The FDA is said to be making a U-turn on uniQure’s one-time gene therapy candidate for Huntington’s disease despite the ...

A new West Coast biotech has emerged into the busy in vivo cell therapy space, this time with the backing of Nobel Prize-winning CRISPR pioneer Jennifer Doudna, Ph.D. Azalea Therapeutics has bloomed ...

Shortly after taking reins at BioMarin, CEO Alexander Hardy laid out three paths the company could take for its struggling ...

Virginia Tech researchers have shown that memory loss in aging may be reversible. Using CRISPR tools, they corrected molecular disruptions in the hippocampus and amygdala, restoring memory in older ...

Health Secretary Robert F. Kennedy Jr. called the treatment 'a powerful glimpse into the future of personalized medicine' ...

Pivotal results from uniQure’s gene therapy for Huntington’s disease have brought new light to patients who have known only ...

This article examines key innovations driving scalable, high-yield purification of recombinant AAVs and how they’re helping ...

Mother Daphne Graskewicz-Prado of Southern Illinois told Newsweek: “I wasn’t going to accept the terminal diagnosis.” ...

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 ...

Even with Inflation Reduction Act (IRA)-related pressure looming for its top product, Teva Pharmaceuticals is continuing to ...

UniQure shares soared on positive AMT-130 Huntington's disease data, but plunged after the FDA reversed its stance on ...

The brain drug developer plans to slash R&D expenses by about 60% in the wake of the FDA surprisingly rejecting its rare disease drug Vyglxia.