Solid Biosciences (SLDB) announced that it received Fast Track designation, FTD, from the U.S. Food and Drug Administration, FDA, for SGT-501, the Company’s novel, AAV-based investigational gene therapy for the treatment of catecholaminergic polymorphic ventricular tachycardia, CPVT. SGT-501 will deliver a functional, full-length, codon-optimized copy of the human cardiac calsequestrin, CASQ2, gene to cardiac muscle cells, an approach intended to address the underlying ryanodine receptor, RYR2, instability and calcium dysregulation seen in CPVT, thereby normalizing cardiac rhythm in diastole.
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