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Epigenetic reprogramming safely modifies multiple genes in T Cells simultaneously for CAR-T therapies

World Pharma News - Wed, 10/22/2025 - 10:00
Arc Institute, Gladstone Institutes, and University of California, San Francisco, scientists have developed an epigenetic editing platform that enables safe modification of multiple genes in primary human T cells, addressing a key manufacturing and scalability challenge in next-generation cell therapies. The research, published October 21, 2025, in Nature Biotechnology, demonstrates how CRISPRoff and CRISPRon can reprogram a patient’s own T cells for therapeutic purposes without the cell toxicity and DNA damage associated with traditional gene editing approaches.

Study helps to understand the role of genetics in the body weight

World Pharma News - Tue, 10/21/2025 - 10:00
Estonia's obesity rate has been steadily climbing and so have scientific efforts to understand why. New study, published in Nature Communications, takes a closer look at the genes behind body weight and how they might point toward future treatments for obesity.

For decades, scientists have sought a single "obesity gene."

Personalized brain stimulation offers new hope for people with hard-to-treat epilepsy

World Pharma News - Mon, 10/20/2025 - 10:00
Doctors and researchers at the University of Pittsburgh and UPMC have developed a new treatment for epilepsy patients who don't respond to medication and aren’t candidates for surgery. Their approach, published today in Nature Communications, uses deep brain stimulation (DBS) that is tailored to each patient's unique brain wiring.

Epilepsy affects more than 50 million people worldwide, and about a third of those do not respond to medication.

New trial finds diabetes drug and nasal insulin improve brain health in  early Alzheimer's disease

World Pharma News - Fri, 10/17/2025 - 10:00
A clinical trial from Wake Forest University School of Medicine shows that two widely available medications, the diabetes drug empagliflozin (Jardiance) and intranasal insulin, safely improve brain health in people with mild cognitive impairment and early Alzheimer's disease. The study, published in Alzheimer’s & Dementia, the journal of the Alzheimer's Association, marks the first time empagliflozin has been tested in non-diabetic patients with Alzheimer's disease. The results show promising effects on memory, brain health and brain blood flow.

Could cardamom seeds be a potential source of antiviral treatment?

World Pharma News - Thu, 10/16/2025 - 10:00
Plant-based materials have traditionally been used to treat a variety of viral infections. Now, researchers have found that cardamom seed extract, as well as its main bioactive ingredient, 1,8-cineole, can have potent antiviral effects through its ability to enhance the production of antiviral molecules known as type I interferons via nucleic acid 'sensors' inside cells.

Plant-based treatments have traditionally been used to treat a wide range of diseases, including viral infections. Medicinal plants and herbs have been a rich source of 'bioactive' compounds (i.e. compounds that have a biological effect), which have been used by pharmaceutical companies in antiviral products.

Acarbose degradation mechanism guides design of next-generation antidiabetic drug

World Pharma News - Wed, 10/15/2025 - 10:00
Type 2 diabetes mellitus (T2DM) is a chronic metabolic disorder marked by hyperglycemia. Acarbose is a potent glycosidase inhibitor widely used in the clinical treatment of T2DM. However, acarbose-preferred glucosidase (Apg) in K. grimontii TD1 degrades acarbose to acarviosine-glucose (M1) and acarviosine (M2), affecting its efficiency.

In a study published in Nature Communications, a research team led by GU Yang from the Shenzhen Institutes of Advanced Technology (SIAT) of the Chinese Academy of Sciences, along with collaborators, revealed the detailed molecular mechanism of Apg hydrolyzing acarbose.

Unlocking the blueprint for a powerful plant-based drug

World Pharma News - Tue, 10/14/2025 - 10:00
Researchers at UBC Okanagan have uncovered how plants produce mitraphylline, a rare natural compound that may help fight cancer.

Mitraphylline belongs to a small group of plant molecules called spirooxindole alkaloids. These compounds have unusual "twisted" ring structures and are known for their strong effects, like fighting tumours and inflammation.

Could slime mold microbes be a source of potent antimicrobials?

World Pharma News - Mon, 10/13/2025 - 10:00
The cellular slime mold Dictyostelium discoideum is a soil microbe that produces diverse natural products with potential antibiotic activity. Previously, three chlorinated compounds had been detected in Dictyostelium, but only the most abundant compound (CDF-1) was identified and shown to be almost as effective an antimicrobial as ampicillin. In research published in FEBS Open Bio, investigators optimized lab culture conditions of Dictyostelium cells to boost the levels of low-abundance chlorinated compounds and to characterize their antimicrobial properties.

Widely prescribed opioid painkiller tramadol not that effective for easing chronic pain

World Pharma News - Fri, 10/10/2025 - 10:00
The strong opioid painkiller, tramadol, is not that effective at easing chronic pain for which it’s widely prescribed, finds a pooled data analysis of the available research, published online in BMJ Evidence Based Medicine.

And it likely increases the risk of serious side effects, including heart disease, the findings indicate, prompting the researchers to conclude that the potential harms of tramadol probably outweigh its benefits, and that its use should be minimised.

Microbiome and nanoparticle discoveries hold promise for treating gut pain

World Pharma News - Thu, 10/09/2025 - 10:00
Abdominal pain is a hallmark of many digestive disorders, including inflammatory bowel disease and irritable bowel syndrome. In an effort to develop targeted treatments for gut pain, scientists have discovered a new enzyme in gut bacteria and are using nanoparticles to deliver drugs inside cells.

Currently, there are no treatments specifically for gut pain, and existing painkillers are often insufficient at managing symptoms.

New antibiotic targets IBD - and AI predicted how it would work before scientists could prove it

World Pharma News - Wed, 10/08/2025 - 10:00
Researchers at McMaster University and the Massachusetts Institute of Technology (MIT) have made two scientific breakthroughs at once: they not only discovered a brand-new antibiotic that targets inflammatory bowel diseases (IBD), but also successfully used a new type of AI to predict exactly how the drug works. To their knowledge, this a global first for the AI.

Combination of two drugs that fight cardiovascular risk could also help treat the most common liver disease

World Pharma News - Tue, 10/07/2025 - 10:00
Metabolic dysfunction-associated steatotic liver disease is the most common liver disease in the world, affecting about one third of the adult population. This disorder is characterized by the accumulation of fat in liver cells, which has severe liver consequences and is also associated with a high mortality rate from cardiovascular disease.​​​​​

The 2025 Nobel Prize in Physiology or Medicine

World Pharma News - Mon, 10/06/2025 - 10:00

The Nobel Assembly at Karolinska Institutet has decided to award the 2025 Nobel Prize in Physiology or Medicine to:

Mary E. Brunkow
Institute for Systems Biology,
Seattle, USA

Fred Ramsdell
Sonoma Biotherapeutics,
San Francisco, USA

Shimon Sakaguchi
Osaka University,
Osaka, Japan

"for their discoveries concerning peripheral immune tolerance"

Immune cell ‘signatures’ could help guide treatment for critically ill patients

World Pharma News - Fri, 10/03/2025 - 10:00
When a patient enters the emergency department in critical condition, doctors must quickly run through a crucial list of questions: Does the patient have an infection? If so, is it bacterial or viral? Do they require treatment? Can the patient recover at home safely or do they need to be hospitalized?

Even when an infection is diagnosed, the treatment plan isn’t always clear.

Novartis receives FDA approval for Rhapsido® (remibrutinib), the only oral, targeted BTKi treatment for chronic spontaneous urticaria (CSU)

World Pharma News - Thu, 10/02/2025 - 10:00
Novartis announced that Rhapsido® (remibrutinib) received US Food and Drug Administration (FDA) approval as an oral treatment for adult patients with chronic spontaneous urticaria (CSU) who remain symptomatic despite H1 antihistamine treatment. Rhapsido is a pill taken twice daily and does not require injections or lab monitoring. It is the first FDA-approved Bruton’s tyrosine kinase inhibitor (BTKi) for CSU.

Meet your worm avatar: How microscopic worms are helping find new drugs for rare diseases

World Pharma News - Wed, 10/01/2025 - 10:00
The research, led by Dr André Brown and the Behavioural Phenomics group at the LMS, was published last week in BMC Biology. It represents a step toward solving a major challenge in medicine: how to develop treatments for the thousands of rare genetic diseases that currently have none. The work builds on a previous study published earlier this year in eLife, and together they mark a shift in how we can model these diseases and test potential treatments at scale.

Experts call for change of heart on hormone replacement therapy after breast cancer

World Pharma News - Tue, 09/30/2025 - 10:00
Breast cancer survivors with severe menopausal symptoms should be supported to make an informed decision about whether to have hormone replacement therapy or not, according to an interdisciplinary panel of experts including researchers from UCL.

The study, published in Menopause, provides the most comprehensive review to date on the use of hormone replacement therapy (HRT), also known as menopausal hormone therapy (MHT), in women who have been treated for breast cancer.

Researchers show novel immune system boost helps fight cancer cells

World Pharma News - Mon, 09/29/2025 - 10:00
In experiments with mouse models of breast, pancreatic, and muscle cancers, researchers at Johns Hopkins All Children’s Hospital report new evidence that a novel means of boosting the natural immune system prevents cancer recurrence and improves survival.

The study, published in Nature Immunology, was federally funded by the National Cancer Institute/NIH.

Low-cost drug shows promise for patients with life threating respiratory infections

World Pharma News - Fri, 09/26/2025 - 10:00
A widely available and affordable drug has been shown to be effective in treating seriously ill COVID-19 patients, according to a new international study led by researchers at the Australian National University (ANU) in collaboration with King's College London.

The study analysed data from almost 500 patients hospitalised with COVID-19 across six countries.

New drug and enzyme class found to have anti-ageing properties

World Pharma News - Thu, 09/25/2025 - 10:00
Researchers from Queen Mary University of London’s School of Biological and Behavioural Sciences, using the simple fission yeast as a model, have shown that new TOR inhibitor rapalink-1 prolongs chronological lifespan.

The new study, published in Communications Biology journal by Juhi Kumar, Kristal Ng and Charalampos Rallis, sheds light on how drugs and natural metabolites can influence lifespan through the Target of Rapamycin (TOR) pathway.