UK grants innovation passport to Solid's Duchenne gene therapy

CHARLESTOWN, Mass. - Solid Biosciences Inc. (NASDAQ:SLDB) announced Thursday that its investigational gene therapy for Duchenne muscular dystrophy, SGT-003, has received an Innovation Passport designation under the UK's Innovative Licensing and Access Pathway (ILAP).

The designation provides Solid with an accelerated regulatory pathway in the UK, enabling the company to work directly with the National Health System, the Medicines and Healthcare products Regulatory Agency, and various health technology assessment bodies.

SGT-003 is among the first three investigational products to join the relaunched ILAP program, which focuses on transformative treatments addressing unmet clinical needs.

"Receiving the Innovation Passport designation is further recognition of SGT-003's potential to transform the treatment paradigm for those living with Duchenne," said Jessie Hanrahan, Chief Regulatory & Preclinical Operations Officer at Solid Biosciences.

The therapy is currently being evaluated in the Phase 1/2 INSPIRE DUCHENNE clinical trial across sites in the US, UK, Italy and Canada. Additionally, Solid has begun screening participants for IMPACT DUCHENNE, an ex-US Phase 3 randomized clinical trial.

Duchenne muscular dystrophy is a progressive, irreversible genetic disease primarily affecting boys, with symptoms typically appearing between ages three and five. The condition affects approximately one in every 3,500 to 5,000 live male births.

SGT-003 contains a microdystrophin construct and a proprietary next-generation capsid designed to target integrin receptors. The therapy's design includes the R16/17 domain, which localizes nNOS to muscle tissue.

The information in this article is based on a company press release statement.

In other recent news, Solid Biosciences has entered into a non-exclusive worldwide license agreement with Kinea Bio for its proprietary AAV-SLB101 capsid. This agreement allows Kinea Bio to utilize the capsid as a delivery backbone for KNA-155, a gene therapy targeting limb-girdle muscular dystrophy type 2B/R2, which is moving into preclinical activities. Analysts have also weighed in on Solid Biosciences, with Cantor Fitzgerald reiterating an Overweight rating and maintaining a $16.00 price target, highlighting the company's Duchenne muscular dystrophy program. The program uses a novel capsid that targets muscle tissue and reduces liver uptake, showing promising early data. JMP Securities similarly reiterated a Market Outperform rating with a $15.00 price target, emphasizing the progress in Solid Biosciences' gene therapy programs for multiple conditions. The firm noted that 15 DMD patients have been dosed ahead of schedule, with plans for an FDA meeting in 2025 to discuss regulatory pathways. These recent developments reflect ongoing advancements in Solid Biosciences' therapeutic initiatives.

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