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Home » Topics » Small molecule, BioWorld Science

Small molecule, BioWorld Science
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Man piecing together a puzzle
Neurology/psychiatric

Discovery and preclinical results of a Rac1 inhibitor for Alzheimer’s

April 17, 2025
At the 2025 International Conference on Alzheimer’s and Parkinson’s Diseases and Related Neurological Disorders (ADPD), researchers from Beijing Joekai Biotechnology LLC presented the discovery process and preclinical results for 50561.
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Cancer cell targeted in crosshairs
Cancer

Specifically targeting TUBG1 shows potential in RB1-deficient tumors

April 11, 2025
Researchers from Lund University and collaborators have investigated the potential of selectively targeting TUBG1 as a therapeutic strategy in cancer treatment.
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Female hands holding heart on cardiogram
Endocrine/metabolic

Northstrive partners with Yuva Biosciences on obesity and cardiometabolic diseases

April 11, 2025
Northstrive Biosciences Inc., a subsidiary of PMGC Holdings Inc., has entered into a development and license agreement with Yuva Biosciences Inc. to discover and develop novel pharmaceutical treatments for obesity, type 2 diabetes and other cardiometabolic conditions using Yuva’s mitochondrial science-focused platform Mitonova, powered by AI.
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Gastrointestinal

PANX1 inhibitor for colitis management presented at ACS Spring 2025

April 2, 2025
Pannexin 1 (PANX1) forms channels that may release signaling metabolites that are involved in a variety of pathophysiological processes, such as asthma, diabetes, hypertension or inflammatory bowel disease (IBD), among others. Inhibition of PANX1 when dysregulated has been proposed as a therapeutic approach in the treatment of IBD.
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Beta cells
Endocrine/metabolic

Stabilizing ‘molecular glues’ protect β cells in type 2 diabetes

April 2, 2025
In type 2 diabetes patients, inadequate hyperglycemic control can lead to insulin-secreting β-cell exhaustion, dedifferentiation and eventual loss. Since adult β cells have limited proliferative capacity, this final stage cannot be reversed.
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Neurology/psychiatric

Cryptic pocket in CB1 receptor is better target for analgesia

April 2, 2025
Cannabinoid CB1 receptors have been a potential target for nonopioid-based pain treatment, but actually targeting the pathway has been hindered by issues with tolerance and unwanted CNS side effects. Peripherally selective CB1 agonists developed to overcome these problems have not fully resolved these issues, meaning the peripheral selectivity has to be substantially enhanced.
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Doctor measuring patient's waist
Endocrine/metabolic

Novo Nordisk licenses Lexicon’s preclinical obesity drug in $1B deal

March 31, 2025
By Karen Carey
As companies continually search for next-generation obesity prospects, one of the leaders in the disease space, Novo Nordisk A/S, has obtained an exclusive license to a preclinical-stage, first-in-class, small-molecule inhibitor of Acyl-CoA synthetase 5 (ACSL5) developed by Lexicon Pharmaceuticals Inc.
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Photomicrograph of bone marrow aspirate showing myeloblasts of acute myeloid leukemia
Cancer

Pleco holds pre-IND meeting with FDA on PTX-252 for AML

March 26, 2025
Pleco Therapeutics BV has held a successful pre-IND meeting with the FDA to discuss the continued development of PTX-252, an intravenous formulation designed to treat patients with acute myeloid leukemia (AML).
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3D dollar sign
Neurology/psychiatric

Augustine Therapeutics closes $85M series A for HDAC6 inhibitors

March 26, 2025
By Nuala Moran
Eight months after announcing the $18.5 million first tranche of its series A, Augustine Therapeutics has closed the oversubscribed round at $85 million and is now ready to begin clinical development of its novel histone deacetylase-6 (HDAC6) inhibitors.
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Inflammatory

Novartis adds Kyorin’s preclinical MRGPRX2 antagonist in $777M deal

March 5, 2025
By Tamra Sami
Kyorin Pharmaceutical Co. Ltd. has out-licensed its preclinical candidate, KRP-M223, and its back-up compounds to Novartis Pharma AG in a deal worth up to $777.5 million. Under the terms, Novartis gains an exclusive worldwide license to develop, manufacture and commercialize Kyorin-discovered KRP-M223.
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