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BioWorld - Thursday, April 23, 2026
Breaking News: Best of BioWorld Science: Q1Breaking News: Best of BioWorld: Q1Breaking News: Best of BioWorld: Q1
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Illustration of brain in head highlighting the blood-brain barier.
Drug design, drug delivery & technologies

An adeno-associated virus to deliver therapeutic genes efficiently to the brain

May 20, 2025
No Comments

It is far easier and safer to inject drugs into veins than directly into the brain, yet it is extremely difficult for systemically delivered drugs to cross the blood-brain barrier and achieve therapeutic concentrations there.


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3D Rendering of tumor microenvironment
Immuno-oncology

New oncolytic virus expressing FLT3L enhances antitumor immunity by activating dendritic cells

May 20, 2025
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Oncolytic viruses trigger immunogenic cell death in tumors, releasing signals that activate innate immune cells and promote tumor-specific T-cell responses. While some oncolytic viruses, including FDA-approved T-VEC, have shown safety and some clinical benefit, their limited effectiveness as standalone treatments underscores the need for combination therapies.
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Ocular

BMP4 inhibition offers dual benefit in AMD: antifibrotic and antineovascularization effects

May 20, 2025
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Age-related macular degeneration (AMD) is the primary cause of blindness in people aged 65 and older in developed nations. Recent estimates suggest that, globally, the number of individuals affected by AMD could rise to approximately 288 million by 2040.
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Photomicrograph of diffuse large B-cell lymphoma
Cancer

MALT1 inhibitor SY-12696 proves efficient for treating BTKi-resistant DLBCL

May 20, 2025
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Mucosa-associated lymphoid tissue lymphoma translocation protein 1 (MALT1) is an important factor from a complex downstream of Bruton tyrosine kinase (BTK) in the B-cell receptor (BCR) signaling pathway.
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CAR T cell attacking cancer cells
Immuno-oncology

PRGN-3008 exerts potent antitumoral activity and surpasses conventional CAR T

May 20, 2025
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CAR T-cell therapy for B-cell malignancies has still to be improved regarding durability, manufacturing complexity or toxicity, among others. Precigen Inc. has presented data regarding the preclinical development and efficacy of PRGN-3008, a PD-1-expression inhibitor cell therapy targeting CD19 that was built in a next-generation ultra CAR T platform.
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Illustration of kidneys floating over gloved hand
Nephrology

Sanegene Bio’s SGB-3383 cleared to enter clinic in China for complement-mediated kidney diseases

May 20, 2025
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Suzhou Sanegene Bio Inc. has gained clinical trial approval in China for SGB-3383 for the treatment of complement-mediated kidney diseases, including IgA nephropathy, C3 glomerulopathy, immune complex-mediated membranoproliferative glomerulonephritis and atypical hemolytic uremic syndrome.
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High-density lipoprotein particles and red blood cells
Endocrine/metabolic

Repair Biotechnologies’ REP-0003 designated orphan drug for homozygous familial hypercholesterolemia

May 20, 2025
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Repair Biotechnologies Inc.’s REP-0003 has been awarded orphan drug designation by the FDA for the treatment of homozygous familial hypercholesterolemia (HoFH).
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Cancer

Rationalizing the design of styrylquinolinium derivatives to bind c-Myc G-quadruplexes in breast cancer

May 20, 2025
No Comments
Breast cancer is the most frequent malignancy among women worldwide, and all subtypes of breast cancer involve upregulation of the c-Myc gene, making it a compelling therapeutic target. G-rich regions of the c-Myc promoter can form G-quadruplex structures, which can be targeted using small molecules containing a styrylquinolinium core, which then downregulate oncogenic c-Myc. The challenge, however, is specificity.
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Woman receiving radiotherapy treatments for cancer
Cancer

Amphiregulin drives metastasis through a paradoxical ‘badscopal’ effect of radiotherapy

May 20, 2025
By Coia Dulsat
No Comments
Researchers at the University of Chicago have shed light on the role of tumor-promoting factors induced by radiotherapy and their potential impact on future therapeutic strategies. The article, published in Nature on May 14, 2025, points to radiation-induced amphiregulin as a key driver of tumor metastasis.
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Cancer cells being destroyed by immunotherapy
Cancer

Avidicure boosting immune activation with $50M seed round

May 19, 2025
By Nuala Moran
No Comments
Newco Avidicure NV arrived on the scene with a hefty $50 million in seed funding to advance novel antibody formats the company says will surpass the best qualities of first-generation antibodies, checkpoint inhibitors, T-cell engagers and antibody-drug conjugates.
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