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

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

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

March 11 (Reuters) - Regenxbio said on Wednesday that interim data from a early-to-mid stage study of its experimental gene therapy in patients with Duchenne muscular dystrophy showed continued ...

UCLA researchers use lipid nanoparticles to insert full CFTR gene into airway cells, restoring function and opening new avenues for CF therapy.

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.

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 ...

Researchers at the University of Basel have developed a gene therapy that could potentially treat a rare and currently fatal muscle disease in children. The study shows in animal models that a single ...

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

Solid Biosciences’ SGT-003 is the only late‑stage program to show early cardiac benefit across biomarkers and function, according to William Blair.

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 ...

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