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BioWorld - Saturday, December 27, 2025
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Precision targeted therapy concept illustrated by dart hitting bullseye of target
Cancer

At AACR-NCI-EORTC 2025, new strategies reshape the field

Oct. 24, 2025
By Coia Dulsat
No Comments
During the first poster session of the 2025 AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics, held in Boston, several presentations highlighted novel strategies that move beyond traditional antibody-drug conjugate (ADC) payloads and targets.
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Biomarkers

FGF5 gene involved in excessive hair growth

Oct. 22, 2025
No Comments
Hirsutism is the excessive hair growth in women in male-typical areas, usually associated with androgen excess and other conditions such as polycystic ovary syndrome (PCOS). The genetic basis of hirsutism remains poorly understood; researchers from the University of Oulu and collaborators performed a meta-analysis of genome-wide association studies from 4,834 cases of hirsutism and 352,966 controls across three cohorts.
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Endocrine/metabolic

Preclinical data on single-dose gene editing system for AATD

Oct. 21, 2025
No Comments
Alpha-1 antitrypsin deficiency (AATD) is a monogenic disease caused by mutations in the SERPINA1 gene, which encodes alpha-1 antitrypsin (AAT), a serine protease inhibitor mainly produced by hepatocytes.
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Biomarkers

New link between PRRX1 gene and open-angle glaucoma

Oct. 21, 2025
No Comments
Primary open-angle glaucoma (POAG) is among the leading causes of irreversible blindness. Early detection and treatment are crucial for vision preservation, and research to identify genes associated with POAG is ongoing.
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Illustration demonstrating structure of the human eye and organization of retinal cells
Genetic/congenital

rAAV2-U1a-hELP1 rescues RGC function in familial dysautonomia

Oct. 20, 2025
No Comments
Familial dysautonomia (FD) is an autosomal recessive neurodevelopmental disorder.
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Purple-tinted test tubes and dropper
Biomarkers

miR-1265 emerges as a biomarker of meningioma

Oct. 17, 2025
No Comments

Meningiomas are usually grade I benign tumors that can turn into aggressive grade II or III malignant tumors. Genome-wide methylation analysis has shown that microRNA 1265 (miR-1265) is hypermethylated in meningiomas.


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Fallopian tubes, ovaries and uterus
Biomarkers

AKR1B1 and AKR1B10 as prognostic markers in endometrial cancer

Oct. 17, 2025
No Comments
Endometrial cancer is the most prevalent gynecological malignancy in high-income countries, and the discovery of new biomarkers may aid in earlier detection and improve the prognosis of patients.
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Art concept for inflammation in the intestines
Gastrointestinal

Orchard’s OTL-104 is safe and efficacious in Crohn’s disease model

Oct. 16, 2025
No Comments

NOD2 deficiency is involved in the pathogenesis of Crohn’s disease (CD) due to failure of gut innate immunity and loss of intestinal tissue homeostasis. Orchard Therapeutics Ltd. has presented preclinical data on OTL-104, human hematopoietic stem cell (HSC) gene therapy for the treatment of NOD2-deficient CD.


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Neurology/psychiatric

Selective GABA-A α3 modulator shows efficacy in essential tremor

Oct. 15, 2025
Essential tremor is a movement disorder marked by involuntary, rhythmic shaking in the hands but sometimes affecting the head, voice and other areas. Its exact cause is still unknown. Recent research suggests that dysfunction in the neurotransmitter systems, particularly involving γ-aminobutyric acid (GABA), contributes significantly to the disorder.
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Cancer

[68Ga]NOTA-R-54 arises as imaging probe for CXCR4+ tumors

Oct. 15, 2025
No Comments
CXC chemokine receptor 4 (CXCR4) is a receptor overexpressed in several tumor types and associated with tumor aggressiveness and risk of metastasis, resistance and recurrence. A novel PET radiopharmaceutical tracer and CXCR4 ligand, [68Ga]NOTA-R-54, was developed and tested for potential use in the treatment of lung cancer.
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