News

CANbridge Acquires Rights to Gene Therapy Candidates

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…

FDA Rejects Quick Approval of PRX-102, Plant-based ERT for Adults

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…

‘Break a Sweat for Fabry’ and Other Activities Mark Awareness Month

Activities ranging from sharing educational posters to “breaking a sweat” are underway to mark Fabry Disease Awareness Month, set aside each April to call attention to this rare genetic disorder. Awareness and education are crucial to increasing the recognition, diagnosis, understanding, and management of Fabry disease, estimated to affect about…