Solid Biosciences is positioning itself for a potentially “transformative” year as it builds on its 2025 success and centres its pipeline around novel platforms, aiming to overcome investment headwinds in the gene therapy sector.
Enhanced delivery methods will be key to making gene therapy “really investable again,” said CEO Bo Cumbo at the JP Morgan Healthcare Conference in San Francisco, US, taking place from 12 to 15 January.
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The clinical-stage genetic medicine company has secured more than 50 agreements, including licences, across industry and academia for use of its proprietary capsid AAV-SLB101, said Cumbo.
Investor activity in the cell and gene therapy space has stalled in recent years, with several companies choosing to leave the space in favour of more lucrative modalities. However, the market now appears to be strengthening again and is expected to reach $63bn by 2031, according to a report by GlobalData.
GlobalData is the parent company of Pharmaceutical Technology.
The AAV-SLB101 capsid is designed to bind endocrine receptors on skeletal and cardiac muscle, while reducing liver biodistribution to address associated toxicities. The main downside of AAV vectors used in currently approved therapies is occurrences of liver toxicity.
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By GlobalDataA culmination of multiple incremental changes differentiates the capsid from previous generations, said Cumbo.
Solid’s platform technology in the clinic
Solid Biosciences is using AAV-SLB101 in its one-time gene therapy SGT-003, designed to restore partial function of the dystrophin protein in the X-linked recessive condition Duchenne muscular dystrophy (DMD), which primarily affects males.
Safety concerns have prompted wider discussions in the DMD gene therapy landscape, as Sarepta’s Elevidys was linked to two fatalities in non-ambulatory patients, leading to a label alteration restricting use to ambulatory patients in 2025.
In the Phase I/II INSPIRE (NCT06138639) trial, 33 patients have now been dosed with SGT-003. The therapy has been generally well tolerated with a steroid-only immunomodulation regimen, which Cumbo says differentiates the company from the rest of the field. He emphasised the importance of demonstrating to regulators that the company has not “snooped” the external control data, noting that Solid is well aligned with the FDA’s expectations.
In parallel, Solid has enrolled its first participant in the Phase III randomised placebo-controlled IMPACT trial (NCT07160634). Cumbo said that the company wants to show the FDA that they are “not cutting corners” and remains hopeful of gaining accelerated approval, with plans to start a rolling biologics licence application (BLA) by the end of the year.
Outside the US, he highlighted that Solid has been awarded the UK’s Innovative Licensing and Access Pathway (ILAP) designation, which aims to accelerate time to market and expedite patient access. He added that the company could launch the UK’s first gene therapy for DMD.
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