We are thrilled that the money raised through the Duchenne Research Fund’s 2013 dinner and donations since then have enabled us to seed fund the incredible Solid GT project, which has today received industry investment (read the press release below). While gene therapy is an exciting piece of the puzzle for beating Duchenne for all boys, it is now more critical than ever that we continue to raise vital funds to push Duchenne therapies into clinical trial as quickly and safely as possible and to get them to our boys NOW.

Update: Our charity’s involvement and the story behind the founding of Solid GT has also been covered by respected biotech publication Xconomy. Read more here.



Duchenne Research Fund, Duchenne Children’s Trust and Joining Jack are pleased to announce that the $5 million they committed 12 months ago to establish Solid GT has been amplified by $42.5 million of funding from leading industry investors.

Life science asset management firm Perceptive Advisors, LLC and leading global biotech Biogen Inc., together with funds managed by Janus Capital Management LLC and private investors, join the three UK charities in advancing the development of Cambridge, Massachusetts-based Solid GT’s novel gene therapy platform for Duchenne muscular dystrophy (DMD).

Andrey Zarur, Founder and Chairman of Solid Biosciences, said: “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.”

Charity founders Kerry Rosenfeld, Emily Crossley and Alex Johnson said: “Duchenne Research Fund, Duchenne Children’s Trust and Joining Jack are delighted that the partnership we formed with Solid GT just 12 months ago has led to such significant investment from key industry players. This is a resounding endorsement of the incredible work that we are proud to have instigated. The Solid GT programme fits perfectly with our agenda, to partner and enhance ground-breaking drug development efforts and clinical trials, and to find a cure for the whole Duchenne community as quickly and safely as possible.”

About Solid GT:
Solid GT, a subsidiary of Solid Biosciences, is a gene therapy platform focused on developing novel, disease-modifying therapeutics for Duchenne muscular dystrophy (DMD). The company’s licensed technology has demonstrated efficacy in various DMD animal models, and lead candidates are currently undergoing IND-enabling studies. Founded in 2014 Solid GT is collaborating with leading patient organizations, experts, universities, hospitals and gene therapy centers, globally. Please visit www.solidbio.com for more information.