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.

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

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

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 ...

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.

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

Researchers at the University of Basel have developed a gene therapy that could potentially treat a rare and currently fatal ...

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

Dr. Paula Cannon is pursuing one of medicine’s most difficult goals: a cure for HIV.

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 ...

Yentli Soto Albrecht has a rare genetic form of ALS and is working on research at Penn that could one day prove life-saving.