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

As Eli Lilly awaits an FDA approval decision for its oral GLP-1 orforglipron, the pharma has discarded three clinical-stage ...

Ultragenyx Pharmaceutical's employees were already braced for changes as the biopharma considered how to rebound from a pair ...

Apertura Gene Therapy, a biotechnology company developing next-generation AAV capsids for delivering genetic medicines, today ...

Gene therapy is a medical treatment that works by changing or fixing a person's genes to treat or prevent disease ...

For most of his 20 years, a New Jersey man knew pain as a daily reality. Now, after a one-time CRISPR gene-editing treatment at Children's Hospital of Philadelphia, Austin Louis says he is finally ...

FDA RMAT boosts inhaled gene therapy KB707, delivering IL‑2/IL‑12 to lung tumors; early trial shows responses with manageable ...

Feb 9 (Reuters) - Regenxbio said on Monday the U.S. Food and Drug Administration had declined to approve its gene therapy for ...

A research team has successfully demonstrated the world's first gene-editing treatment for Leber's hereditary optic ...

Following over a year of slow uptake, Vertex Pharmaceuticals and CRISPR Therapeutics expect Casgevy revenues to nearly triple ...

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

Gene therapy holds the promise of preventing and curing disease by manipulating gene expression within a patient's cells.