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Category: biotech/medical – Page 147

Precision DNA editing targets root cause of severe childhood epilepsy in preclinical study

Gene editing can repair a DNA error in mice that causes Dravet syndrome, a rare, incurable, and potentially deadly form of childhood epilepsy. After the edit, the mice have far fewer seizures and live much longer. As published in Science Translational Medicine, the results suggest that a one-time genetic correction could someday treat the root cause of the disease rather than just managing its symptoms. The work represents a major step for genetic medicine, as restoring disease-relevant brain function with gene editing tools remains a major challenge.

The study also reflects growing momentum behind gene editing as a therapeutic platform for rare diseases. In February 2026, the Food and Drug Administration issued its Plausible Mechanism Framework guidance, outlining a regulatory pathway for individualized therapies targeting specific genetic conditions. It recognizes that for rare genetic diseases, a well-characterized biological mechanism can serve as the foundation for approval where large clinical trials are not feasible.

“For families affected by Dravet syndrome, our study provides proof of concept that a genetic correction approach could have real impact, a future with treatments that don’t just manage the disease but actually address its cause,” said Matthew Simon, a senior study director at The Jackson Laboratory (JAX) Rare Disease Translational Center (RDTC) who co-led the study. “We’re at an inflection point in genetic medicine, where we can now actually repair the DNA itself.”

Chemists discover and isolate a new boron–oxygen molecule

Oxygen is a cornerstone of chemistry, largely because it is so good at building the organic molecules that make up our world. Some oxygen-based compounds called peroxides are famous for being highly reactive—they act like oxygen delivery trucks, transferring atoms to other molecules. This process is essential for everything from creating new medicines to industrial manufacturing.

In a study published in Nature Chemistry, researchers from the labs of MIT professors Christopher C. Cummins and Robert J. Gilliard, Jr. have revealed a brand-new type of peroxide containing boron. This molecule, called a dioxaborirane, represents a major advance in a field where such structures were long-proposed, but considered too unstable to actually isolate.

Enhancing Non-small Cell Lung Cancer Susceptibility to Anti-PD-1/PD-L1 Therapy through PD-L1 Ligand–Ir(III) Complex Conjugates

Immunotherapy targeting programmed cell death protein 1 (PD-1) and programmed death ligand 1 (PD-L1) has transformed the management of several types of cancers, including non-oncogene-addicted non-small cell lung cancer (NSCLC) [1], although its efficacy remains limited by resistance mechanisms and constraints inherent to monoclonal antibodies [1]. To overcome these drawbacks, small-molecule PD-L1 inhibitors have been developed, and we previously contributed by identifying the nanomolar triazine-based ligand Tr-10 [2]. In parallel, combinatorial strategies aimed at improving the efficacy of anti-PD-1/PD-L1 immunotherapy have gained increasing attention. Notably, platinum-based chemotherapy combined with immune checkpoint inhibitors is recommended as a first-line treatment for advanced NSCLC with PD-L1 expression <50% [3]. Here, we investigated a novel combination involving our anti-PD-L1, Tr-10 [2], and a bis(phenyl-pyridine)iridium(III) complex, Ir-2 (Fig. 1A) [4]. Iridium (Ir) complexes, unlike platinum drugs, are chemically inert and induce endoplasmic reticulum (ER) stress and overproduction of reactive oxygen species (ROS) [5,6], both culminating in damage-associated molecular pattern (DAMP) release and immunogenic cell death (ICD). Moreover, their photophysical properties enable PD-L1-targeted bioimaging when coupled with PD-L1 ligands (Fig. S1) [7].

Immunosenescence and Inflammaging as Drivers of Neurodegeneration: Cellular Mechanisms, Neuroimmune Crosstalk, and Therapeutic Implications

Aging is accompanied by profound alterations in immune function, termed immunosenescence, and by a chronic, low-grade inflammatory state known as inflammaging. These processes are increasingly recognized as central drivers of age-related neurodegenerative diseases, including Alzheimer’s Disease, Parkinson’s Disease, Amyotrophic Lateral Sclerosis and Multiple Sclerosis. In the central nervous system, senescent microglia and astrocytes lose their homeostatic and neuroprotective functions, while systemic immune aging and blood–brain barrier dysfunction further amplify neuroinflammation and impair protein aggregate clearance. This sustained pro-inflammatory environment promotes synaptic dysfunction, neuronal loss and cognitive decline.

Engineered brain ‘bypass’ that rewires specific circuits could boost resilience to stress

Broken or disrupted circuits in the brain contribute to many neurological disorders. A new custom-built biological “wire” developed at Duke University School of Medicine points the way toward a new treatment approach—bypassing broken brain connections, rather than relying on long-term medication or external stimulation.

Researchers led by Kafui Dzirasa, MD, Ph.D., have developed a technology called LinCx that allows scientists to create new electrical connections between carefully chosen neurons. Unlike existing tools that often influence many cells at once, this approach enables selective, long-lasting changes in how defined brain circuits function. The study is published in Nature.

“By introducing a way to plug in new electrical connections with cellular-level precision, our study marks a major step forward in the ability to edit brain circuitry and understand how neural networks give rise to behavior,” said Dzirasa, the A. Eugene and Marie Washington Presidential Distinguished Professor of Psychiatry & Behavioral Sciences, Behavioral Medicine & Neurosciences.

New rules for used prosthetic feet could curb ‘medical equipment graveyards’

Researchers have proposed new standards into the decades-old prosthetic donations market, improving the quality of lower limb prosthetic feet by two-thirds—a major quality of life boost for recipients.

An interdisciplinary team of charities, prosthetists and academics led by King’s College London designed and implemented the very first set of standardized regulations for exporting prosthetic feet to the Global South, reducing unusable donations from 16% to 5%.

In so doing, the team have laid the foundation for improved prosthetics provision in the UK and an ethical framework for a global circular economy of prosthetics—the first of its kind.

Revolutionary Muscle & Fat Therapies: Future of Body Augmentation

This gene therapy company says its muscle-building treatment could last around 5 years.
The surprising part?
They believe the fastest path to market may be cosmetic enhancement using consumer demand to accelerate therapies for frailty and age-related muscle loss.

https://www.oisinbio.com/

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