Skip to content
Solid Biosciences Logo

Solid Biosciences: SGT-003 has been granted Rare Pediatric Disease, Orphan Drug, and Fast Track Designations by the FDA in the U.S.

April 1, 2024

Defeat Duchenne Canada is pleased to share the news that Solid Biosciences SGT-003 has been granted Rare Pediatric Disease, Orphan Drug, and Fast Track Designations by the FDA in the U.S.

Solid Biosciences Inc. today announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation for SGT-003, the company’s next-generation Duchenne muscular dystrophy (Duchenne) gene therapy candidate. Rare Pediatric Disease Designation is granted by the FDA for serious or life-threatening diseases in which manifestations primarily affect children ages 18 years and younger. In addition, the disease must affect fewer than 200,000 people in the United States.

“Solid’s receipt of Rare Pediatric Disease Designation for SGT-003 highlights the continuing need for transformational treatments for this devastating disease.”

“The key components of SGT-003 were rationally designed to improve on first generation gene therapies to provide skeletal muscle tropism, enhanced durability, and improved clinical outcomes. With site activation scheduled in April, and patient screening beginning shortly thereafter, we anticipate dosing patients in Q2 of this year.”

Bo Cumbo, President and Chief Executive Officer at Solid Biosciences.

The planned Phase 1/2 trial, INSPIRE Duchenne, is a first-in-human, open-label, multicenter trial to determine the safety and tolerability of SGT-003 in pediatric patients with DMD at a dose of 1E14vg/kg. SGT-003 will be administered as a one-time intravenous infusion to patients in two cohorts with a minimum of three patients each, with the potential for cohort expansion. Cohort 1 will study patients with DMD ages 4 to < 6 and cohort 2 will study patients with DMD ages 6 to < 8. We anticipate providing an initial safety update for the first three to four patients enrolled in the INSPIRE Duchenne trial in mid-2024, and we anticipate providing initial expression and functional data from those patients in the fourth quarter of 2024.

“Preclinical data suggests that SGT-003 has potential to significantly improve on existing treatments for Duchenne by using a muscle tropic proprietary capsid to deliver a DNA sequence encoding a shortened form of the dystrophin protein which, importantly, includes the nNOS binding domain. nNOS is believed to play a crucial role in both muscular function and endurance.”

“We look forward to rapidly bringing SGT-003 to the clinic and hope to all Duchenne patients in need.”

Dr. Gabriel Brooks, M.D., Chief Medical Officer at Solid Biosciences.

Read the full press release below.

About SGT-003

SGT-003 uses a proprietary, rationally designed capsid (AAV-SLB101) to deliver a DNA sequence encoding a shortened form of the dystrophin protein (microdystrophin), containing the R16-R17 nNOS binding domain. Preclinical data suggests this may be important for both muscular function and durability of benefit in patients.

Related Articles