Duchenne Muscular Dystrophy Gene Therapy On Hold After Safety Incident


This is the third safety incident in the IGNITE DMD clinical trial that has resulted in a clinical hold since its inception in 2017.

Ilan Ganot

Ilan Ganot

Following the occurrence of a serious safety incident, the FDA has placed a hold on Solid Biosciences’ clinical trial for SGT-001, its gene therapy candidate for Duchenne muscular dystrophy (DMD).1

This is the third safety incident in the IGNITE DMD clinical trial that has resulted in a clinical hold since its inception in 2017.2

In a statement,1 the company said that 1 patient of 3 who were dosed at a 2E14 vg/kg dose in late October experienced a “serious adverse event (SAE) deemed related to the study drug that was characterized by complement activation, thrombocytopenia, a decrease in red blood cell count, acute kidney injury, and cardio-pulmonary insufficiency. Neither cytokine- nor coagulopathy-related abnormalities were observed.” The patient is currently recovering and being monitored by his care team.

RELATED: Gene Therapy Ushers in an Era of Hope for Rare Disorders

To date, 6 patients have been dosed in the phase 1/2 study NCT03368742), which is assessing the safety and efficacy of the novel adeno-associated viral vector-mediated gene therapy that delivers a synthetic, functional version of the dystrophin gene to muscle cells. The one-time, potentially curative treatment would be relevant for all patients with DMD regardless of gene mutation. The candidate has been granted rare pediatric disease designation and orphan drug designation by the FDA.

“In the coming weeks, we anticipate that we will have a better understanding of the biological activity and potential benefit of SGT-001,” Solid Biosciences chief executive officer, president, and co-founder Ilan Ganot said in a statement.1 “We look forward to sharing this additional data and working with the FDA to resolve the clinical hold and determining next steps for the program.”

Earlier this year, the company reported disappointing 3-month biopsy data from its low-dose cohort, which showed low levels of microdystrophin protein expression in the 3 patients dosed.3 At that time, the company said it would engage with the appropriate parties to initiate the planned escalated dose as soon as possible.


1. Solid Biosciences Provides SGT-001 Program Update [news release]. Cambridge, MA: Solid Biosciences. November 12, 2019. investors.solidbio.com/news-releases/news-release-details/solid-biosciences-provides-sgt-001-program-update. Accessed November 12, 2019.

2. Solid Biosciences Reports First Quarter 2019 Financial Results and Provides Business Update [news release]. Cambridge, MA: Solid Biosciences. May 13, 2019. investors.solidbio.com/news-releases/news-release-details/solid-biosciences-reports-first-quarter-2019-financial-results. Accessed November 12, 2019.

3. Solid Biosciences Announces Preliminary SGT-001 Data and Intention to Dose Escalate in IGNITE DMD Clinical Trial for Duchenne Muscular Dystrophy [news release]. Cambridge, MA: Solid Biosciences. February 7, 2019. investors.solidbio.com/news-releases/news-release-details/solid-biosciences-announces-preliminary-sgt-001-data-and. Accessed November 12, 2019.

Related Videos
Ro'ee Gilron, PhD
Monica Verduzco-Gutierrez, MD
Shahid Nimjee, MD, PhD
Peter J. McAllister, MD, FAAN
Video 6 - "Utilization of Neuroimaging in Alzheimer’s Disease"
Video 5 - "Contribution of Multiple Pathways to the Development of Alzheimer’s Disease"
Michael Levy, MD, PhD
Michael Levy, MD, PhD
© 2024 MJH Life Sciences

All rights reserved.