Insights:Steps Forward For Duchenne Muscular Dystrophy

We set out to fulfill the need for a company focused solely on solving Duchenne muscular dystrophy (DMD) for every patient, regardless of their genetic mutation or stage of disease. I believe that we have been true to this goal, and will continue to be, because of our promise to the community: to advance only the best science, do things the right way, act with a sense of passion and urgency, and listen to and partner with those who are experiencing the disease firsthand.

2016 was an important year for Solid. We grew our team from six to more than 30 people, achieved important regulatory milestones and opened an office in London. We also generated, and are continuing to generate, a ton of preclinical data to ensure that we know all there is to know about our lead gene therapy compound, SGT-001. It’s what our patients deserve.

Understanding the manufacturing challenges inherent to this program, we also invested heavily in process development and built capabilities that will allow us, upon success in clinical trials, to scale up production and make our treatment widely available to patients.

With this strong foundation in place, our priorities for 2017 are to move SGT-001 into the clinic, grow our pipeline and further build our company with more great talent and resources.

In the second half of the year, we plan to initiate our first clinical study to evaluate the safety and efficacy of SGT-001, a therapy that we believe has the potential to benefit most DMD patients, irrespective of genetic mutations.

We are also working to build our pipeline of therapies that address a range of symptoms associated with DMD. We added two programs to this pipeline in 2016, and walked away from others that didn’t meet our requirements and expectations. Our goal is to generate strong preclinical data for these candidates and move forward only those that impress us most.

I am especially proud of our team at Solid and feel the commitment and passion across our growing company. However, we would not be where we are today without our partnerships with the DMD community. Together, we are working hard to advance the science and are taking important steps forward to solve this terrible disease.

I will keep you updated on our progress.


Ilan Ganot, CEO of Solid Biosciences