Efficacy News and Research

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Targeted nanoparticles enable in vivo production of CAR T cells for cancer therapy

Researchers present a new method to safely and preferentially generate CAR T cells directly inside the body using targeted lipid nanoparticles that deliver mRNA directly to T cells.

20 Jun 2025

Exploring the intricate epigenetic regulatory network of lncRNAs in hepatocellular carcinoma

A new review published in Genes & Diseases explores the intricate epigenetic regulatory network of long noncoding RNAs (lncRNAs) in hepatocellular carcinoma (HCC), shedding light on the profound impact of DNA methylation, histone modifications, RNA methylation, and microRNAs on lncRNA transcription.

20 Jun 2025

Targeting IGF2BP1 offers a new approach to cancer therapy

The RNA-binding protein IGF2BP1 has emerged as a key regulator of cancer hallmarks, influencing tumor proliferation, metastasis, immune evasion, and resistance to cell death.

20 Jun 2025

New app helps relieve dry eye through optimized blinking routine

An Aston University optometrist, Professor James Wolffsohn, has determined an optimum blinking exercise routine for people suffering with dry eye disease, and has developed a new app, MyDryEye, to help them complete the routine to ease their symptoms.

19 Jun 2025

NHCS and Mayo Clinic join forces to advance global cardiovascular care

The National Heart Centre Singapore (NHCS) and Mayo Clinic have collaborated under a Memorandum of Understanding (MOU) to accelerate cardiovascular innovation and research, and to foster knowledge for future cardiovascular care worldwide.

19 Jun 2025

FIM programs show promise in improving diet quality and food security

Food is medicine (FIM) programs that incorporate healthy food into health care for people with or at high risk for chronic disease show great potential in improving diet quality and food security, but additional research is needed to understand clinical outcomes, according to a systematic review of randomized controlled trials in the U.S conducted by the American Heart Association.

19 Jun 2025

Full tumor DNA sequencing becomes standard for children with cancer

Readouts of the full DNA of children with cancer at diagnosis have been implemented as the standard of care in a European first.

19 Jun 2025

New gene therapy strategy reactivates dormant genes to treat blood disorders

Researchers have found a promising new method for gene therapy. They successfully restarted inactive genes by bringing them closer to genetic switches on the DNA called enhancers.

19 Jun 2025

New meta-analysis highlights effective non-drug therapies for knee osteoarthritis

Knee braces, water therapy and exercise are the most promising non-drug therapies for treating knee osteoarthritis, according to a new meta-analysis publishing June 18, 2025 in the open-access journal PLOS One by Yuan Luo of the First People's Hospital of Neijiang, China.

19 Jun 2025

Mass photometry recognized by U.S. Pharmacopeia in new AAV reference standards for gene therapy quality

Refeyn, the company behind pioneering mass photometry technology, announces that mass photometry has been recognized by the U.S. Pharmacopeia (USP)—and referenced in the new draft USP general chapter <1067>—as a key orthogonal method for characterizing USP's new adeno-associated virus (AAV) reference standards.

19 Jun 2025

Global guideline released by WHO on managing sickle cell disease in pregnancy

The World Health Organization (WHO) today released its first-ever global guideline on the management of sickle cell disease (SCD) during pregnancy, addressing a critical and growing health challenge that can have life-threatening consequences for both women and babies.

19 Jun 2025

New switchable CAR-T therapy enters phase 1 trial for advanced breast cancer

The Calibr-Skaggs Institute for Innovative Medicines, the drug development division of Scripps Research, today announced a milestone in cancer treatment with the dosing of the first patient in a phase 1 trial (NCT06878248) evaluating CLBR001 + ABBV-461, a modular, switchable chimeric antigen receptor T cell (sCAR-T) therapy in patients with advanced or metastatic breast cancer who have no suitable treatment options.

18 Jun 2025

Qureight's synthetic study arms validate clinical efficacy in pioneering rare lung disease treatment

Qureight, a deep-learning analytics company accelerating drug development through AI-powered data curation, today announced that its digitally-twinned synthetic control arm has been used in a breakthrough clinical trial application in place of a human placebo group, marking a world-first in lung disease research.

17 Jun 2025

Uncovering unexpected impacts of popular GLP-1 diabetes drugs

GLP-1 drugs show promise in diabetes management, offering heart and kidney benefits, but also highlight the need for vigilance regarding new health risks.

17 Jun 2025

Researchers identify a new treatment strategy for neuroblastoma

Researchers at Karolinska Institutet and Lund University in Sweden have identified a new treatment strategy for neuroblastoma, an aggressive form of childhood cancer.

16 Jun 2025

Deep B cell depletion offers new hope for rheumatologic and musculoskeletal diseases

CD19-CAR T-cell treatment is a novel option of deep B cell depletion with promising results across different rheumatologic and musculoskeletal diseases (RMD).

15 Jun 2025

New nanoparticle platform breaks through MRSA biofilms

A major cause of hospital-acquired infections, the super bacteria Methicillin-resistant Staphylococcus aureus (MRSA), not only exhibits strong resistance to existing antibiotics but also forms a dense biofilm that blocks the effects of external treatments.

14 Jun 2025

Boosting CAR T cell survival to improve solid tumor therapy

Chimeric Antigen Receptor (CAR)-T cells are a promising cancer therapy that are made from the patient's own T cells, which are reprogrammed to fight their cancer. One of the limitations of CAR-T cell therapy is the ability of these cells to survive long enough to target the entire tumor.

13 Jun 2025

Empagliflozin preserves kidney function in heart attack patients

SGLT2 inhibitors have become a major drug used to treat diabetes, heart failure, and chronic kidney disease.

13 Jun 2025

New drug combination shows high remission rates in acute myeloid leukemia

A combination therapy that adds a recently approved drug to the current standard of care for newly diagnosed acute myeloid leukemia (AML) showed high rates of complete remission in an early-phase clinical trial conducted at UNC Lineberger Comprehensive Cancer Center and 11 other sites nationwide.

13 Jun 2025