Solid GT

The Duchenne Research Fund is proud to have funded one of the first gene therapy research programmes for Duchenne muscular dystrophy. This led to the IGNITE DMD Phase I/II clinical trial in the United States, using Solid’s first-generation gene therapy candidate SGT-001. Data published from the trial in March 2022 showed patients have improved motor and lung function two years on from receiving the treatment, compared with the declines that would be expected without treatment.

Building on Solid’s significant progress in advancing gene therapy for Duchenne, the company announced in 2022 it will now focus on its next-generation gene therapy candidate, SGT-003, which uses a novel next-generation capsid to deliver the microdystrophin protein. In pre-clinical studies of SGT-003 performed in mouse models and in vitro in human Duchenne cell lines, multiple fold increases in microdystrophin expression have been observed, compared with the first-generation candidate.

Enrollment has now concluded on the IGNITE DMD trial, although patients will continue to be followed up several years from their initial dose. Solid aims to begin dosing with SGT-003 in late-2023 – regulatory processes permitting.

Read on to learn more about the background to this ground-breaking research.

In 2014 we collaborated with Duchenne UK and Joining Jack to jointly invest $5 million into Solid Biosciences, a US-based disease-focused biotech company, for the Solid GT project, which aims to advance gene therapy as a treatment for all boys with Duchenne.

Gene therapy is currently one of the most exciting prospects for halting the progression of Duchenne muscular dystrophy. Existing steroid treatments only attempt to slow the progression of the disease. However, gene therapy involves delivering a therapeutic gene into sufferers’ cells to replace the faulty gene, with the hope of stopping disease progression. There has been significant success in the use of gene therapy in the last decade to treat haemophilia, Parkinson’s disease and some cancers.

The collaboration between the three UK charities was formed as a limited company under the name of DRIL (Duchenne Research (UK) Investment Limited) which invested the money to help find an effective gene therapy treatment and enable its passage to clinical trial as quickly and safely as possible. In November 2015, Solid GT received $42.5 million of funds from leading biotech and life science investors – a resounding endorsement of the work the three charities instigated.

In 2017, the company received a further $50 million of investment to help take the program forward into clinical trial. It was decided it was prudent to realise some of the investment in Solid Biosciences in order to continue to support and encourage other avenues of research and programmes as advised by our Scientific Advisory Board. The remainder of the holdings in Solid Biosciences were retained.

In 2018 the IGNITE DMD clinical trial in humans commenced: a Phase I/II clinical trial evaluating the SGT-001 microdystrophin gene therapy as a potential treatment for Duchenne. Preliminary results were published in 2019, with Solid announcing their intention to dose escalate. Results published in 2022 showed improved motor and lung function in patients, two years on from treatment.

In 2020 Solid announced a strategic collaboration with Ultragenyx Pharmaceutical, combining their expertise to develop additional gene therapies for patients living with Duchenne muscular dystrophy.
Click here for the latest Solid press release.

In September 2022 Solid announced its intention to acquire AavantiBio to create a genetic medicine company focused on neuromuscular and cardiac rare disease, with a continued, strong focus on Duchenne. This merger completed in December 2022. Solid will now focus its Duchenne research efforts on its next-generation gene therapy candidate, SGT-003.Click here to read the full press release.