CANbridge Pharmaceuticals has entered a strategic collaboration and license agreement to gain global rights to LogicBio Therapeutics’ gene therapy candidates for rare diseases with limited treatment options. Under the agreement, CANbridge now owns worldwide rights to develop, manufacture, and commercialize gene therapy candidates for Fabry disease and Pompe…

The U.S. Food and Drug Administration (FDA) has rejected Protalix BioTherapeutics and Chiesi Global Rare Diseases’ request for accelerated approval of PRX-102 (pegunigalsidase alfa) as the first plant-based, every-other-week enzyme replacement therapy (ERT) for adults with Fabry disease. This decision was not related to any concerns with the safety or effectiveness of PRX-102 shown…

More than half of the people with rare diseases and their caregivers, asked in a survey, were undecided or less than willing to be vaccinated for COVID-19 if a vaccine was approved under emergency use authorization instead of the routine process, the EveryLife Foundation for Rare Disease reports. These findings…