Gene therapy represents a paradigm shift in modern medicine, offering unprecedented opportunities to combat diseases at their ...

Vincent Gaynor remembers, almost to the minute, when he realized his part in birthing the breakthrough gene therapy Zolgensma ...

Dravet syndrome and other developmental epileptic encephalopathies are rare but devastating conditions that cause a host of ...

The high cost of sickle cell gene therapies is pushing private insurers and Medicaid programs to develop new payment models.

Researchers have demonstrated, for the first time in the world using mice, the ability to overcome significant challenges in ...

Cell and gene therapies can be life-changing for patients with certain conditions, but the process of receiving them poses barriers for patients and caregivers that require multistakeholder solutions, ...

There are some challenges associated with gene therapy -- including ... about certain aspects of gene therapy -- particularly the safety of the process and the side effects of chemotherapy ...

Researchers have developed a new, highly effective “gene switch” to deliver targeted cell therapy. The ETH Zurich team states that this cell therapy has the potential to offer a more precise and ...

PHC Corporation has signed a Master Collaboration Agreement with CCRM to work together on the development of primary ...

Clinical trial complexity, logistical challenges associated with treatments, and resource constraints at trial sites cited as top factors that ...

A one-time injection of Lumevoq demonstrated sustained visual acuity improvements with a favorable safety profile at 5 years ...