gene therapy

By using phages to deliver genes for therapeutic proteins, biologic drug delivery could become much easier.

A gene therapy targeting organs beyond the brain could improve quality of life and possibly extend survival in children with the neurodegenerative disease.

Michelle Werner and her team at Alltrna are developing novel tRNA therapeutics to overwrite mutations shared by hundreds of rare genetic diseases.

Enzyme replacement therapy has been the mainstay of Pompe disease. New and emerging therapies may provide more options for patients.

New dual AAV vector delivery overcomes challenges in delivering large genes.

The gene therapy treats the rare genetic disease AADC deficiency by delivering a functional gene into the brain.

Industry leaders name the clinical trials and developments they’re most excited to see in the new year.

New gene therapies in clinical trials seek to treat Alzheimer’s disease and heal the brain with a dose of helpful genetic material.

Previous genome editing techniques only work when delivered early in life. This approach could help preserve hearing that is lost later on. 

A new method called “REVeRT” expands the capabilities of gene delivery platforms by transporting larger genes than previously possible.