Explore how humanized liver chimeric mouse models smooth the transition between the laboratory and clinical trials.

How to detect impurities during gene therapy vector production

Adeno-associated viral vectors can deliver gene therapies, but AAV-neutralizing antibodies might prevent the medicines from working.

The midwestern state has quietly laid the groundwork for a biotech hub.

Canines treated with an adeno-associated viral (AAV) vector showed evidence that the therapeutic DNA held within the virus can integrate into the host genome, risking the activation of oncogenes.

The US Food and Drug Administration halts a study by Solid Biosciences after a patient experiences severe side effects following treatment.

Preliminary data from Sarepta Therapeutics has exceeded expectations, causing a surge in company stock prices this week.

Biotech stocks fell in response to the news. 

Pancreatic cells engineered to produce insulin did not immediately provoke an immune response.

Researchers deploy ancestors of today’s adeno-associated viruses to deliver gene therapies without immune system interference.

In a mouse model of a rare disease, scientists have figured out how to reduce the elevated cancer risk tied to a gene therapy treatment.

The European Commission approves the Western hemisphere’s first gene therapy, aimed at correcting a lipid-processing disorder.