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

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

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

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

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 ...

Gene-editing tools like CRISPR have unlocked new treatments for previously uncurable diseases. Now, researchers at the University of British Columbia are extending those possibilities to the skin for ...

The FDA recommended that REGENXBIO run a new study, treat more patients and include a placebo arm to support a resubmission ...

The Food and Drug Administration paused trials for two experimental gene therapies from Regenxbio after one child developed a brain tumor, the company announced Wednesday.

The decision comes two weeks after the agency halted testing due to safety concerns and represents the latest regulatory ...

Black History Month is poignant for one family after a man got a historic, innovative treatment at Children's Hospital of Philadelphia.

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