The two main approaches for discovering disease genes reveal distinct aspects of biology, a new study shows. While both ...

"FDA halts uniQure’s plans for Huntington’s disease gene therapy" was originally created and published by Pharmaceutical ...

We have written about the promise of gene therapy to correct genetic disease many times. An exciting development happened ...

The approval was based on data from a Phase II trial, two retrospective chart review studies and an expanded access programme ...

Embargoed for 9am CET (3am ET) on Monday November 3rdAmgen Ventures-backed AAVantgarde Bio has raised $143 million in series B funding that the Italian biotech will use to | Amgen Ventures-backed ...

Genetic testing is transforming healthcare through personalized medicine, enabling early diagnosis, targeted treatments, and ...

Harmony also touts late-stage asset EPX-100 (clemizole hydrochloride), which the biotech is running through a pair of phase 3 trials in Dravet syndrome and Lennox-Gastaut Syndrome— both rare forms of ...

Motor neuron diseases, such as amyotrophic lateral sclerosis (ALS) and hereditary spastic paraplegia (HSP), share physical ...

The first surgery in a clinical trial testing the safety and efficacy of gene-edited pig kidney transplants in patients with ...

Image credit: You-Chiun Chang/University of Texas at Austin. Some genetic disorders—such as cystic fibrosis, hemophilia and Tay Sachs disease—involve many mutations in a person’s genome, often with ...

Understanding how cells turn genes on and off is one of biology's most enduring mysteries. Now, a new technology developed by chemist Brian Liau and his collaborators at Harvard offers an ...