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 channelled considerable funds from both our 2013 and 2016 fundraising dinners towards gene therapy for Duchenne, and are extremely grateful to you, our incredibly generous donors, for your continued support! Partnering with Duchenne UK (formerly the Duchenne Children’s Trust) and Joining Jack back in 2014, together we invested $5 million to seed-fund Solid Biosciences’ ground-breaking gene therapy platform.

We were excited about gene therapy because, unlike other treatments that were in pre-clinical development at that time, it has the potential to help all boys with Duchenne – regardless of type of mutation, age or stage of disease.

Fast forward to 2018: it has been a big year for gene therapy, with Solid Biosciences, Sarepta Therapeutics and Pfizer all dosing their first patients in three separate clinical trials.

Last week, Solid announced that the FDA has lifted the clinical hold on IGNITE DMD, its Phase I/II clinical trial for its investigational microdystrophin gene transfer for the treatment of Duchenne. Solid can now continue to dose patients in this trial. We are proud to have supported the development of this exciting treatment and to have played our small part in bringing it to the clinic.

Also last week, Sarepta Therapeutics announced positive preliminary results from three of its patients dosed in its Phase 1/2a gene therapy clinical trial, conducted by Jerry Mendell MD at Nationwide Children’s Hospital in Ohio. All three children showed that they were producing microdystrophin in their muscle fibres. They also saw a significant drop in the enzyme creatine kinase (CK) which is indicative of muscle damage. No serious side effects were seen.

We are very encouraged by these recent events and want to thank you all for your continued interest and for raising vital funds for Duchenne research.

There is still a lot of work to do to ensure the success of all these treatments and their eventual availability to as many children and young adults with Duchenne as possible. We will continue to update you when there is further news.

In the meantime, we have been focused on funding research programmes that we believe will be vital in complementing gene therapy and ensuring its success now and for future generations. We have so far invested more than half a million pounds in biomarker projects that aim to help accelerate gene therapy research and trials. In addition, we have some exciting new projects in the pipeline that we look forward to sharing with you in the coming weeks.

Click here to read more about our projects.