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Scientist, microscope and dropper
Dental

D-human β defensin 3 is periodontitis treatment hopeful

Oct. 10, 2025
No Comments
Antimicrobial peptides such as human β-defensin 3 (hBD3) have shown promise as a treatment for periodontitis; however, their poor stability and high production costs have hindered their clinical translation until now.
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Infection

Dual-mechanism peptidomimetics to overcome bacterial drug resistance

Oct. 10, 2025
No Comments
In their efforts to develop next-generation antibiotics, researchers from Hunan University and collaborators designed amidated oligopyridinium peptidomimetics.
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Gastrointestinal

Agonist/inverse agonist combo improves primary sclerosing cholangitis

Oct. 10, 2025
No Comments
Primary sclerosing cholangitis is an autoimmune disease affecting the liver in which there is reduced expression and function of the bile acid receptor GPBAR1 in the cholangiocytes.
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Cancer

Novel selective degrader disclosed for transcription factor IKZF2

Oct. 10, 2025
No Comments
IKZF2, a transcription factor in the Ikaros family, plays a key role in stabilizing regulatory T (Treg) cells and promoting immune suppression within the tumor microenvironment (TME). Selective targeting of IKZF2 in intratumoral Tregs has the potential to enhance antitumor immunity and improve the efficacy of immunotherapy, while minimizing immune-related toxicities associated with systemic Treg depletion.
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Pancreas
Cancer

RP-001 taxoid shows promise in pancreatic cancer

Oct. 10, 2025
No Comments
Recurv Pharma Inc. has developed a novel taxoid compound formulated in an oil-in-water nanoemulsion, named RP-001, as a potential therapeutic approach for the management of pancreatic ductal adenocarcinoma (PDAC), either as a single agent or combined with immune checkpoint inhibitors.
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3D illustration of acute lymphoblastic leukemia in blood.
Immuno-oncology

IND approval for Allterum’s 4A10 for acute lymphoblastic leukemia

Oct. 10, 2025
No Comments
Allterum Therapeutics Inc. has obtained IND clearance from the FDA for 4A10, paving the way for initiation of a first-in-human phase I trial in patients with relapsed or refractory acute lymphoblastic leukemia (ALL). 4A10 is a monoclonal antibody with a human immunoglobulin G subclass 1 (IgG1) backbone that specifically binds to CD127 (IL-7Rα).
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Cancer

Clarifying ALDH1A expression patterns helps target right isoform

Oct. 10, 2025
No Comments
Basal-like breast cancer is a particularly aggressive subtype of the malignancy, for which it has been difficult to identify good therapeutic targets. In collaboration, European researchers mined public transcriptome datasets to show that the aldehyde dehydrogenase 1A isoform ALDH1A3 was predominantly expressed in epithelial cells within basal-like tumors, whereas the isoforms ALDH1A1 and ALDH1A2 were predominantly expressed in stromal and immune-associated cells.
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Illustration of DNA double helixes inside drop of blood
Hematologic

Gene therapy for hemophilia stalls but doesn’t stop at ESGCT

Oct. 10, 2025
By Mar de Miguel
No Comments
While recent advances in gene therapy have offered unprecedented options for patients with hemophilia, new data presented at the 32nd Annual Congress of the European Society of Gene and Cell Therapy (ESGCT), held in Seville Oct. 7-10, revealed persistent concerns regarding the durability of these treatments and their potential liver toxicity.
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Other news to note for Oct. 9, 2025

Oct. 9, 2025
Additional early-stage research and drug discovery news in brief, from: Circio, IGC Pharma, JCR Pharmaceuticals, Nurexone Biologic, Resalis Therapeutics, Telomir Pharmaceuticals.
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Neurology/psychiatric

Casma Therapeutics identifies MCOLN1 activators

Oct. 9, 2025
Casma Therapeutics Inc. has developed bicyclic heteroaromatic compounds acting as mucolipin-1 (MCOLN1; TRPML1) activators reported to be useful for the treatment of Duchenne muscular dystrophy, neurodegeneration, atherosclerosis, cancer, infections, liver, lysosomal storage and inflammatory bowel diseases.
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