Solid GT
Ground-breaking gene therapy platform for Duchenne (IGNITE DMD Clinical Trial)The Duchenne Research Fund is proud to have funded one of the first gene therapy research programmes for Duchenne muscular dystrophy, which led to the IGNITE DMD Phase I/II clinical trial currently under way in the United States. Solid Biosciences will continue dosing patients in the first quarter of 2021. Read on to learn more about the background to this ground-breaking research.
In 2014 we combined with Duchenne UK (previously Duchenne Children’s Trust) 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.
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.
Pre-clinical research
$1.67 million contribution
Funded 2014-15
Work ongoing 2014-21
Cambridge, MA, USA
Latest research news
Gavriel Rosenfeld talks to Dr Ronni Cohn about living with Duchenne
On Wednesday 17th March the Duchenne Research Fund hosted a virtual evening giving supporters an update on ongoing research funded by the charity. We were so thrilled that Gavriel Rosenfeld, the son of the DRF’s founders Kerry & Doron Rosenfeld, joined the virtual event …
DRF funds Duchenne clinic at Great Ormond Street
We are pleased to share that we have awarded a grant of £35,000 to Great Ormond Street Hospital to establish a dedicated clinic for children who are non-ambulant (no longer able to walk). The grant supports the post of Senior Physiotherapist Nicola Burnett and has enabled more than 80 patients to be seen …
DRF grants £40,000 to DMD Care UK project
We have awarded £40,000 to the newly launched DMD Care UK, partnering with the project’s founders Duchenne UK and Joining Jack. Together our three charities will invest £130,000 in the nationwide initiative that aims to ensure the best care for everyone living with Duchenne in the UK …
Solid partnership
Click here to learn more about the Duchenne Research Fund’s partnership with Solid Biosciences. You can also watch a short clip where our co-founder Kerry Rosenfeld talks the importance of the Solid GT programme.