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

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

The FDA in July last year declined to approve UX111 for Sanfilippo syndrome, a rare neurodegenerative disorder, citing ...

The Food and Drug Administration has rejected a rare-disease gene therapy from Regenxbio, the company said Monday.

Two dose-limiting toxicities have been recorded in uniQure’s trial evaluating an investigational AAV gene therapy designed to ...

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

Among them was David Liu, a biochemist at the Broad Institute and Harvard University in Cambridge, Massachusetts. Liu isn’t a ...

Investing.com -- REGENXBIO (NASDAQ:RGNX) stock fell 27% after the company received a Complete Response Letter (CRL) from the U.S. Food and Drug Administration (FDA) rejecting its gene therapy RGX-121 ...

Delivery of therapeutic genes is essential for gene therapy. Adeno-associated viruses (AAVs) are a prime vector for carrying ...

Gene therapy holds the promise of preventing and curing disease by manipulating gene expression within a patient's cells. However, to be effective, the new gene must make it into a cell's nucleus. The ...

University of California San Diego-led team has discovered that restoring a key cardiac protein called connexin‑43 in a mouse ...

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