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

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

Following over a year of slow uptake, Vertex Pharmaceuticals and CRISPR Therapeutics expect Casgevy revenues to nearly triple in 2026, as patient access to the sickle cell disease and beta thalassemia ...

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 ...

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 ...

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 ...

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

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

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

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

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