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 ...

AFTX-201 uses a proprietary capsid to deliver a human BAG3 transgene, aiming to restore cardiac function with lower doses than conventional gene therapies.

Shares of uniQure plummeted more than 40% in premarket trading Monday after the company confirmed the FDA has rejected its current data package for AMT-130, a highly anticipated gene therapy for ...

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

Progeria, an ultrarare and fatal genetic disease characterized by rapid aging, can affect children and young adults.

Recent developments of interest in cardiovascular medicine ...

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 ...

Zahra Mahmoudjafari explains how cell and gene therapy programs succeed with strong infrastructure, pharmacy leadership, ...

Proof-of-concept trial in a single patient shows that cells can survive transplantation without immunosuppression ...

Outpatient migration, bispecific adoption, and non-oncology trial expansion are increasing capacity pressure, making flexible ...

Still, broad uptake has long been an uphill battle for the gene therapy as it competes with a variety of treatments including, Roche’s blockbuster Hemlibra, Sanofi’s newer sales driver Altuviiio and ...