Researchers at the Yong Loo Lin School of Medicine, National University of Singapore (NUS Medicine) have developed a revolutionary new method to improve compact gene-editing tools known as base ...

Viral vector gene therapy represents a rapidly advancing field at the intersection of molecular medicine, biotechnology, and clinical translation. Viral ...

Adoptive cell therapy (ACT) has emerged as a transformative approach in cancer immunotherapy, with remarkable clinical success in hematological ...

Drug delivery researchers have vastly improved the potential of genetic therapies by overcoming the challenge of consistently getting genes and gene-editing tools where they need to be within cells.

A gene borrowed from the naked mole rat, an animal famous for extraordinary longevity and disease resistance, helped mice live a little longer and stay healthier, pointing to a surprising biological ...

Dustin Vidrine's sight is disappearing — shrinking, really. He has retinitis pigmentosa, a rare, inherited eye disease that runs in his family. By the time he was in his 20s, he noticed that his ...

Under the new "plausible mechanism" framework, "one well-controlled clinical investigation supported by confirmatory evidence can support approval," said HHS Secretary Robert F. Kennedy Jr. Photo ...

Growing revenues from Casgevy will help CRISPR Therapeutics fund the development of its pipeline therapies. The gene-editing biotech isn't profitable yet. CRISPR Therapeutics has a deep pipeline of ...

Hemophilia usually is an inherited bleeding disorder in which the blood does not clot properly. Patients with severe hemophilia A may have to manage their condition with regular at-home injections or ...

Boehringer Ingelheim is discarding an inhaled gene therapy designed to treat cystic fibrosis (CF) after terminating a phase 1/2 trial. The shuttered study, dubbed Lenticlair 1, failed to produce ...

Cell and gene therapies, or CGT, have come a long way since they were first introduced. In the last few decades, both cell therapy—the transplantation of living cells—and gene therapy—the use of ...

A first-of-its-kind inhalable gene therapy for lung cancer that genetically modifies people’s lung cells has been fast-tracked towards potential approval after promising clinical trial results. “Very ...