N EW ORLEANS – A single injection of a gene-edited protein was deemed productive and safe in patients with dyslipidemia that ...

The FDA previously placed two clinical studies on hold, including the Phase III trial in which the liver toxicity occurred. Intellia is working with experts to create a risk management program for nex ...

Your heart will not just give you fear and uncertainty after a heart attack; it can leave scar tissue that stiffens your ...

Opus Genetics has completed a Type B Regenerative Medicine Advanced Therapy (RMAT) meeting with the US Food and Drug ...

Pioneering CRISPR trial shows single infusion permanently lowers bad cholesterol by 50% in patients who don't respond to medication.

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

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

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 ...

After telegraphing an investigation in November, the FDA has moved to restrict the use of bluebird bio’s gene therapy Skysona in certain patients. The FDA has updated Skysona’s indication, allowing it ...

Until recently, even the most advanced gene therapies could only be given after a child was born—often racing against time to prevent irreversible damage. In the first part of this series, we explored ...

Onasemnogene abeparvovec gene therapy post-nusinersen or risdiplam showed meaningful motor improvements in children with SMA, with a manageable safety profile. The study cohort included older, heavier ...

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