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Home » Topics » Science » Drug design, drug delivery and technologies

Drug design, drug delivery and technologies
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Illustration of mast cell emitting granules
Drug design, drug delivery & technologies

Dual-action synthetic peptides could fight superbugs

June 2, 2025
By Mar de Miguel
No Comments
A peptide with a dual mechanism of action – it dissolves the bacterial membrane and activates the immune system – could be an effective weapon against microorganisms that have evolved ways to evade antibiotics, as superbugs do. Scientists at the University of Pennsylvania (UPenn) have designed stable synthetic peptides that activate mast cell receptors, which are cells involved in the innate and adaptive immune response. This dual approach eliminates bacteria and recruits neutrophils to finish the job.
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Art concept for mouse model
Drug design, drug delivery & technologies

From mice to big animal models in gene therapy for rare diseases

May 23, 2025
By Mar de Miguel
No Comments
The lack of animal models that mimic human disease impedes the study of many pathologies that still lack treatment beyond symptom relief. This is what has happened so far with PURA syndrome, a rare disorder affecting brain development for which a mouse model has finally been developed. Other times, small and large models exist, but an effective treatment remains elusive, as is the case with Krabbe disease, a fatal disease in children that could be prevented with the advances in gene therapy.
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Silhouette of head and brain with DNA double helixes
Neurology/psychiatric

Armamentarium, the new genetic weapon to study brain disorders

May 22, 2025
By Mar de Miguel
No Comments
A collaboration of scientists from the NIH Brain Initiative consortium has published eight simultaneous studies in Neuron, Cell, Cell Genomics, Cell Reports and Cell Reports Methods, with the results of the Armamentarium project, a new set of gene therapy tools for the research and treatment of human brain disorders. The methodology, based on genetic techniques, RNA detection, genomic enhancers and viral vectors, is designed to access different CNS cell types, neuronal and non-neuronal cells, with common and reproducible protocols now available for any laboratory.
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Nuvec gene delivery system
Gastrointestinal

N4 Pharma completes in vivo study of siRNA and mRNA-loaded oral Nuvec formulations

May 21, 2025
No Comments
N4 Pharma plc has successfully completed the first in vivo study of orally delivered Nuvec using its lead program N4 101, an orally administered anti-inflammatory treatment for inflammatory bowel disease (IBD).
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DNA double helix under a magnifying glass
Drug design, drug delivery & technologies

Base and prime editions to achieve genetic cure

May 21, 2025
By Mar de Miguel
No Comments
Since the development of the base and prime editing technique by David Liu at the Broad Institute, their applications in biomedicine have continued to grow, reaching 17 clinical trials for base editing and one clinical assay for prime editing. The 28th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) marked a historic milestone this year by presenting the first case of treatment with base editors of a baby with a deadly metabolic disease.
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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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Koreabio Humanase KOH Sungho
Drug design, drug delivery and technologies

FDA shift from animal testing fuels organoid, organ-on-chip demand

May 14, 2025
By Marian (YoonJee) Chu
The U.S. FDA’s decision to phase out animal testing for INDs is driving a new market of alternative, nonanimal testing technologies like organoids and organs-on-a-chip, speakers at Bio Korea 2025 said.
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Art concept for transcription factors binding enhancer sequences
Drug design, drug delivery & technologies

AI-designed DNA allows synthetic control of hematopoiesis

May 9, 2025
By Mar de Miguel
No Comments
Scientists at the Center for Genomic Regulation (CRG) have developed an AI-based tool to design thousands of sequences that regulate DNA. They have also synthesized these molecules, called enhancers, to control gene activation in mouse hematopoietic stem cells, which they have tested in vitro.
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Targeted cancer cell
Cancer

Viverita and Boehringer Ingelheim collaborate on cancer target discovery and validation

May 8, 2025
No Comments
Viverita Therapeutics Inc. has established a strategic research collaboration with Boehringer Ingelheim Pharma GmbH & Co. KG aimed at accelerating the discovery and validation of novel therapeutic targets for cancer.
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DNA and silhouette
Genetic/congenital

Organoids plus gene editing bring insights into pediatric metabolic disease

May 8, 2025
By Anette Breindl
No Comments
“I’m a pediatrician in metabolic diseases, and every day in my clinical work I’m confronted with our lack in effective therapies for our patients.” That was the sobering introduction by Sabine Fuchs in her talk at the 2025 Congress of the European Association for the Study of the Liver in Amsterdam this week. The nature of metabolic diseases makes it difficult to develop treatments for them. “There are over 1,500 diseases known by now, and it is just very difficult to develop therapies for each and every individual rare disease.”
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