Alex’s Wish encouraged by Solid Biosciences gene therapy project update

We have received an encouraging update on a gene therapy research project that we helped to fund.

In 2016 the charity invested £50,000 with life science company Solid Biosciences Inc in support of its IGNITE DMD clinical trial study for SGT-001 gene therapy.

Solid’s SGT-001 is a novel adeno-associated viral (AAV) vector-mediated gene transfer therapy designed to address the underlying genetic cause of Duchenne – a serious muscle-wasting disease affecting 1 in every 3,500 boys.

Duchenne is caused by mutations in the dystrophin gene that result in the absence or near absence of dystrophin protein. Data from Solid’s clinical program suggests that SGT-001 has the potential to slow or stop the progression of Duchenne, regardless of genetic mutation or disease stage.

Ilan Ganot, Chief Executive Officer, President and Co-Founder of Solid Biosciences, said : “2022 will be an important year for Solid as we look to advance genetic medicines for Duchenne. We intend to dose additional patients in the IGNITE DMD Phase I/II clinical trial of SGT-001 utilising our updated risk mitigation strategy and second-generation manufacturing process. In the first half of 2022 we expect to share additional data from IGNITE DMD, including data from a ninth patient, who was dosed in November 2021.”

Emma added: “We are so grateful to Solid for their transformative work in seeking new treatments for Duchenne and to all our wonderful supporters who enable Alex’s Wish to help fund this vital research.”

Posted in Funding