and the Francis Crick Institute, has closed a series A financing raising £90 million ($114 million) to support its work focused on the discovery and development of a novel class of payloads for antibody-drug conjugates (ADCs).
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Elderly woman and jigsaw puzzle
Neurology/psychiatric

Glutaminyl cyclase inhibition is effective in Parkinson’s disease

July 8, 2024
Targeting human glutaminyl-peptide cyclotransferase (QPCT) has emerged as a potential strategy in Alzheimer’s disease (AD). As the main substrates of this enzyme are involved in biological processes associated with neurodegenerative diseases, Parkinson’s disease (PD) and others may be other options to explore as well.
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Illustration of clot forming in blood vessel
Cardiovascular

Antibody shows potential in thrombotic thrombocytopenic purpura

July 8, 2024
Von Willebrand factor (vWF) plays a crucial role in hemostasis, and elevated levels are associated with risk of thrombosis. Gain-of-function mutations in the C-domain of vWF are linked to an increased risk of thrombosis. Previous work has shown that disrupting cysteine-rich C-domain in vWF led to reduced platelet recruitment in a shear-dependent fashion, making it a promising target for pharmacology. Apollo Therapeutics Ltd. has presented data on antibodies targeting the C-domain of vWF, including AP-21 as antithrombotics.
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Eye wireframe illustration
Ocular

DP Technology nominates Lp-PLA2 inhibitor candidates

July 8, 2024
Beijing DP Technology Co. Ltd. has nominated DPT-0415, a novel small molecule targeting lipoprotein‐associated phospholipase A2 (Lp-PLA2), as a preclinical candidate for the treatment of diabetic retinopathy (DR) and diabetic macular edema (DME).
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CAR T cell with implanted gene strand
Immuno-oncology

ORIC-613 is dual-target CAR T-cell therapy

July 8, 2024
Researchers from Oricell Therapeutics (Shanghai) Co. Ltd. presented the discovery and preclinical characterization of ORIC-613, a dual-targeting CAR T-cell therapy being developed for the treatment of pancreatic and gastric cancer.
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Neurology/psychiatric

HDAC inhibitor improves Charcot-Marie-Tooth disease in mice

July 8, 2024
Therapeutics for peripheral neuropathies, such as Charcot-Marie-Tooth (CMT) disease, are still quite limited and focus on managing symptoms. Augustine Therapeutics NV has presented an orally available histone deacetylase 6 (HDAC6) inhibitor, AGT-100216, for the treatment of CMT. AGT-100216 was designed to work through improving axonal regeneration and nerve functional restoration.
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Other news to note for July 5, 2024

July 5, 2024
Additional early-stage research and drug discovery news in brief, from: Pneumagen.
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Musculoskeletal

Caregen discovers new peptides with cartilage regenerative effects

July 5, 2024
Caregen Co. Ltd. has described peptides reported to be useful for the treatment of cartilage injury, fracture, intervertebral disc herniation, degenerative intervertebral disc disorder, osteoarthritis, osteomalacia and muscular injury.
Read More
Cancer

New GTPase KRAS inhibitors disclosed in Risen Pharma patent

July 5, 2024
Researchers at Risen (Shanghai) Pharma Eng Co. Ltd. and Risen (Suzhou) Pharma Tech Co. Ltd. have divulged GTPase KRAS inhibitors reported to be useful for the treatment of cancer.
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