Breaking WORLD PHARMA NEWS

New technology facilitates delivery of advanced medicines

Researchers at Karolinska Institutet in Sweden have developed a technique that enables efficient delivery of therapeutic proteins and RNA to cells. The method, presented in Nature Communications, shows promising results in animal studies to deliver gene editors and protein therapeutics.

The method is based on so-called extracellular vesicles (EVs), tiny bubbles that are naturally secreted by cells and can transport biologically active molecules between cells.

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A candidate drug dismantles a metabolic barrier to anti-tumor immunity

A Ludwig Cancer Research study has identified a specific mode of fat uptake by immune cells within tumors that serves as a metabolic checkpoint against anti-cancer immune responses. Harnessing that insight, researchers led by Ludwig Lausanne's Ping-Chih Ho and Yi-Ru Yu - along with Sheue-Fen Tzeng and Chin-Hsien Tsai, former post-docs in the Ho lab who now lead their own labs at Taipei Medical University in Taiwan - have developed a humanized antibody to dismantle that barrier as a potential cancer immunotherapy.

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Novartis to acquire Regulus Therapeutics and farabursen, an investigational microRNA inhibitor to treat ADPKD

Novartis today announced that it has entered into an agreement to acquire Regulus Therapeutics, a San Diego-based, publicly traded (Nasdaq: RGLS) clinical-stage biopharmaceutical company focused on developing microRNA therapeutics. Regulus' lead asset, farabursen, is a potential first-in-class, next-generation oligonucleotide targeting miR-17 for the treatment of autosomal dominant polycystic kidney disease (ADPKD).

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Fungi dwelling on human skin may provide new antibiotics

University of Oregon (UO) researchers have uncovered a molecule produced by yeast living on human skin that showed potent antimicrobial properties against a pathogen responsible for a half-million hospitalizations annually in the United States.

It's a unique approach to tackling the growing problem of antibiotic-resistant bacteria.

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Unlocking a new era in liver cancer treatment: The power of neddylation-targeted therapies

Hepatocellular carcinoma (HCC), the most common form of liver cancer, continues to pose a significant global health challenge due to its high mortality rate and limited treatment options. However, a new frontier in cancer therapy has emerged, focusing on neddylation modification, a crucial cellular process that influences cancer progression. The latest insights into this mechanism provide hope for targeted therapies that could revolutionize the treatment landscape for HCC.

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Scientists finally confirm vitamin B1 hypothesis from 1958

Chemists have confirmed a 67-year-old theory about vitamin B1 by stabilizing a reactive molecule in water - a feat long thought impossible. The discovery not only solves a biochemical mystery, but also opens the door to greener, more efficient ways of making pharmaceuticals.

The molecule in question is a carbene, a type of carbon atom with only six valence electrons. Generally, carbon is stable with eight electrons around it. With only six electrons, it is chemically unstable and highly reactive.

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Empowering antibodies to better activate the immune system

Antibodies are best known for their ability to latch onto and neutralize bacteria, viruses and other pathogens. But these immune proteins can do more than that: They also activate other components of the immune system, which then go to work to clear an infection. A new study from Scripps Research explores the factors that influence how effectively antibodies engage specific immune cells.

Their results, described April 22, 2025 in Cell Reports, indicate that a higher ratio of antibodies to viral protein - in this case, a part of HIV - better engages two specific types of immune cells.

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BlueRock Therapeutics announces publication in Nature of 18-month data from Phase 1 clinical trial for bemdaneprocel, an investigational cell therapy for Parkinson's disease

BlueRock Therapeutics LP, a clinical-stage cell therapy company and wholly owned subsidiary of Bayer AG, announced the publication of the 18-month data from its Phase 1 exPDite clinical trial for bemdaneprocel in the journal Nature.

"The concept of "rebuilding" brain networks that have been lost to disease is compelling," said Claire Henchcliffe, MD, chair of the UC Irvine School of Medicine's Department of Neurology at the University of California, Irvine and one of the study's Principal Investigators,

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Existing drug has potential for immune paralysis

When the immune system does not function properly, individuals become more susceptible to infections caused by viruses, bacteria, or fungi. Researchers from Radboud university medical center have demonstrated that an existing drug can revive immune cells that are not functioning correctly. These findings provide leads for further research in patients admitted to the intensive care unit (ICU) with sepsis.

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Study finds pinworm medication has potential to treat aggressive skin cancer

A common pinworm medication may stop and reverse cancer growth in Merkel cell carcinoma, an aggressive form of skin cancer, according to research led by University of Arizona Cancer Center researchers and published in the Journal of Clinical Investigation.

Merkel cell carcinoma is a rare but fast-growing neuroendocrine cancer that is three to five times more likely than melanoma to be deadly.

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