After facing a delayed decision deadline and a clinical hold, Regenxbio’s Hunter syndrome gene therapy has been rejected by ...

A gene therapy that patients breathe in has been found to shrink lung tumours by inserting immune-boosting genes into ...

Gene therapy holds the promise of preventing and curing disease by manipulating gene expression within a patient's cells. However, to be effective, the new gene must make it into a cell's nucleus. The ...

Despite high awareness, biosimilars remain on the periphery—particularly in the first-line setting. While many physicians are open to their use in principle, uptake remains modest, constrained in part ...

Researchers in the lab of Hans-Peter Kiem, MD, PhD, at Fred Hutch Cancer Center have devised a method that could one day treat genetic hematologic disorders by correcting how the body makes blood ...

Regenxbio (RGNX) shares fell after the U.S. Food and Drug Administration issued a complete response letter for RGX-121 ...

As we continue our series exploring genetic medicine, both in this story and in Destiny's Child No Longer: Rewriting Genetic Fate, gene therapy stands at a crossroads. On June 16, Sarepta Therapeutics ...

Four patients with infantile-onset Pompe’s disease received a single intravenous injection of an adeno-associated virus serotype 9 vector carrying codon-optimized complementary DNA encoding human acid ...

Researchers have reported that a topical gene therapy which can correct disease-causing mutations directly in human skin, raising the prospect of durable treatments for rare genetic skin ...

All participants had adverse events, but most were moderate in severity ...

Gene-editing tools like CRISPR have unlocked new treatments for previously uncurable diseases. Now, researchers at the University of British Columbia are extending those possibilities to the skin for ...