Two dose-limiting toxicities have been recorded in uniQure’s trial evaluating an investigational AAV gene therapy designed to ...

Gene-editing tools like CRISPR have unlocked new treatments for previously uncurable diseases. Now, researchers at the University of British Columbia are extending those possibilities to the skin for ...

A new gene therapy can reverse the effects of heart failure and restore heart function in a large animal model. The therapy increases the amount of blood the heart can pump and dramatically improves ...

The FDA in July last year declined to approve UX111 for Sanfilippo syndrome, a rare neurodegenerative disorder, citing ...

Explore how community blood centers are shaping the future of cell and gene therapies, from customized collections to ...

Researchers at the Nora Eccles Harrison Cardiovascular Research and Training Institute, University of Utah, and the University of Utah School of Medicine, have demonstrated that a gene therapy can ...

Boston biotech Beam Therapeutics has announced initial data from a phase 1/2 trial of its gene therapy for a genetic lung and liver disease, with no serious adverse events reported among the nine ...

The FDA AMT designation verifies NanoMosaic platform's ability to enhance efficiency, product quality, and scalability across AAV gene therapy manufacturing.

Gazi University in Ankara has officially launched the production process for Türkiye's first local and national gene therapy research product. The project, which targets rare genetic diseases, is ...

Qatar set to be among top five best countries in precision health revolutionLifestyle, behaviour changes can help ‘turn on’ g ...