In a world-first trial, scientists used a one-off CRISPR gene edit to switch off a liver “fat brake” gene, slashing stubborn LDL cholesterol and triglycerides in patients whose levels refused to budge ...

The Gene Therapy R&D market presents robust opportunities, driven by clinical advancements in CAR-T therapies and a strong pipeline, particularly for cancer and rare diseases. Despite challenges from ...

Revolutions in gene therapy are rapidly changing the landscape of modern medicine. Revolutions in gene therapy are rapidly changing the landscape of modern medicine, forcing society and science alike ...

Adeno-associated virus (AAV) has emerged as a reliable vehicle for delivering therapeutic DNA into patient cells, with eight AAV-based gene therapies approved worldwide as of mid-2025. 1 Yet questions ...

An experimental gene therapy seems to slow the progression of Huntington’s disease by about 75 per cent, and researchers are ...

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 ...

The field of cell and gene therapy has seen a wave of exciting advances in the past 12 months. Following the landmark approval of the first CRISPR-based cell therapy in late 2023, investigators have ...

Until recently, even the most advanced gene therapies could only be given after a child was born—often racing against time to prevent irreversible damage. In the first part of this series, we explored ...

Researchers have found a promising new method for gene therapy. They successfully restarted inactive genes by bringing them closer to genetic switches on the DNA called enhancers. The intermediate ...

Boston biotech Beam Therapeutics has announced initial data from a phase 1/2 trial of its gene therapy for a genetic lung and liver disease, with no serious adverse events reported among the nine ...

Following the apparent elimination of a late-stage gene therapy study last week, Swiss drug juggernaut Roche has confirmed that it’s axing a hemophilia candidate inherited in its 2019 buyout of Spark ...