Sarepta’s Elevidys Safety Concerns Open Major Opportunity for Solid Biosciences’ (SLDB) SGT-003 in Duchenne Therapy

Solid Biosciences Inc. (NASDAQ:SLDB) is one of the stocks that will double in 2026. On December 4, Needham analyst Gil Blum initiated coverage of Solid Biosciences with a Buy rating and $16 price target. Blum noted that the recent deaths of two non-ambulatory Duchenne muscular dystrophy patients who received Sarepta’s drug, Elevidys, raise concerns about the treatment’s benefit-risk profile. This safety issue may create a significant opportunity for competing gene therapies, specifically citing Solid Biosciences’ SGT-003. SGT-003 is intentionally designed to minimize liver toxicity while maintaining efficacy, and early trial results for SGT-003 have already shown robust microdystrophin expression.

In its Q3 2025 earnings report, Solid Biosciences disclosed that the company ended the quarter with $236.1 million in cash, cash equivalents, and available-for-sale securities, compared to $148.9 million as of December 31, 2024. This cash position is projected to fund the company’s operational runway into H1 2027. The net loss for Q3 was $45.8 million, up from $32.7 million in Q3 2024, primarily due to an increase in R&D expenses, which rose to $38.9 million from $27.3 million in Q3 2024, mainly driven by increased costs for the SGT-003 program.

Solid Biosciences’ SGT-003 Duchenne gene therapy trial (INSPIRE DUCHENNE) showed strong efficacy as of September 29, with all 10 participants evaluated at Day 90 achieving a mean microdystrophin expression of 58% and key DAPC restoration. The therapy was generally well tolerated with a steroid-only regimen, showing favorable cardiac safety signals and no DILI. The company has dosed 23 participants as of October 31, and plans to meet with the FDA in H1 2026 to discuss accelerated approval pathways after initiating the Phase 3 IMPACT DUCHENNE trial. The pipeline is also advancing, with new Phase 1b trials for FA (SGT-212) and CPVT (SGT-501) starting in Q4 2025.

Solid Biosciences Inc. (NASDAQ:SLDB) develops therapies for neuromuscular and cardiac diseases in the US. In addition, the company develops platform technologies, including capsid libraries, genetic regulators, immunomodulation technologies, manufacturing purity, and dual gene expression.

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Disclosure: None. This article is originally published at Insider Monkey.