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BioWorld - Sunday, March 29, 2026
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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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3D rendering of 2-Arachidonoylglycerol (2-AG) endocannabinoid neurotransmitter molecule

ECNP 2025: Endocannabinoids have metabolic role in mental health

Oct. 16, 2025
By Anette Breindl
No Comments
At the 2025 European College of Neuropsychopharmacology (ECNP) Congress in Amsterdam this week, researchers presented new findings on the role of endocannabinoids in stress, addiction … and the integrity of the blood-brain-barrier.
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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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BioFuture 2025: The challenge of accurate drug pricing

Oct. 15, 2025
By Lee Landenberger
No Comments
Government and market demands on companies and their pricing strategies have shifted dramatically in the past 10 years and the pricing landscape continues to change, creating uncertain ground beneath developers’ feet. At the BioFuture conference in New York, a panel discussed how big and small companies go about their pricing strategies and build educated guesses into how that strategy might work out in the market.
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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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LDL particle binding to the LDL receptor
Genetic/congenital

Epigenetic approach shows durable PCSK9 silencing in primates

Oct. 15, 2025
By Mar de Miguel
No Comments
A preclinical study presented at the 32nd Annual Congress of the European Society of Gene and Cell Therapy (ESGCT), held in Seville Oct. 7-10, showed a new epigenetic editing technology that enables durable gene silencing using ELXRs, short for Epigenetic Long-Term X-Repressors. With this approach, scientists at Scribe Therapeutics Inc. successfully inhibited the expression of the PCSK9 gene, a key regulator of cholesterol metabolism, in human cells, mice and nonhuman primates.
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Bio Japan investor panel

Bio Japan 2025: Investors riff about what they look for in Japan

Oct. 14, 2025
By Tamra Sami
No Comments
Japan’s investor community is ramping up both inbound and outbound investment to create a cross-border fertile hub of innovation in Japan, investors said during a panel discussion at Bio Japan 2025 held in Yokohama Oct. 8 to 10.
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