Sometimes opportunity arrives in the form of tragedy. Recent deaths of two patients with Duchenne muscular dystrophy have raised serious questions about the market-leading gene therapy and created an opening for emerging alternatives like Solid Biosciences Inc. (SLDB) and its experimental treatment SGT-003.
That's the view from Needham analyst Gil Blum, who just launched coverage on Solid with a Buy rating and $16 price target. His optimism hinges on a shifting competitive landscape in the DMD gene therapy space.
A Window Opens
The backdrop matters here. Sarepta Therapeutics Inc. (SRPT) has dominated the DMD gene therapy market with Elevidys, but two recent patient deaths linked to the treatment have cast a shadow over its efficacy. That's created room for competitors with potentially safer profiles.
Enter SGT-003, Solid's gene therapy designed specifically to minimize liver toxicity without sacrificing effectiveness. According to Blum, early results look promising: the treatment shows robust microdystrophin expression with no clinical signs of acute liver injury, and this despite using only a minimal immune suppressive regimen.
Timeline and Catalysts
The regulatory picture should become clearer in the first half of 2026, when updates will determine whether SGT-003 can get an accelerated approval pathway. Blum thinks that outcome seems likely.
Beyond DMD, Solid has Phase I studies planned for the fourth quarter of 2025 in Friedreich's ataxia and catecholaminergic polymorphic ventricular tachycardia, which could create additional value in 2026.
On the financial front, Solid exited the third quarter with $268 million in cash, which Blum believes provides sufficient runway into the first half of 2027.
Shares of Solid Biosciences jumped 10.98% to $5.76 following the analyst note on Wednesday.
