Ultragenyx said that the trial of the gene therapy will continue while data from the second primary endpoint is collected.

Drug delivery researchers have vastly improved the potential of genetic therapies by overcoming the challenge of consistently getting genes and gene-editing tools where they need to be within cells.

March 11 (Reuters) - Regenxbio said on Wednesday that interim data from a early-to-mid stage study of its experimental gene ...

A new method for safely inserting large chunks of DNA into genomes has now measured up in mice, potentially paving the way ...

Enh3ance trial results indicate DTX301 reduces ammonia levels by 18% and supports dietary liberalization in patients with OTC deficiency.

Oppenheimer initiated coverage of Ocugen (NASDAQ:OCGN) with an Outperform rating and a $10 price target on Wednesday, ...

CEO Ron Cooper said the company’s focus is advancing its DDX platform, a proprietary non-viral gene therapy technology, with its lead program detalimogene being developed for non-muscle invasive ...

Bayer has discontinued an early-stage clinical gene therapy for a rare genetic disorder in favor of a similar candidate. | Bayer has discontinued an early-stage clinical gene therapy for a rare ...

A new chapter is unfolding for gene therapies in Europe. With retinal programs advancing into Phase I/II trials and ...

A new study has brought scientists one step further in the direction of developing a cure for a brutal group of rare brain disorders known as SYNGAP1-related disorders, or SRDs. Researchers were able ...

Biogen’s salanersen is now heading into three Phase III trials in SMA.

Forbes contributors publish independent expert analyses and insights. A patient in a late-stage gene editing trial for a rare heart condition died from fatal liver complications after receiving an ...