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BioWorld - Wednesday, April 8, 2026
Home » Topics » Drug design, drug delivery and technologies, BioWorld Science

Drug design, drug delivery and technologies, BioWorld Science
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Drug design, drug delivery & technologies

Poly(A) tail mimetics boost gene expression in haploinsufficiency models

April 10, 2025
Writing in Molecular Therapy Nucleic Acids, researchers hypothesized that using poly(A) tail mimetics to enhance mRNA expression from haploinsufficiency-associated genes could be a disease-modifying treatment strategy.
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Influenza viruses with RNA, surface proteins hemagglutinin and neuraminidase
Immune

Orexo, Abera report proof-of-concept data for powder-based intranasal vaccine

April 10, 2025
Orexo AB has announced promising in vivo proof-of-concept data for a powder-based intranasal vaccine candidate based on Abera Bioscience AB’s BERA vaccine platform and formulated with Orexo’s Amorphox technology.
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Neurology/psychiatric

Lilly licenses rights to use Sangamo’s neurotropic AAV capsid in genomic medicines

April 4, 2025
Sangamo Therapeutics Inc. has entered into a license agreement with Eli Lilly and Co., allowing Lilly to use Sangamo’s novel proprietary neurotropic AAV capsid, STAC-BBB, to deliver intravenously administered genomic medicines to treat certain diseases of the central nervous system.
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Neurology/psychiatric

Cryptic pocket in CB1 receptor is better target for analgesia

April 2, 2025
Cannabinoid CB1 receptors have been a potential target for nonopioid-based pain treatment, but actually targeting the pathway has been hindered by issues with tolerance and unwanted CNS side effects. Peripherally selective CB1 agonists developed to overcome these problems have not fully resolved these issues, meaning the peripheral selectivity has to be substantially enhanced.
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Abstract illustration of pig and human with medical motifs
Drug design, drug delivery & technologies

Ten days of normal survival of a pig liver in a human being

April 1, 2025
By Mar de Miguel
Transplanting an animal organ into a human is now a closer reality following the successful xenotransplantation of a genetically modified pig liver into a patient diagnosed with brain death in China. The operation was intended to evaluate organ function over a 10-day period. This is a complex experimental trial that did not involve removing the patient's liver and still requires further study. However, the positive preclinical results suggest this strategy could save the lives of those waiting for a human organ, at least in certain cases.
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Drug design, drug delivery & technologies

New DNA-PK inhibitors disclosed in West Pharmaceutical patent

April 1, 2025
West Pharmaceutical Services Inc. has divulged pyrazolopyridine-containing compounds reported to be useful as DNA-dependent protein kinase (DNA-PK) inhibitors.
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3D illustration of RNA
Neurology/psychiatric

Ono and Reborna partner to discover RNA-targeting small molecules for neurological disorders

March 31, 2025
Ono Pharmaceutical Co. Ltd. has entered into a drug discovery collaboration agreement with Reborna Biosciences Inc. to generate RNA-targeting novel small molecules in the field of the central nervous system.
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Abstract illustration of pig and human with medical motifs
Drug design, drug delivery & technologies

Ten days of normal survival of a pig liver in a human being

March 26, 2025
By Mar de Miguel
Transplanting an animal organ into a human is now a closer reality following the successful xenotransplantation of a genetically modified pig liver into a patient diagnosed with brain death in China. The operation was intended to evaluate organ function over a 10-day period. This is a complex experimental trial that did not involve removing the patient's liver and still requires further study. However, the positive preclinical results suggest this strategy could save the lives of those waiting for a human organ, at least in certain cases.
Read More
3D Rendering of tumor microenvironment
Drug design, drug delivery & technologies

AI-based spatial omics unveils molecular talks in tumors

March 25, 2025
By Mar de Miguel
The three-dimensional analysis of cell types and their locations by spatial transcriptomics provides key information of their interactions within tissues or organs. Based on this technology, scientists at the Wellcome Sanger Institute have developed an AI tool called Nichecompass, which shows a comprehensive view of the cancer microenvironments, the different cells, their locations, and how they communicate with each other through different molecules inside the tumor. This AI could process data in an hour and compare samples before and after a treatment.
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Digital cancer cells illustration
Drug design, drug delivery & technologies

AI reads cancer cells’ 3D shape to predict their drug response

March 24, 2025
By Mar de Miguel
Scientists at the Institute of Cancer Research (ICR) in the U.K. are developing a technology that analyzes, in vitro, how the 3D morphology of cancer cells changes when exposed to a compound, using AI to predict their response to new treatments. The researchers estimate that their methodology could accelerate drug development by 6 years, by ruling out unsuccessful drugs and thus reducing the number of preclinical trials.
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