Full Press Release Details
Dear Duchenne Community,
We are writing to share an important update on INSPIRE DUCHENNE , our Phase 1/2 clinical trial of SGT-003 for the treatment of patients living with Duchenne. At Solid Biosciences, our mission is driven by the needs and hopes of the Duchenne community, and we are grateful for the trust and partnership you have shown us throughout this journey.
We are pleased to share that we have successfully begun administering our next-generation, investigational microdystrophin gene therapy candidate, SGT-003, to patients with Duchenne in the clinical study. To date, dosing has been well tolerated in these patients with no serious adverse events reported. Based on the safety profile seen to date, we are working diligently on applications to expand the INSPIRE DUCHENNE study to additional clinical sites and are in the process of manufacturing additional material to support an expected increase in the number of patients in our current and future trials. As a reminder, we currently have two clinical sites open and enrolling patients – Nationwide Children’s Hospital led by Dr. Kevin Flanigan and UCLA led by Dr. Perry Shieh. We aim to have a total of six clinical sites active and recruiting patients in the fourth quarter of 2024 in North America, including one site in Canada now that our clinical trial application (CTA) has been authorized by Health Canada. We are also planning to activate additional sites in Europe during the first half of 2025. This expansion will allow more families the opportunity to participate in the study and contribute to the advancement of this new, potential treatment option.
We understand how crucial it is for the patient community to stay informed about clinical research, especially when it comes to making educated and important decisions about available treatment options and clinical research participation. Solid Biosciences remains committed to transparency and intends to share initial data from the INSPIRE DUCHENNE study in the fourth quarter of this year. We believe that keeping the community updated is essential to our mission of improving the lives of those affected by Duchenne.
We deeply value the partnership we have with the Duchenne community and recognize the vital role that families play in the development of new treatments. Your participation in clinical research is paramount, and we are immensely grateful to all the families who choose to be a part of this important work. As we continue to progress, we will keep you updated through our extensive network of advocacy partners around the globe.
Please feel free to reach out to our Patient Advocacy team with any questions: ClinicalTrials@www.solidbio.com
For more information on the INSPIRE DUCHENNE study:
Sincerely,
The Solid Biosciences Team