Atamyo Therapeutics is seeking approvals in Europe to launch its first trial testing ATA-100, its investigational gene therapy for a…
Articles by Marisa Wexler, MS
The COVID-19 pandemic has had a substantial impact on the mental health and quality of life of people affected by…
The U.S. Food and Drug Administration (FDA) has granted fast track designation to AOC 1001, an investigational treatment for…
Note: This story was updated Oct. 8, 2021, to clarify that Sarepta did not confirm previously announced plans to…
Treatment with the investigational cell therapy CAP-1002 significantly improved arm and heart function in boys and young men at advanced…
BBP-418, an investigational disease-modifying medication that aims to improve muscle strength and function in people with limb-girdle muscular…
A new viral vector that is better than conventional vectors — tools designed to deliver genetic material into cells —…
Benitec Biopharma is taking steps to get the needed regulatory clearances to start clinical testing in Europe and the…
The U.S. Food and Drug Administration (FDA) has granted fast track status to EDG-5506, Edgewise Therapeutics’ investigational oral therapy…
Sarepta Therapeutics has executed an exclusive license agreement for a gene therapy candidate that aims to treat limb-girdle…