gene-therapy

A new type of cell therapy shows promise as the first treatment for advanced liver disease, following results from a clinical trial.

A rapidly advancing area of biomedical innovation is shining a spotlight on miR-128-3p, a small yet powerful molecule with the potential to reshape how diseases—especially cancer—are detected, monitored, and treated. This microRNA, part of a broader class of non-coding RNAs, plays a critical role in regulating gene activity and maintaining cellular homeostasis.

Many disease-driving proteins sit outside cells, where they have been difficult to remove with conventional medicines. A new study shows how they can be linked to the cell’s own waste-disposal system and sent for degradation. That opens a new way to treat diseases in which the problem is not only what a protein does, but that it keeps being there.

Nature Communications, Published online: 23 May 2026; doi:10.1038/s41467-026-73380-x Pancreatic ductal adenocarcinoma (PDAC) classical and basal subtypes can interconvert. Here, the authors discover that ZNF274 loss drives a classical to basal transition in PDAC via suppression of ZEB1 and HERV-driven interferon signaling, increasing the sensitivity to CDK7 inhibition.

Nature Communications, Published online: 23 May 2026; doi:10.1038/s41467-026-73545-8 In mice, a next-generation gene therapy for spinal muscular atrophy improved survival and motor function more than a Zolgensma-like treatment, with fewer signs of liver and heart toxicity, supporting its promise as a safer, more effective approach.

Pancreatitis is a complex inflammatory disease driven by both genetic and environmental factors. It poses substantial clinical challenges due to a lack of disease-modifying therapies.

Nature Medicine, Published online: 22 May 2026; doi:10.1038/d41591-026-00027-1 A rare case of genomic integration of an adenovirus vector led to a child developing a tumor — successfully removed by surgery — which highlights the importance of long-term safety monitoring in patients who receive gene therapy.

Engage Biologics Inc., a preclinical biotechnology company pioneering non-viral DNA delivery, announced that it has been acquired by Eli Lilly and Company. Engage is developing the Tethosome platform, a novel non-viral DNA delivery system designed to overcome key limitations in DNA delivery, includi...

Our two guests this week: Quin Wills, CEO of Ochre Bio, and Stéphane Barges, CEO of Lexogen. The post The problem at the heart of drug discovery: Lexogen & Ochre Bio on the power of AI on human data appeared first on Labiotech.eu . © Labiotech UG and Labiotech.eu. Unauthorized use and/or duplication of this material without express and written permission from this site’s author and/or owner is st…

The life science group Sartorius has opened a new competence center in Freiburg, Germany, dedicated to the development and production of quality-critical materials for the cell and gene therapy market. In Freiburg, the company manufactures essential components such as cytokines and growth factors us...

Scientists are uncovering a surprising truth about aging cells: some may damage the body, while others help protect it. The discovery is fueling a new wave of precision anti-aging therapies aimed at removing only the harmful “zombie” cells without disrupting the body’s natural repair systems.

Nature, Published online: 21 May 2026; doi:10.1038/d41586-026-01593-7 Genetic sleuthing uncovers a rare case of cancer caused by a virus administered as part of a child’s treatment for a genetic disorder.

An experimental gene-silencing therapy safely lowered LRRK2 protein levels in an early Parkinson’s trial, but its effect on symptoms remains untested. An experimental medicine built to silence a gene closely associated with Parkinson’s disease has produced encouraging results in its first clinical test in people, according to a study published in Nature Medicine. The treatment, [...]

Nature Medicine, Published online: 21 May 2026; doi:10.1038/s41591-026-04430-6 This Review surveys the fast-developing landscape of CAR T cells in autoimmune disease from both a technological and clinical standpoint, exploring the concept of deep B cell depletion and immune ‘reset’.

Researchers have identified a key protein that may help failing hearts regain function, offering new insight into why some hearts recover while others do not.

Scientists at the UCLA Health Jonsson Comprehensive Cancer Center have developed a new cytokine-armored CAR-T cell therapy that helps the immune system better attack aggressive brain tumors in mice while reducing dangerous side effects that have long limited immune-based treatments for glioblastoma, one of the deadliest and most treatment-resistant brain cancers.