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

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced screening and enrollment are underway in Cohort 8 of ENDEAVOR (Study 9001-103).

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

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 ...

The Advanced Research Projects Agency for Health ( ARPA-H) awarded BioCurie up to $9.3 million to accelerate the development of scalable, data-driven genomic medicine production.

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 ...

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

A pioneering gene therapy could help treat a rare seizure disorder called Dravet syndrome, according to new clinical trial ...

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

By Christy Santhosh March 2 (Reuters) - Intellia Therapeutics said on Monday the U.S. Food and Drug Administration has lifted the clinical hold on a late-stage trial of its gene therapy for a heart ...

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

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