Solid Biosciences Outpaces Competitors In Duchenne Gene Therapy Development (CORRECTED)

Editor’s Note: This article has been corrected to reflect that Truist initiated coverage of Solid Biosciences on Wednesday.

On Wednesday, Truist initiated coverage on Solid Biosciences Inc. SLDB, a clinical-stage biotech developing gene therapies for diseases of the skeletal and cardiac muscles.

Truist initiated with a Buy rating and a price target of $16.

The new unified platform combines Solid Biosciences’ expertise in designing microdystrophin (uDys) transgenes with AavantiBio’s advanced AAV manufacturing process. Analyst Joon Lee suggests this could create a top-tier gene therapy for Duchenne muscular dystrophy, SGT-003.

Also Read: Solid Biosciences Positioned For Next-Gen Duchenne Muscular Dystrophy Treatment Success: Analyst

The analyst highlights that an interim review of SGT-003 human trial data, expected in the first quarter of 2025, could reduce risks for Solid Biosciences' lead Duchenne muscular dystrophy program and its broader musculoskeletal gene therapy platform.

Additionally, the company plans to submit a second investigational new drug application for Catecholaminergic polymorphic ventricular tachycardia in the first half of 2025.

The Truist analyst adds that Solid Biosciences is ahead of Kate Therapeutics and Novartis AG NVS in developing DMD treatments, as their programs are still in preclinical stages. Solid Biosciences' SGT-003 has the potential to stand out thanks to its unique uDys design, which includes a nitric oxide synthase domain that could offer additional benefits.

Analyst Lee highlights that the field of DMD gene therapy is highly competitive, with approved treatments and several others in clinical trials. However, Solid Biosciences stands out with its unique approach, combining an advanced payload (uDys+nNOS) and a specialized delivery method (AAV-SLB101).

During a recent FDA-hosted meeting, parents of DMD patients voiced concerns that existing treatments, like Sarepta Therapeutics Inc’s SRPT Elevidys—the only approved gene therapy for DMD—are not enough.

“Based on the recent FDA hosted meeting where parents of DMD patients expressed the need for better options, we think there’s not only room but a need for better gene therapies,” the analyst said.

On Wednesday, the FDA cleared the company’s Investigational New Drug application for SGT-212 for Friedreich’s ataxia, a degenerative disease caused by insufficient frataxin protein levels.

Phase 1b trial initiation is expected in the second half 2025.

Price Action: SLDB stock is up 4.7% at $4.10 at last check Wednesday.

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