“Beyond UniQure, the pipeline includes highly promising approaches that will ideally move the goal from merely managing ...

Long-term follow-up shows 95% success rate, no serious complications in largest ADA-SCID gene therapy study to date ...

An experimental genetic therapy can cure most cases of the 'Bubble Boy' disease, ADA-SCID. In the rare disorder, children are ...

A one-time gene therapy using a patient’s own stem cells has effectively cured a deadly immune disorder in 95% of treated ...

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 ...

A new study has brought scientists one step further in the direction of developing a cure for a brutal group of rare brain ...

When she was born, doctors informed Opal Sandy’s parents that their child would never hear. At the age of 11 months, she had ...

Opus Genetics has shared data on its gene therapy in children with an ultrarare eye disease, setting the biotech up to talk to the FDA about the next steps for the program. North Carolina-based Opus ...

AGC Biologics, your friendly CDMO expert, announced an agreement to provide comprehensive development and manufacturing for Rarity PBC's gene therapy.

The goal of gene therapy is to permanently cure hereditary diseases. One of the most promising technologies for this is the ...

A CRISPR-Cas9-based therapy targeting the gene encoding transthyretin greatly reduced transthyretin levels in patients with ...