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

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

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 ...

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, ...

Shares of uniQure plummeted more than 40% in premarket trading Monday after the company confirmed the FDA has rejected its current data package for AMT-130, a highly anticipated gene therapy for ...

The trial clearance for Shanghai-based Unixell is another step forward for the rapidly advancing Chinese biotech ecosystem, ...

Ultragenyx Pharma (RARE) stock is in focus as gene therapy DTX301 met its main goal in a late-stage trial for the most common ...

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 ...

Personalized therapies for diabetes, while showing promise in international studies, is met with healthy skepticism from ...

What if the darkness that enveloped a child's world suddenly… vanished? What if hope, once a whisper, roared into life with a ...

This time, her baby did test positive for SMA type 1, a severe neurodegenerative genetic condition that typically results in ...