Solid GTGround-breaking gene therapy platform for Duchenne (IGNITE DMD Clinical Trial)
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. Scroll down for further news about Solid Biosciences.
$1.67 million contribution
Cambridge, MA, USA
The three charities’ critical early support set us up to go down this path. Their investment in Solid GT created the platform that has now enabled us to raise one of the most successful rounds of financing ever in private biotech. For the first time in history, Duchenne muscular dystrophy will have a fully-funded platform to explore gene therapy through a rigorous scientific and clinical process.Andrey Zarur
Recent Solid GT news
Five years ago we identified gene therapy as a viable treatment for Duchenne. Today we are reflecting on the exciting and inspiring events of the past week in the gene therapy arena which are now bringing hope and cautious optimism to the Duchenne community. We have...
Message from Solid Biosciences: "We’re pleased to share with you that the FDA has lifted the clinical hold on IGNITE DMD, our clinical trial investigating SGT-001 as a potential treatment for Duchenne muscular dystrophy (DMD). SGT-001 is a microdystrophin gene...
Solid Biosciences recently reported that it has raised up to $50 million which will enable the biotech to continue to advance its programs for Duchenne muscular dystrophy, including development of a microdystrophin gene. We are forever grateful to our incredible...
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.