Solid Bio Green on Fast Track Nod
Solid Biosciences Inc. (NASDAQ: SLDB) shares made moderate gains at first Wednesday. The Charlestown, Mass.-based concern, a clinical stage life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, today announced that it received Fast Track designation 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).
This morning’s news release reported 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.
Fast Track designation is granted to products that are developed to treat serious or life-threatening conditions and demonstrate the potential to address unmet medical needs. This designation is intended to facilitate development and expedite review of qualifying drugs. SGT-501 will benefit from this designation through more frequent interactions with the FDA along with the potential to be eligible for priority review.
SGT-501 is an AAV-based gene therapy candidate designed to deliver a functional, full length, codon-optimized copy of the human cardiac calsequestrin (CASQ2) gene to heart muscle cells. In the context of RYR2 variants, increasing CASQ2 protein levels enhances buffering of free calcium in the sarcoplasmic reticulum, stabilizing the RYR2, which results in reduced diastolic calcium leak into the cytosol.
SLDB shares took on 13 cents, or 2.1%, to $6.36.
