Solid Biosciences announced that its investigational gene therapy for Duchenne muscular dystrophy, SGT‑003, has been awarded a UK Innovation Passport under the newly relaunched Innovative Licensing and Access Pathway (ILAP). The passport is the entry point for ILAP, a streamlined, end‑to‑end regulatory and market‑access route that brings the company into early, structured dialogue with the MHRA, NICE, the Scottish Medicines Consortium and other UK health‑technology assessment bodies.
ILAP is designed to accelerate the development and patient access to transformative new medicines by facilitating early collaboration with regulators and HTA agencies. By joining ILAP, Solid can work directly with UK authorities to shape its regulatory roadmap, potentially reducing the time required to obtain approval and secure reimbursement for patients in the UK market. SGT‑003 is one of only three investigational medicinal products to join the relaunch, giving Solid a rare opportunity to influence the pathway and gain a first‑to‑market advantage.
SGT‑003 is a next‑generation AAV gene product that delivers a proprietary microdystrophin construct via a novel AAV‑SLB101 capsid engineered to target muscle tissue more effectively while reducing liver uptake. Nonclinical studies have shown enhanced cardiac and skeletal muscle transduction and a favorable safety profile, positioning the therapy as a promising candidate for a disease with no curative treatment options.
The therapy is currently being evaluated in the Phase 1/2 INSPIRE DUCHENNE trial, which is enrolling participants in the United States, the United Kingdom, Italy and Canada. Interim data from the trial, released in February 2025, show that six participants have been dosed, with an average microdystrophin expression of 110% by western blot and measurable improvements in muscle health biomarkers. Solid has also activated its first clinical trial site and is screening patients for the upcoming IMPACT DUCHENNE Phase 3 study, an ex‑US, randomized, double‑blind, placebo‑controlled trial that will further assess safety and efficacy in a larger cohort.
By securing the Innovation Passport, Solid positions itself to potentially become the first DMD gene therapy approved in the UK, accelerating patient access and creating a first‑to‑market advantage that could translate into a significant commercial opportunity for the company. The ILAP designation also signals confidence from UK regulators and HTA bodies, which may smooth the reimbursement process once the therapy receives regulatory approval.
Chief Regulatory & Preclinical Operations Officer Jessie Hanrahan said, "Receiving the Innovation Passport designation is further recognition of SGT‑003’s potential to transform the treatment paradigm for those living with Duchenne. The early engagement with UK authorities will help us navigate the regulatory and reimbursement landscape more efficiently, bringing the therapy to patients faster."
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