Taking Lessons From Hematology Experts in Tampa

According to Ultragenyx, the CRL relates to a need for additional CMC information and improvements and observations from inspections of manufacturing facilities.

Mind Moments®, a podcast from our sister site NeurologyLive®, held an exclusive interview with Rajesh Pahwa, MD.

Derek Jackson, BS, MA, the vice president of cell & gene therapy product development at Pacira BioSciences, discussed findings related to the immunogenicity of the therapy’s high-capacity adenovirus vector.

In Episode 4 of ImmunoLogic, Caroline Diorio, MD, FRCPC, FAAP, discussed her research on adverse events associated with cell therapy.

In the CRL, the FDA stated that the statutory requirement for “substantial evidence of effectiveness” was not met by the BLA and that more clinical data will be needed.

SGT-501 is intended to address CPVT by providing a full-length, codon-optimized copy of the cardiac calsequestrin gene to the muscle cells of the heart.