In a worldwide first, a one-and-done gene therapy has been approved to treat a form of hereditary deafness. Many of the ...

The regulatory body wants public feedback on whether the definition should be updated to comprise biological and synthetic products.

The consultation, launched jointly with the Department of Health in Northern Ireland, seeks views on updates to the legal ...

This story is part of a series on the current progression in Regenerative Medicine. In 1999, I defined regenerative medicine as the collection of interventions that restore tissues and organs damaged ...

Encoded Therapeutics gives a heads-up to other gene therapy company about the trade-offs with a drug meant to improve safety.

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 ...

While gene therapy for cystic fibrosis is still in the research phase, researchers aim to correct the defective gene responsible for the disease. Current research is promising. Cystic fibrosis (CF) is ...

The search for gene therapies to treat rheumatoid arthritis (RA) has moved in fits and starts over the past two decades, with trials starting and stopping, and their focus shifting to osteoarthritis ...

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 ...

A planned first-in-human trial aims to treat GM1 gangliosidosis in utero, possibly laying the foundation for prenatal gene ...