— Received Rare Pediatric Disease and Orphan Drug Designation for Duchenne musculardystrophy (Duchenne) gene therapy candidate SGT-003 with patient dosing in Phase 1/2 trialexpected Q2 2024 — — Company entered into non-exclusive licensing agreement for use of its proprietary, muscle-targeted AAV-SLB101 capsid — — Company ends first quarter 2024 with approximately $206.1 million in cash and investments.Solid has anticipated cash runway into 2026 — CHARLESTOWN, Mass., May 15, 2024 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (NASDAQ:SLDB), a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, today reported financial results for the first quarter ended March 31, 2024, and provided a business update. "In the first quarter of 2024, we continued our focus on executing across our pipeline and we remain on track to initiate patient dosing in our SGT-003 trial in the second quarter of 2024. We anticipate providing a topline readout of safety, microdystrophin expression and functional benefit by year end," said Bo Cumbo, President and CEO, Solid Biosciences. "Supported by the $108.9 million capital raise completed in January 2024, our strong cash position of $206.1 million provides us with both anticipated runway into 2026 and the resources to execute across our portfolio, including bringing SGT-003 for Duchenne muscular dystrophy into the clinic, advancing SGT-501 for catecholaminergic polymorphic ventricular tachycardia (CPVT) towards IND submission and continuing to progress the rest of our diversified pipeline of neuromuscular and cardiac gene therapy candidates." Company Updates Solid expects to initiate dosing in the Phase 1/2 trial of SGT-003 in pediatric patients with Duchenne in the second quarter of 2024. Initial safety, microdystrophin expression and functional benefit data from the first 3-4 patients enrolled in the trial is expected by the fourth quarter of 2024. Solid expects to file an Investigational New Drug Application (IND) for its first cardiac gene therapy candidate, SGT-501 for the treatment of catecholaminergic polymorphic ventricular tachycardia (CPVT), in the first quarter of 2025. Solid continues to advance the rest of its therapeutics portfolio with ongoing preclinical studies in BAG3, mouse studies in TNNT2 and preclinical work in RBM20. Platform enabling work in both capsids (NHP, mouse and pig studies) and portfolio wide CMC process development work is ongoing. Recent Company Highlights On April 1, 2024, Solid announced that the FDA granted Rare Pediatric Disease Designation for its Duchenne gene therapy candidate SGT-003. This designation encourages development of new drugs for the prevention and treatment of rare pediatric diseases. If a new biologics license application (BLA) is approved for SGT-003, the Company may be eligible to receive a priority review voucher. This ...