AVALA Hospital, in partnership with Retina Associates New Orleans, has successfully performed Louisiana’s first ENCELTO procedure, a cell-based gene therapy for adults with macular telangiectasia type ...

Hemophilia, a genetic bleeding disorder that prevents the body from clotting blood, can turn a simple paper cut into a deadly bleeding episode. But Indiana residents suffering from the disease now ...

The first gene therapies approved to treat sickle cell disease in December 2023 are struggling on the market. But there are glimpses of forward momentum as Vertex and Genetix Bio provide updates.

The special properties of methylcellulose foam could make it a vehicle for bedside genetic engineering, according to a proof-of-principle study from bioengineers at Fred Hutch Cancer Center. In the ...

A revolutionary treatment brings new hope to those suffering from a devastating rare skin disorder, while showcasing an innovative funding model that could transform rare disease research In a ...

A senior FDC official said the product is a “failed therapy” and confirmed that regulators have asked uniQure to run a new trial to prove the therapy actually helps Huntington’s patients. The ...

Regeneron has posted updated clinical data on DB-OTO, providing more evidence of the gene therapy’s potential to treat a rare genetic form of deafness as it plans for a potential FDA filing later this ...

Gene therapy achieved its first major breakthroughs in the early 1990s with the treatment of a rare disease called severe combined immunodeficiency. However, the field was dealt a serious setback in ...

The U.S. Food and Drug Administration (FDA) has chosen Dr. Vinay Prasad, a professor at the University of California-San Francisco, to lead its Center for Biologics Evaluation and Research. An ...