A pioneering gene therapy could help treat a rare seizure disorder called Dravet syndrome, according to new clinical trial ...

Bayer has discontinued an early-stage clinical gene therapy for a rare genetic disorder in favor of a similar candidate. | Bayer has discontinued an early-stage clinical gene therapy for a rare ...

A senior FDC official said the product is a “failed therapy” and confirmed that regulators have asked uniQure to run a new ...

In a strikingly blunt briefing, a senior FDA official accused uniQure of pushing “distorted” data to mask a “failed” therapy ...

Gene therapies have the potential to cure some diseases, but they are extraordinarily expensive. Location can also be a big ...

After three months, OPGx-BEST1 was well tolerated with no reports of observed ocular inflammation or adverse events.

UCLA researchers use lipid nanoparticles to insert full CFTR gene into airway cells, restoring function and opening new avenues for CF therapy.

Intellia is now able to continue both Phase III trials of nex-z. Credit: Piotr Swat / Shutterstock.com (Piotr Swat / Shutterstock.com) The US Food and Drug Administration (FDA) has released the ...

The FDA has granted Fast Track designation to AFTX-201 for the treatment of BAG3-associated dilated cardiomyopathy.

Researchers at the University of Basel have developed a gene therapy that could potentially treat a rare and currently fatal ...

A Frisco mom’s yearslong fight led to a gene therapy breakthrough for the rare SLC6A1 disorder. Her son Maxwell is the first patient treated and is already showing promising progress.

CRISPR gene therapy Casgevy can nearly eliminate sickle cell crises, but difficult stem‑cell collection is delaying access ...