Solid Biosciences(US:SLDB) Globenewswire·2025-01-15 21:05Core Insights - Solid Biosciences Inc. is transitioning into a multi-program leader in precision genetic medicines, focusing on neuromuscular and cardiac diseases, with a corporate update scheduled for the J.P. Morgan Healthcare Conference on January 15, 2025 [1][2] Neuromuscular Pipeline - The company has expanded its pipeline to include gene therapy treatments for Duchenne muscular dystrophy (Duchenne) and Friedreich's ataxia (FA) [3][4] - SGT-003 for Duchenne has dosed four patients in the INSPIRE DUCHENNE clinical trial, showing good tolerance with no serious adverse events (SAEs) reported; initial data from three patients is expected in Q1 2025 [4][5] - SGT-212, the first gene therapy utilizing a dual route of administration for FA, has received FDA IND clearance, with a first-in-human clinical study anticipated in 2H 2025 [4][5] Cardiac Pipeline - SGT-501 for catecholaminergic polymorphic ventricular tachycardia (CPVT) is on track for IND submission in 1H 2025, expanding the clinical-stage portfolio to three unique candidates [4][6] - SGT-601 for TNNT2 Thin Filament Cardiomyopathy is undergoing preclinical IND-enabling studies, with anticipated IND submission in 2H 2026 [6] Financial Position - As of December 31, 2024, the company has approximately $148.9 million in cash and investments, expected to fund strategic priorities into 2026 [4] Collaborations and Innovations - Solid has entered a collaboration with Mayo Clinic to develop an AAV gene therapy platform for genetic cardiomyopathies and channelopathies, receiving an exclusive license for six undisclosed cardiac gene therapy programs [13] - The company is building a library of enabling technologies, including capsids and promoters, with ongoing preclinical studies for immunomodulation [7][13] Manufacturing and Technology - Solid is focused on manufacturing excellence, with improvements in full-to-empty capsid ratios observed in early-stage cardiac programs [8]