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.

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.

The first gene therapies approved to treat sickle cell disease in December 2023 are struggling on the market. But there are glimpses of forward momentum as Vertex and Genetix Bio provide updates.

Despite innovative new research, many cell and gene therapies do not make it all the way to the patients. Researchers and clinicians in Lund have now presented a new model for cooperation that will ...

Revolutions in gene therapy are rapidly changing the landscape of modern medicine. Revolutions in gene therapy are rapidly changing the landscape of modern medicine, forcing society and science alike ...

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

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 ...

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

Taysha Gene Therapies, Inc. (Nasdaq: TSHA) (Taysha or the Company), a clinical-stage biotechnology company focused on ...

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