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.

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 ...

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

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

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.

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

Insmed has launched a first-in-human Phase 1 trial testing its experimental ALS gene therapy INS1202 in people with the disease.

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 ...

After the FDA’s first-ever public listening meeting on data-sharing in the cell and gene therapy space, new draft guidance aims to standardize the practice. But recent decisions call into question ...