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news.Solid Biosciences has received the US Food and Drug Administration’s (FDA) rare pediatric disease designation for its gene therapy candidate for Duchenne muscular dystrophy (Duchenne), SGT-003.
The trial will involve two cohorts, with cohort 1 including DMD patients aged four to less than six and cohort 2 with patients aged six to less than eight. Credit: Kelly Sikkema/Unsplash.
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Duchenne is a genetic disorder, primarily affecting boys and is known to be fatal.
This disease is claimed to affect roughly one in every 3,500 to 5,000 live male births, while its prevalence in the US alone is estimated to be between 5,000 and 15,000 cases.
Solid Biosciences’ SGT-003 is designed to deliver a microdystrophin gene, which encodes for a truncated version of the dystrophin protein, essential for muscle function.
The proprietary capsid, ‘AAV-SLB101’, used in SGT-003 may help in enhancing muscular function and the durability of the treatment’s benefits.
The upcoming Phase I/II trial, named INSPIRE Duchenne, will assess the safety and tolerability of SGT-003 in paediatric patients suffering from DMD at 1E14vg/kg dose.
INSPIRE Duchenne is also the first-in-human, open-label, as well as multi-centre trial, the company noted.
The trial will involve a one-time intravenous infusion of SGT-003 in two separate cohorts, each comprising a minimum of three patients, with the possibility of expanding the cohorts.
Cohort 1 will include DMD patients aged four to less than six, while cohort 2 will include those aged six to less than eight.
By mid-2024, the company is planning to release an initial safety update related to the first three to four patients enrolled in the INSPIRE Duchenne trial.
It will be followed by the company providing the initial expression and functional data from these patients in the fourth quarter of this year.
Solid Biosciences president and CEO Bo Cumbo said: “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.”
