Gene Therapy News and Research

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Gene therapy is an experimental technique that uses genes to treat or prevent disease. In the future, this technique may allow doctors to treat a disorder by inserting a gene into a patient’s cells instead of using drugs or surgery.

Lipid nanoparticles help CAR T cells melt pancreatic cancer barriers

Often diagnosed when surgery is no longer an option, pancreatic cancer is one of the most difficult cancers to treat and has one of the lowest rates of survival among major malignancies.

31 Mar 2026

FDA approves first gene therapy for rare pediatric immune disorder

Dr. Donald Kohn has been developing gene therapies for rare pediatric immune disorders for over 30 years.

27 Mar 2026

Abselion appoints Dale Gordon as chair

With Dale Gordon as Chair, Abselion strengthens its leadership in bioprocessing, focusing on innovative protein quantification solutions for global markets.

From Abselion 23 Mar 2026

Review examines clinical trial phases in drug development

Announcing a new article publication for BIO Integration journal. Clinical trials bridge basic research and clinical application, serving as essential steps in drug development.

13 Mar 2026

The 0.05 EU/mg breakthrough: How Sino Biological ProPure™ is redefining the Ultra-Low Endotoxin standard

When we discuss bacterial infections, we often think of exotoxins—those soluble proteins actively secreted by bacteria—while overlooking endotoxins, which are more insidious but equally dangerous.

From Sino Biological Inc. 12 Mar 2026

Hanson Wade - 7th Gene Therapy Analytical Development Summit Europe

Clinical and Regulatory Momentum Puts Gene Therapy Analytics in the Spotlight Ahead of the 7th Gene Therapy Analytical Development Summit.

12 Mar 2026

4basebio strengthens commercial leadership, promoting Christine Wolosin to Chief Commercial Officer

4basebio PLC, a specialist in synthetic DNA manufacturing and nucleic acids for next-generation therapeutics, announces the promotion of Christine Wolosin to Chief Commercial Officer.

From 4basebio PLC 10 Mar 2026

Engineered proteins track gene expression in living primate brains

Gene therapy has been successfully used to treat a number of diseases, including immune deficiencies, hereditary blindness, hemophilia and, recently, Huntington's disease, a fatal neurological disorder.

27 Feb 2026

Unexpected behavior observed during interactions between immune cells and dying cells

Over the years, cell biology has built a detailed picture of how cells compartmentalize their internal functions. Central to this organization is the nucleus, which houses the genetic material and is separated from the cytoplasm by a robust nuclear envelope.

25 Feb 2026

Breakthrough gene therapy for sickle cell disease remains out of reach in Africa

As Uganda rolls out mandatory nationwide screening of newborns for sickle cell disease this month, a gene therapy celebrated in the United States and Europe remains financially out of reach for most patients in Africa.

25 Feb 2026

Neutral polymer enables safer DNA delivery into cells

Researchers from Tokyo Metropolitan University have created a new molecule which carries DNA into biological cells, to treat or vaccinate against illnesses.

22 Feb 2026

Genetic mismatch identified as major risk factor in cord blood transplantation

A Japanese study of more than 7,400 patients has identified a genetic mismatch that sharply increases the risk of severe acute graft-versus-host disease (GVHD) following umbilical cord blood transplantation.

20 Feb 2026

Axol Bioscience acquires ophthalmology business from Newcells Biotech

Axol Bioscience Ltd. (Axol), a leading provider of induced pluripotent stem cell (iPSC) technologies for drug discovery and research, today announced that it has acquired the ophthalmology business of Newcells Biotech (Newcells), a leading drug discovery partner specializing in the development of in vitro models and bespoke assay services.

18 Feb 2026

Lund's new model aims to accelerate patient access to cell and gene therapies

Despite groundbreaking research, many cell and gene therapies do not make it all the way to the patients.

17 Feb 2026

New gene editing approach offers hope for cystic fibrosis patients

UCLA researchers have developed a lipid nanoparticle-based gene-editing approach capable of inserting an entire healthy gene into human airway cells, restoring key biological function in a laboratory model of cystic fibrosis and establishing a potential new path toward mutation-agnostic gene therapy for inherited lung diseases.

17 Feb 2026

Review: Pathogenesis, diagnosis, and management of harlequin ichthyosis

Harlequin ichthyosis (HI) is a rare, severe genetic skin disorder caused by ABCA12 mutations, leading to defective lipid transport and loss of skin barrier function.

17 Feb 2026

Global trends shape progress in cell and gene therapies

Cell and gene therapies, or CGT, have come a long way since they were first introduced. In the last few decades, both cell therapy -- the transplantation of living cells -- and gene therapy -- the use of genetic material to modify cell functions -- have been increasingly incorporated into clinical practice.

13 Feb 2026

Case report examines potential cancer risks after mRNA vaccination

A new case report was published in Volume 17 of Oncotarget on February 6, 2026, titled "Exploring the potential link between mRNA COVID-19 vaccinations and cancer: A case report with a review of haematopoietic malignancies with insights into pathogenic mechanisms."

12 Feb 2026

'Partial reprogramming' of engram neurons restores memory performance in mice

Age-related memory decline and neurodegenerative diseases like Alzheimer's are often thought of as irreversible.

10 Feb 2026

New guidance for the diagnosis and management of inherited hyperbilirubinemia

Inherited hyperbilirubinemia, encompassing Gilbert syndrome, Crigler-Najjar syndrome, Dubin-Johnson syndrome, and Rotor syndrome, represents a spectrum of disorders rooted in genetic defects affecting bilirubin metabolism and transport.

10 Feb 2026